Sangamo does not believe Cellectis' CRISPER patent applies to Sangamo ZFN technology.
CRISPR patent claimant Cellectis has declined to comment on rumours US drug giant Pfizer is in talks about a €1.5bn takeover.
Friday, May 29, 2015
Sangamo patent question mentioned in Cellectis/Pfizer merger article
From Biopharma-reporter:
Sangamo does not believe Cellectis' CRISPER patent applies to Sangamo ZFN technology.
Sangamo does not believe Cellectis' CRISPER patent applies to Sangamo ZFN technology.
Tuesday, May 26, 2015
Jefferies June 1st 1:30 EST
Sangamo BioSciences Announces Presentation At The Jefferies 2015 Global Healthcare Conference
Friday, May 22, 2015
ARM and ASGCT announce new partnership
Ed Lanphier of Sangamo BioSciences (SGMO) is co-chair of new Gene Therapy section.
http://www.steamfeed.com/gene-therapy-section-formed-within-the-alliance-for-regenerative-medicine/
Washington, DC and Salt Lake City, UT (PRWEB) May 20, 2014 The Alliance for Regenerative Medicine (ARM) and the American Society of Gene & Cell Therapy (ASGCT) today announced their partnership. ARM’s new Gene and Gene–Modified Cell Therapy Section (GTS) brings together the leading gene therapy companies and organizations in the U.S. and Europe to advocate for policies and programs to accelerate the development of new therapeutics to treat and cure a range of diseases for which no effective treatment options are available. ARM’s focus on advocacy and clinical and commercial development will create a powerful alliance with the deep scientific and translational expertise resident in ASGCT.
The new ARM Gene Therapy Section is one of three technology sections that comprise the ARM membership. The other two sections focus on Cell Therapy and Tissue Engineering and Biomaterials. The Gene Therapy Section will dedicate its efforts to addressing regulatory, manufacturing, commercial and financial issues crucial to the success of the sector. In addition, the GTS will focus on building public awareness for this field of medicine and an appreciation for its potential to transform healthcare. The GTS will be led by Sarah Haecker, Ph.D., a member of ARM’s senior staff. Sarah received her Ph.D. in Molecular Biology and Bioethics (with focus on gene transfer applications) and her postdoctoral scientific and business training in the Human Gene Therapy Program and the Center for Technology Transfer at the University of Pennsylvania. The three co-chairs of the group are Edward Lanphier, President, Chief Executive Officer, Sangamo BioSciences; Jeffrey Walsh, Chief Operating Officer, bluebird bio; and Karen Kozarsky, Ph.D., Vice President, Research & Development, ReGenX Biosciences.
“As ARM’s membership has grown, the organization has created specific technical sub-groups, such as the GTS, to focus on the unique development and commercialization needs of major sectors within advanced therapies,” said Lanphier. “The addition of this new technology section is particularly exciting as the gene therapy field is making tremendous progress and holds great promise for transforming the lives of so many patients. It is our hope that gathering this group of technical, clinical and commercial experts in the field will help to accelerate product development and commercialization of these innovative technologies.”
“ASGCT’s mission to bring together diverse stakeholders and advance the field of genetic and cellular therapies is closely aligned with ARM’s goals, and we are looking forward to working with the organization,” said Harry L. Malech, M.D., President-Elect of ASGCT. “ARM’s staff and members bring a wealth of knowledge involving regulatory and commercialization challenges in the field, and we feel this will nicely complement ASGCT’s scientific and medical expertise.”
ARM is the leading advocacy organization in the U.S. and Europe representing companies and organizations focused in the regenerative medicine and gene therapy field, and serves as an invaluable resource for all of its members. ASGCT, a nonprofit medical and scientific organization focused on genetic and cellular therapies, recently became a member of ARM and the two groups will work together to lead the advocacy and education efforts for the GTS of ARM.
Members of ARM’s Gene Therapy Section:
Abeona Therapeutics, AGTC, Alpha-1 Foundation, ALS Association, Association of Clinical Research Organizations (ACRO), American Society of Gene & Cell Therapy, Baxter/Chatham Therapeutics, Benitec Ltd., bluebird bio, Calimmune, Celgene Corporation, CIRM, Cornell University, Friends of Cancer Research, GenVec, Genzyme-Sanofi, Global Biotherapeutics, Juventas Therapeutics, MaxCyte, Memorial Sloan Kettering Cancer Center, National Multiple Sclerosis Society (NMSS), NeoStem, Oxford Biomedica, Parkinson’s Action Network, Progenitor Cell Therapy (PCT), Pfizer, Prevent Cancer Foundation, ReGenX, Sangamo BioSciences, Shire, SironRX Therapeutics, Stop ALD, TissueGene, UniQure and Voyager Therapeutics
About The American Society of Gene & Cell Therapy
The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website at http://www.asgct.org.
About the Alliance for Regenerative Medicine:
The Alliance for Regenerative Medicine (ARM) is a Washington, DC-based multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, DC to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today ARM has more than 150 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.
http://www.steamfeed.com/gene-therapy-section-formed-within-the-alliance-for-regenerative-medicine/
Washington, DC and Salt Lake City, UT (PRWEB) May 20, 2014 The Alliance for Regenerative Medicine (ARM) and the American Society of Gene & Cell Therapy (ASGCT) today announced their partnership. ARM’s new Gene and Gene–Modified Cell Therapy Section (GTS) brings together the leading gene therapy companies and organizations in the U.S. and Europe to advocate for policies and programs to accelerate the development of new therapeutics to treat and cure a range of diseases for which no effective treatment options are available. ARM’s focus on advocacy and clinical and commercial development will create a powerful alliance with the deep scientific and translational expertise resident in ASGCT.
The new ARM Gene Therapy Section is one of three technology sections that comprise the ARM membership. The other two sections focus on Cell Therapy and Tissue Engineering and Biomaterials. The Gene Therapy Section will dedicate its efforts to addressing regulatory, manufacturing, commercial and financial issues crucial to the success of the sector. In addition, the GTS will focus on building public awareness for this field of medicine and an appreciation for its potential to transform healthcare. The GTS will be led by Sarah Haecker, Ph.D., a member of ARM’s senior staff. Sarah received her Ph.D. in Molecular Biology and Bioethics (with focus on gene transfer applications) and her postdoctoral scientific and business training in the Human Gene Therapy Program and the Center for Technology Transfer at the University of Pennsylvania. The three co-chairs of the group are Edward Lanphier, President, Chief Executive Officer, Sangamo BioSciences; Jeffrey Walsh, Chief Operating Officer, bluebird bio; and Karen Kozarsky, Ph.D., Vice President, Research & Development, ReGenX Biosciences.
“As ARM’s membership has grown, the organization has created specific technical sub-groups, such as the GTS, to focus on the unique development and commercialization needs of major sectors within advanced therapies,” said Lanphier. “The addition of this new technology section is particularly exciting as the gene therapy field is making tremendous progress and holds great promise for transforming the lives of so many patients. It is our hope that gathering this group of technical, clinical and commercial experts in the field will help to accelerate product development and commercialization of these innovative technologies.”
“ASGCT’s mission to bring together diverse stakeholders and advance the field of genetic and cellular therapies is closely aligned with ARM’s goals, and we are looking forward to working with the organization,” said Harry L. Malech, M.D., President-Elect of ASGCT. “ARM’s staff and members bring a wealth of knowledge involving regulatory and commercialization challenges in the field, and we feel this will nicely complement ASGCT’s scientific and medical expertise.”
ARM is the leading advocacy organization in the U.S. and Europe representing companies and organizations focused in the regenerative medicine and gene therapy field, and serves as an invaluable resource for all of its members. ASGCT, a nonprofit medical and scientific organization focused on genetic and cellular therapies, recently became a member of ARM and the two groups will work together to lead the advocacy and education efforts for the GTS of ARM.
Members of ARM’s Gene Therapy Section:
Abeona Therapeutics, AGTC, Alpha-1 Foundation, ALS Association, Association of Clinical Research Organizations (ACRO), American Society of Gene & Cell Therapy, Baxter/Chatham Therapeutics, Benitec Ltd., bluebird bio, Calimmune, Celgene Corporation, CIRM, Cornell University, Friends of Cancer Research, GenVec, Genzyme-Sanofi, Global Biotherapeutics, Juventas Therapeutics, MaxCyte, Memorial Sloan Kettering Cancer Center, National Multiple Sclerosis Society (NMSS), NeoStem, Oxford Biomedica, Parkinson’s Action Network, Progenitor Cell Therapy (PCT), Pfizer, Prevent Cancer Foundation, ReGenX, Sangamo BioSciences, Shire, SironRX Therapeutics, Stop ALD, TissueGene, UniQure and Voyager Therapeutics
About The American Society of Gene & Cell Therapy
The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website at http://www.asgct.org.
About the Alliance for Regenerative Medicine:
The Alliance for Regenerative Medicine (ARM) is a Washington, DC-based multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, DC to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today ARM has more than 150 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.
U of Texas features UT/Sangamo Cystic Fibrosis research first published at ASGCT
https://www.uth.edu/media/story.htm?id=77135222-2d65-44bd-84be-cad1d47a05e9
Scientists correct cystic fibrosis mutation in stem cells
Scientists working on innovative treatments for people with hereditary diseases report they were able to repair genetic mutations responsible for cystic fibrosis in stem cells.
The research led by scientists at The University of Texas Health Science Center at Houston (UTHealth) appeared in Stem Cell Reports, the official journal of The International Society for Stem Cell Research.
Tens of thousands of people worldwide have this life-threatening disease that clogs their lungs with mucus and makes it increasingly hard to breathe. The disease causes chronic bacterial infections in the lung and gradual lung destruction.
The mutations were corrected in stem cells derived from people with cystic fibrosis using zinc finger nuclease-mediated genome editing.
Brian R. Davis, Ph.D., the study’s senior author and director of the Center for Stem Cell and Regenerative Medicine at UTHealth, described the research as promising but early.
“We’ve created stem cells corrected for the cystic fibrosis mutation that potentially could be utilized therapeutically for patients,” he said. “While much work remains, it is possible that these cells could one day be used as a form of cell therapy.”
Even further in the future, Davis said, the cells might be used to generate new organs such as a lung. “This has been done to the hearts and lungs of mice. You basically take the organ, remove the existing cells and reseed the organ scaffolding with new stem cells,” Davis said.
More immediately, Davis believes both the mutant and corrected stem cells could provide scientists with new research tools to evaluate encouraging cystic fibrosis treatments.
People with cystic fibrosis have mutations in a gene called CFTR, producing a defective protein. This interferes with the regular flow of salt and fluids in and out of cells of the lungs. When the flow is impeded, a cascade of problems occurs.
Davis and his colleagues ran tests to demonstrate that the corrected CFTR protein worked normally.
“This study points to a strategy for developing treatments for cystic fibrosis, including patients with rare forms of the disease,” said Eric J. Sorscher, M.D., study co-author and professor of hematology and oncology at the University of Alabama, Birmingham, School of Medicine.
The scientists began the research by taking skin cells from people with cystic fibrosis and converting them into a type of stem cell called an induced pluripotent stem cell. They then corrected the genetic mutation and produced a new line of CFTR mutation-free stem cells.
The genetic correction was accomplished precisely, in many ways similar to the cut and paste correction used in word processors. (ed. note, one would have to assume this was the Sangamo (SGMO) contribution)
Right now, the median age of survival of people with cystic fibrosis is 41 years of age, but many children still die from the disease.
“Cystic fibrosis is one the most common inherited genetic diseases,” said Davis, whose laboratory is in the Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases.
If both parents are carriers for the defective gene, their children have a one in four chance of getting cystic fibrosis.
UTHealth co-authors include Ana M. Crane Ph.D.; Philipp Kramer Ph.D.; Jacquelin Bui-Griffith Ph.D.; Xuan Shirley Li, Ph.D.; Manuel L. Gonzalez-Garay, Ph.D.; Wei Liao; Daniela Mora M.D.; and Sangbum Choi, Ph.D.
Also contributing to the study were: Wook Joon Chung, Ph.D., of the University of Alabama, Birmingham; Finn Hawkins, MBBCh, and Darrell N. Kotton, M.D., of Boston University and the Boston Medical Center; and Jianbin Wang, Ph.D., Helena C. Sun, Ph.D., David E. Paschon, Ph.D., Dmitry Guschin, Ph.D., Philip D. Gregory, D.Phil., and Michael C. Holmes, Ph.D., of Sangamo BioSciences, Inc.
Davis is on the faculty of The University of Texas Graduate School of Biomedical Sciences at Houston, which is operated by UTHealth and The University of Texas MD Anderson Cancer Center. Davis holds the C. Harold and Lorine G. Wallace Distinguished University Chair at UTHealth.
Funding for this study included grants from the National Institutes of Health (RC1HL099559, P30 DK072482) and the Cystic Fibrosis Foundation (CFF Folding Consortium, DAVIS12GO, R464). The study is titled “Targeted Correction and Restored Function of CFTR gene in Cystic Fibrosis Induced Pluripotent Stem Cells.”
Scientists correct cystic fibrosis mutation in stem cells
Scientists working on innovative treatments for people with hereditary diseases report they were able to repair genetic mutations responsible for cystic fibrosis in stem cells.
The research led by scientists at The University of Texas Health Science Center at Houston (UTHealth) appeared in Stem Cell Reports, the official journal of The International Society for Stem Cell Research.
Tens of thousands of people worldwide have this life-threatening disease that clogs their lungs with mucus and makes it increasingly hard to breathe. The disease causes chronic bacterial infections in the lung and gradual lung destruction.
The mutations were corrected in stem cells derived from people with cystic fibrosis using zinc finger nuclease-mediated genome editing.
Brian R. Davis, Ph.D., the study’s senior author and director of the Center for Stem Cell and Regenerative Medicine at UTHealth, described the research as promising but early.
“We’ve created stem cells corrected for the cystic fibrosis mutation that potentially could be utilized therapeutically for patients,” he said. “While much work remains, it is possible that these cells could one day be used as a form of cell therapy.”
Even further in the future, Davis said, the cells might be used to generate new organs such as a lung. “This has been done to the hearts and lungs of mice. You basically take the organ, remove the existing cells and reseed the organ scaffolding with new stem cells,” Davis said.
More immediately, Davis believes both the mutant and corrected stem cells could provide scientists with new research tools to evaluate encouraging cystic fibrosis treatments.
People with cystic fibrosis have mutations in a gene called CFTR, producing a defective protein. This interferes with the regular flow of salt and fluids in and out of cells of the lungs. When the flow is impeded, a cascade of problems occurs.
Davis and his colleagues ran tests to demonstrate that the corrected CFTR protein worked normally.
“This study points to a strategy for developing treatments for cystic fibrosis, including patients with rare forms of the disease,” said Eric J. Sorscher, M.D., study co-author and professor of hematology and oncology at the University of Alabama, Birmingham, School of Medicine.
The scientists began the research by taking skin cells from people with cystic fibrosis and converting them into a type of stem cell called an induced pluripotent stem cell. They then corrected the genetic mutation and produced a new line of CFTR mutation-free stem cells.
The genetic correction was accomplished precisely, in many ways similar to the cut and paste correction used in word processors. (ed. note, one would have to assume this was the Sangamo (SGMO) contribution)
Right now, the median age of survival of people with cystic fibrosis is 41 years of age, but many children still die from the disease.
“Cystic fibrosis is one the most common inherited genetic diseases,” said Davis, whose laboratory is in the Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases.
If both parents are carriers for the defective gene, their children have a one in four chance of getting cystic fibrosis.
UTHealth co-authors include Ana M. Crane Ph.D.; Philipp Kramer Ph.D.; Jacquelin Bui-Griffith Ph.D.; Xuan Shirley Li, Ph.D.; Manuel L. Gonzalez-Garay, Ph.D.; Wei Liao; Daniela Mora M.D.; and Sangbum Choi, Ph.D.
Also contributing to the study were: Wook Joon Chung, Ph.D., of the University of Alabama, Birmingham; Finn Hawkins, MBBCh, and Darrell N. Kotton, M.D., of Boston University and the Boston Medical Center; and Jianbin Wang, Ph.D., Helena C. Sun, Ph.D., David E. Paschon, Ph.D., Dmitry Guschin, Ph.D., Philip D. Gregory, D.Phil., and Michael C. Holmes, Ph.D., of Sangamo BioSciences, Inc.
Davis is on the faculty of The University of Texas Graduate School of Biomedical Sciences at Houston, which is operated by UTHealth and The University of Texas MD Anderson Cancer Center. Davis holds the C. Harold and Lorine G. Wallace Distinguished University Chair at UTHealth.
Funding for this study included grants from the National Institutes of Health (RC1HL099559, P30 DK072482) and the Cystic Fibrosis Foundation (CFF Folding Consortium, DAVIS12GO, R464). The study is titled “Targeted Correction and Restored Function of CFTR gene in Cystic Fibrosis Induced Pluripotent Stem Cells.”
Dow Agro Presentation: Exzact Precision Technology:Scientific and Regulatory Advancements in Plant-Genome Editing with ZFNs
"The combined suite of technologies making up the EXZACT Presision Technology Platform has been developed in several crop species including Maize, soybean,canola and wheat."
External Collaborations
EXZACT Precision Technology is available and accessible both to the public and private
sectors through a Dow AgroSciences’ licensing agreement. Like the private sector public-sector breeders and scientists have significant opportunities to employ Ezact
Precision Technology in their breeding programs, especially in minor crops.
Sciences has entered into several licensing agreements with partners around the world to
develop targeted gene improvements ranging from deletions, edits and gene insertions
in row, community and specialty crops such as maize, canola, cassava, wheat, tobacco,
tomato and forestry trees.
As an example, the Department of Environment and Primary Industries (DEPI) of
the State of Victory, Australia, through its commercial arm, Agriculture Victoria Services
Pty Ltd. (AVS), strengthened a collaborative agreement to improve the performances of
Australian canola varieties. The project uses the EXZACTTM Precision Genome Editing
Technology platform to continue developing new varieties of canola with enhanced
performance designed to benefit farmers in Australia and globally. In addition, AVS will
also use the EXZACTTM Precision Genome Editing Technology platform to enhance the
genetics of crops important to Australian primary producers.
Through valuable collaborative efforts, a variety of improved crop varieties is being
developed with value-adding traits ranging from more-nutritious and insect-resistant
cassava and higher-yielding tomatoes, to oil crops with healthier, improved oil profiles
and crops with improved herbicide tolerance.
Advances in custom ZFN designs, high resolution analytics, novel donor designs, delivery
technologies and genomics will continue to expand the utility of the EXZACTTM
Precision Technology for trait discovery and product development. Benefits of targeting
genes, genomic deletions and edits at desired locations in plant genomes will continue
to be realized, resulting in reduced cycle times and costs for developers while resulting in
improved, high-value crops for the farmer and consumer.
FiercePharma article on Cost Effectiveness of UniQure Gene Therapy Treatment
German cost watchdogs look askance at uniQure's $1.4M gene therapy
http://www.fiercepharma.com/story/german-cost-watchdogs-look-askance-uniqures-14m-gene-therapy/2015-05-21
German cost-effectiveness watchdogs aren't among pharma's favorite people. Since lawmakers instituted a tough price-setting process, state officials have questioned the benefits of one new drug after another--to the point where some drugmakers decided not to launch there at all.
But a debate now playing out there is different. The first of its kind, actually; the Federal Joint Committee is assessing uniQure's ($QURE) Glybera, the first-ever gene therapy approved in Europe, and now the world's most expensive drug at €1.1 million ($1.4 million). And the questions raised in Germany offer a preview of reimbursement conversations in other countries, about this and other uber-pricey gene therapies.
So far, officials are skeptical. The "added value" of Glybera, used to treat a rare metabolic disease that triggers pancreatitis, is "classified as non-quantifiable," the committee said in a Thursday statement. "No scientifically sound statement on the extent of additional benefit is possible based on the data submitted," Germany's Federal Joint Committee said in a statement.
The group asked for more data on safety and efficacy to power a new assessment due next June.
As Bloomberg notes, German insurers set drug prices based on input from government assessors--and this equivocal decision could interfere with UniQure's ability to collect full price for its med. But UniQure and its marketing partner Chiesi hope to use math to prove their case: CEO Jörn Aldag points out that Glybera is given once, and studies show it's effective for 6 years. That's $170,000 per year, less than orphan drugs such as Alexion's ($ALXN) Soliris, which tops $300,000.
Makers of potential gene therapies--and payers, of course--have been mulling over a variety of payment models to make the ultra-expensive treatments affordable, including spreading the cost out over time and making payments contingent on results. In the meantime, though, the early entrants face an uphill battle.
As a gene therapy, however, Glybera isn't 100% representative. UniQure's attempts to win regulatory approval repeatedly faltered on lack of evidence. And now, European regulators are digging back in, worried that the drug doesn't remain effective as long as claimed.
Aldag is philosophical about the current hurdles. "It is often hard to be the first," Aldag told Bloomberg. "This is where people are learning. Not only are we learning, but so are the regulatory agencies."
http://www.fiercepharma.com/story/german-cost-watchdogs-look-askance-uniqures-14m-gene-therapy/2015-05-21
German cost-effectiveness watchdogs aren't among pharma's favorite people. Since lawmakers instituted a tough price-setting process, state officials have questioned the benefits of one new drug after another--to the point where some drugmakers decided not to launch there at all.
But a debate now playing out there is different. The first of its kind, actually; the Federal Joint Committee is assessing uniQure's ($QURE) Glybera, the first-ever gene therapy approved in Europe, and now the world's most expensive drug at €1.1 million ($1.4 million). And the questions raised in Germany offer a preview of reimbursement conversations in other countries, about this and other uber-pricey gene therapies.
So far, officials are skeptical. The "added value" of Glybera, used to treat a rare metabolic disease that triggers pancreatitis, is "classified as non-quantifiable," the committee said in a Thursday statement. "No scientifically sound statement on the extent of additional benefit is possible based on the data submitted," Germany's Federal Joint Committee said in a statement.
The group asked for more data on safety and efficacy to power a new assessment due next June.
 |
| uniQure CEO Jörn Aldag |
Makers of potential gene therapies--and payers, of course--have been mulling over a variety of payment models to make the ultra-expensive treatments affordable, including spreading the cost out over time and making payments contingent on results. In the meantime, though, the early entrants face an uphill battle.
As a gene therapy, however, Glybera isn't 100% representative. UniQure's attempts to win regulatory approval repeatedly faltered on lack of evidence. And now, European regulators are digging back in, worried that the drug doesn't remain effective as long as claimed.
Aldag is philosophical about the current hurdles. "It is often hard to be the first," Aldag told Bloomberg. "This is where people are learning. Not only are we learning, but so are the regulatory agencies."
Sangamo adds new job openings to website
Beefing up the research area and Also Senior manager of Regulatory Affairs.
The following is a list of open positions with Sangamo BioSciences. (SGMO)
(Updated 05/20/15)
Research Associate III - Technology
Research Associate IV - Genome Modification
Scientist - Protein Engineering / Genome Editing
Scientist II - Research
Scientist II - Genome Modification LSD
Associate Director, Project Management
Senior Process Development Engineer
Director, Vector Production
Director, Quality Control
Director, Analytical Methods Development
Development Associate III
The following is a list of open positions with Sangamo BioSciences. (SGMO)
(Updated 05/20/15)
Research
Research Associate - ProductionResearch Associate III - Technology
Research Associate IV - Genome Modification
Scientist - Protein Engineering / Genome Editing
Scientist II - Research
Scientist II - Genome Modification LSD
Development
Senior Manager/Associate Director, Regulatory AffairsAssociate Director, Project Management
Technical Operations
Development Associate IVSenior Process Development Engineer
Director, Vector Production
Director, Quality Control
Director, Analytical Methods Development
Development Associate III
Administrative
Business Analyst/Summer InternThursday, May 21, 2015
Motley Fool Article - sangamo HIV mention
http://www.fool.com/investing/general/2015/05/21/following-hepatitis-c-this-could-be-the-next-disea.aspx
The researchers at Sangamo BioSciences (NASDAQ:SGMO) hope that they may be able to functionally cure HIV patients one day.
The company is using their zinc finger therapeutic approach to modify a key gene receptor known as CCR5 that plays a critical role in HIV's ability to infect T-cells in the immune system. If Sangamo is able to effectively shut down HIV's ability to spread by targeting CCR5, then it could revolutionize patient treatment. Currently, HIV is treated with an array of pills that must be taken daily for the rest of a patient's life and that can cause a bevy of side effects.
In February, researchers presented data showing that Sangamo's SB-728-T reduced viral load below the limit of quantification in one out of three patients. If that can be duplicated or improved upon in larger, later stage trials, then Sangamo could end up delivering on its promise of a potential cure to HIV. However, before we get too excited about this possibility, we need to realize that Sangamo's research remains in the early stages, so it will be a while before we know for certain whether or not this approach will work.
The researchers at Sangamo BioSciences (NASDAQ:SGMO) hope that they may be able to functionally cure HIV patients one day.
Source: GlaxoSmithKline via Facebook.
In February, researchers presented data showing that Sangamo's SB-728-T reduced viral load below the limit of quantification in one out of three patients. If that can be duplicated or improved upon in larger, later stage trials, then Sangamo could end up delivering on its promise of a potential cure to HIV. However, before we get too excited about this possibility, we need to realize that Sangamo's research remains in the early stages, so it will be a while before we know for certain whether or not this approach will work.
BLUE - Presenting new and updated data from HGB-205 study in beta-thalassemia major and severe sickle cell disease, including first patient with sickle cell disease ever treated with gene therapy
From Press Release:
bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, today announced that data from the ongoing Phase 1/2 HGB-205 study of LentiGlobin BB305 Drug Product will be presented in an oral presentation on June 13, 2015 at the 20th Congress of the European Hematology Association (EHA) in Vienna, Austria.
bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, today announced that data from the ongoing Phase 1/2 HGB-205 study of LentiGlobin BB305 Drug Product will be presented in an oral presentation on June 13, 2015 at the 20th Congress of the European Hematology Association (EHA) in Vienna, Austria.
“The early data included in our abstract provide further validation for our approach and important insights into the safety and mechanism of action of LentiGlobin in both beta-thalassemia and sickle cell disease,” said David Davidson, chief medical officer, bluebird bio. “As noted in the abstract, we are pleased to report that the two patients with beta-thalassemia major, on whom we first reported last year at EHA, remained transfusion independent at 14 and 11 months post-transplant. In addition, it is very encouraging that the patient with sickle cell disease is increasing production of HbAT87Q, which has anti-sickling properties, and has not had a post-treatment hospitalization for a sickle cell disease-related event. At EHA we will present further follow up data on all three subjects.”
Abstract Highlights (Data as of February 2015): - Beta-thalassemia: Beta-thalassemia major subjects (1201 and 1202) remained transfusion independent at 14 months and 11 months, respectively
- Sickle Cell Disease: This subject (1204) entered the trial receiving chronic transfusions and began the process of being weaned from transfusions after day 37, receiving the last transfusion on day 88
- Increasing production of HbAT87Q; the first-ever SCD patient treated with gene therapy (subject 1204) had a HbAT87Q level of 24% at 4.5 months follow up, compared to an HbAT87Q level of 9.6% at three months post-transplant
- Note that this subject did not engraft until after month one, so their level of HbAT87Q production at months three and 4.5 are actually months two and 3.5, after engraftment
- At 4.5 months follow up, total anti-sickling hemoglobin (HbAT87Q + HbF) was 31.6%
- Subject 1204 has not had any hospitalizations for SCD-related complications post-transplant
- Increasing production of HbAT87Q; the first-ever SCD patient treated with gene therapy (subject 1204) had a HbAT87Q level of 24% at 4.5 months follow up, compared to an HbAT87Q level of 9.6% at three months post-transplant
- Safety: No subject has experienced a drug product-related adverse event, and integration site analyses demonstrate highly polyclonal reconstitution without clonal dominance
Based on historical clinical observations in patients with SCD, bluebird bio believes that individuals who achieve ≥ 30 percent of anti-sickling hemoglobin (HbAT87Q + HbF) have the potential to reduce or eliminate the serious and life-threatening events associated with SCD.
The abstract is now available online on the EHA conference website. Information contained in the abstract reflects data available as of February 2015. Details of bluebird bio’s presentation are as follows: Title: Outcomes of Gene Therapy for B-Thalassemia Major and Severe Sickle Cell Disease via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta Globin Vector
Abstract Code: S466
Session Name: Gene therapy, cellular immunotherapy and vaccination
Date: Saturday, June 13, 2015
Oral Presentation Time: 11:30 - 11:45 a.m. CET
Location: Reed Messe Vienna, Room Stolz 2
Abstract Code: S466
Session Name: Gene therapy, cellular immunotherapy and vaccination
Date: Saturday, June 13, 2015
Oral Presentation Time: 11:30 - 11:45 a.m. CET
Location: Reed Messe Vienna, Room Stolz 2
Monday, May 18, 2015
Stem Cell "Wild West" takes root amid lack of US regulation
Source: AP
http://hosted.ap.org/dynamic/stories/U/US_STEM_CELLS_CLINICS?SITE=AP&SECTION=HOME&TEMPLATE=DEFAULT&CTIME=2015-05-18-03-07-13
The liquid is dark red, a mixture of fat and blood, and Dr. Mark Berman pumps it out of the patient's backside. He treats it with a chemical, runs it through a processor - and injects it into the woman's aching knees and elbows.
http://hosted.ap.org/dynamic/stories/U/US_STEM_CELLS_CLINICS?SITE=AP&SECTION=HOME&TEMPLATE=DEFAULT&CTIME=2015-05-18-03-07-13
The liquid is dark red, a mixture of fat and blood, and Dr. Mark Berman pumps it out of the patient's backside. He treats it with a chemical, runs it through a processor - and injects it into the woman's aching knees and elbows.
The "soup," he says, is rich in shape-shifting stem cells - magic bullets that, according to some doctors, can be used to treat everything from Parkinson's disease to asthma to this patient's chronic osteoarthritis.
"I don't even know what's in the soup," says Berman. "Most of the time, if stem cells are in the soup, then the patient's got a good chance of getting better."
It's quackery, critics say. But it's also a mushrooming business - and almost wholly unregulated...
Berman, a Beverly Hills plastic surgeon, is co-founder of the largest chain, the Cell Surgical Network. Like most doctors in the field, he has no formal background in stem cell research. His company offers stem cell procedures for more than 30 diseases and conditions, including Lou Gehrig's disease, multiple sclerosis, lupus and erectile dysfunction.
There are clinics that market "anti-aging" treatments; others specialize in "stem-cell facelifts" and other cosmetic procedures. The cost is high, ranging from $5,000 to $20,000.
National Acamedy of Science to convene Gene Editing International Summit
From Reuters:
Last month, scientists in China reported carrying out the first experiment using CRISPR gene-editing to alter the DNA of human embryos. Although the embryos were not viable and could not have developed into babies, the announcement ignited an outcry from scientists warning that such a step, which could alter human genomes for generations, was just a matter of time.
It is a step reminiscent of one in 1975, when NAS convened the Asilomar Conference. That led to guidelines and federal regulations of recombinant DNA, the gene-splicing technology that underlay the founding of Genentech and other biotech companies and revolutionized the production of many pharmaceuticals.
The NAS committee will, similarly, recommend guidelines for gene-editing technologies.
NEW YORK (Reuters) - The leading U.S. scientific organization, responding to concerns expressed by scientists and ethicists, has launched an ambitious initiative to recommend guidelines for new genetic technology that has the potential to create "designer babies."
The technology, called CRISPR-Cas9, allows scientists to edit virtually any gene they target. The technique is akin to a biological word-processing program that finds and replaces genetic defects.
The technique has taken biology by storm, igniting fierce patent battles between start-up companies and universities that say it could prove as profitable and revolutionary as recombinant DNA technology, which was developed in the 1970s and 1980s and launched the biotechnology industry.
But CRISPR has also brought ethical concerns. Last month, scientists in China reported carrying out the first experiment using CRISPR gene-editing to alter the DNA of human embryos. Although the embryos were not viable and could not have developed into babies, the announcement ignited an outcry from scientists warning that such a step, which could alter human genomes for generations, was just a matter of time.
In response, the National Academy of Sciences (NAS) and its Institute of Medicine will convene an international summit this fall where researchers and other experts will "explore the scientific, ethical, and policy issues associated with human gene-editing research," the academies said in a statement.
In addition, NAS - an honorary body that was chartered by Congress in 1863 and performs studies for the federal government and others - will appoint a multidisciplinary, international committee to study the scientific basis and the ethical, legal, and social implications of human gene editing.It is a step reminiscent of one in 1975, when NAS convened the Asilomar Conference. That led to guidelines and federal regulations of recombinant DNA, the gene-splicing technology that underlay the founding of Genentech and other biotech companies and revolutionized the production of many pharmaceuticals.
The NAS committee will, similarly, recommend guidelines for gene-editing technologies.
"We provided leadership in the past on emerging, controversial new areas of genetic research, such as human embryonic stem cell research (and) human cloning," NAS President Ralph Cicerone and IOM President Victor Dzau said in a joint statement. "We are prepared to work with the scientific and medical communities to achieve a comprehensive understanding of human gene editing and its implications."
(Reporting by Sharon Begley; Editing by Steve Orlofsky)
Nature Biotech: In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency
Plasmid constructs provided by Sangamo BioSciences (SGMO).
Access to full abstract requires rights.
http://www.nature.com/nbt/journal/vaop/ncurrent/full/nbt.3241.html
Surfactant proteins A, B, C, and D are specialized proteins that make up about 5% of the pulmonary surfactant. Surfactant protein B (SP-B) and C (SP-C) are mainly involved in preventing alveolar collapse while surfactant protein A (SP-A) and D (SP-D) play a role in the lung’s immune defense. ABCA3 is a protein that transports surfactant within the alveolar Type II cell, the cell type in the lung that produces pulmonary surfactant. The thyroid transcription factor (TTF1) is a protein that activates surfactant associated genes, among others. Problems with any of these can cause lung damage.
Genome editing using a variety of different nucleases holds great potential to knock out or repair disease-causing genes. An ideal nuclease delivery vehicle is short-lived, does not integrate into the genome and can enter target cells efficiently. These requirements have not yet been achieved simultaneously by any nuclease…
Access to full abstract requires rights.
http://www.nature.com/nbt/journal/vaop/ncurrent/full/nbt.3241.html
Surfactant proteins A, B, C, and D are specialized proteins that make up about 5% of the pulmonary surfactant. Surfactant protein B (SP-B) and C (SP-C) are mainly involved in preventing alveolar collapse while surfactant protein A (SP-A) and D (SP-D) play a role in the lung’s immune defense. ABCA3 is a protein that transports surfactant within the alveolar Type II cell, the cell type in the lung that produces pulmonary surfactant. The thyroid transcription factor (TTF1) is a protein that activates surfactant associated genes, among others. Problems with any of these can cause lung damage.
Genome editing using a variety of different nucleases holds great potential to knock out or repair disease-causing genes. An ideal nuclease delivery vehicle is short-lived, does not integrate into the genome and can enter target cells efficiently. These requirements have not yet been achieved simultaneously by any nuclease…
Sunday, May 17, 2015
Moussatos of Wedbush maintains $30 price target for SGMO
As reported in analystratings.com Linda Moussatos,PHD of Wedbush Securities maintained an Outperform rating on Sangamo Biosciences (NASDAQ: SGMO), with a price target of $30. The company’s shares opened Friday at $11.51.
"Moussatos commented, “Sangamo recently announced that it would shelve its current ZFN therapeutic strategy for β-thalassemia in favor for a new combined ZFN therapeutic approach for β-thalassemia and sickle cell disease. We understand that preclinical data (see summary of ASGCT Abstract 53 below) support the development of ZFN mediated deletion of Bcl11a enhancer for these programs. While there is a delay caused by dropping the Phase 1 ready Bcl11a gene knockout strategy for β-thalassemia, consolidation of the strategy used for β-thalassemia and sickle cell disease programs could provide benefits–including cost savings, a more direct read through for clinical efficacy and safety between the two indications, and a faster collective time for approval for both programs.”"
About Liana Moussatos:
Liana Moussatos joined Wedbush Securities as a managing director and senior research analyst from Pacific Growth Equities, where she was a senior research analyst. Previously, she was director and portfolio manager of the UBS Global Biotech Funds for five years at UBS Global Asset Management. Moussatos also was with Bristol-Meyers Squibb, where she was a manager in University and Government Licensing, External Science and Technology. She also worked with Sloan-Kettering Cancer Institute in the Office of Industrial Affairs, and with the National Cancer Institute in the Office of Technology Development. Moussatos received a bachelor's degree in entomology and a master's degree in zoology and biochemistry from Clemson University. She also earned a Ph.D. in plant pathology from the University of California, Davis, and completed a postdoctoral research fellowship in cellular and molecular physiology at the Yale School of Medicine
"Moussatos commented, “Sangamo recently announced that it would shelve its current ZFN therapeutic strategy for β-thalassemia in favor for a new combined ZFN therapeutic approach for β-thalassemia and sickle cell disease. We understand that preclinical data (see summary of ASGCT Abstract 53 below) support the development of ZFN mediated deletion of Bcl11a enhancer for these programs. While there is a delay caused by dropping the Phase 1 ready Bcl11a gene knockout strategy for β-thalassemia, consolidation of the strategy used for β-thalassemia and sickle cell disease programs could provide benefits–including cost savings, a more direct read through for clinical efficacy and safety between the two indications, and a faster collective time for approval for both programs.”"
About Liana Moussatos:
Liana Moussatos joined Wedbush Securities as a managing director and senior research analyst from Pacific Growth Equities, where she was a senior research analyst. Previously, she was director and portfolio manager of the UBS Global Biotech Funds for five years at UBS Global Asset Management. Moussatos also was with Bristol-Meyers Squibb, where she was a manager in University and Government Licensing, External Science and Technology. She also worked with Sloan-Kettering Cancer Institute in the Office of Industrial Affairs, and with the National Cancer Institute in the Office of Technology Development. Moussatos received a bachelor's degree in entomology and a master's degree in zoology and biochemistry from Clemson University. She also earned a Ph.D. in plant pathology from the University of California, Davis, and completed a postdoctoral research fellowship in cellular and molecular physiology at the Yale School of Medicine
Wednesday, May 13, 2015
Sangamo changes path in Beta-Thanlassemia program- delays trial to 2016
RICHMOND, Calif., May 13, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced that it will consolidate development paths for the zinc finger nuclease (ZFN)-mediated genome editing programs targeting beta-thalassemia and sickle cell disease (SCD). This decision was based on preclinical data that support the development of the "BCL11A Enhancer" target for these clinical programs, indicating that it has the potential to provide the most efficient path to clinical proof of concept and subsequent development. While the beta-thalassemia program was initiated with a BCL11a knockout strategy, the SCD program already employs the BCL11A Enhancer approach. The decision to consolidate the strategy for these two programs was made by the joint steering committee (JSC) governing the programs, including Sangamo's collaborator Biogen.
Biogen's experience in the development of novel therapeutics has been critical as we work to advance these ZFP Therapeutics into the clinic," saidEdward Lanphier , Sangamo's president and chief executive officer. "While our joint decision will result in a delay in the initiation of the beta-thalassemia Phase 1 clinical trial, we believe that the efficiency of the consolidated development path and potential benefit to patients clearly support this decision."
Mr. Lanphier added, "We are committed to rapidly moving this exciting new therapeutic approach powered by our ZFN genome editing technology into human clinical trials. The alignment of the beta-thalassemia and SCD programs to use the same specific ZFN reagent will enable more rapid and efficient co-development and provide both beta-thalassemia and sickle cell disease patients with a potentially safe and efficacious single-administration treatment with a life-long therapeutic effect."
"The quality of the clinical candidate and a focus on patient benefit drives our development decisions," saidOlivier Danos , Ph.D., Biogen's senior vice president of gene therapy. "Sangamo's ability to rapidly move from identification of a new DNA target to a highly specific genome editing therapeutic lead candidate has enabled us to quickly deploy the latest scientific knowledge against both of these important genetic diseases."
Sangamo intends to file a new Investigational New Drug (IND) application for the "BCL11A Enhancer" approach for beta-thalassemia and anticipates initiating a Phase 1 clinical trial in 2016.
About the BCL11A Enhancer and BCL11A Knockout approachesBoth beta-thalassemia and SCD manifest several months after birth, when patients' cells switch from producing functional fetal globin to a mutant form of adult beta-globin, which causes their condition. Naturally occurring increased levels of fetal hemoglobin have been shown to reduce the severity of both SCD and beta-thalassemia disorders in adulthood. The collaborative development program uses ZFN-mediated genome editing of a patient's own hematopoietic stem and progenitor cells (HSPCs) to increase production of fetal globin in cells that will ultimately become red blood cells (RBCs). This novel approach uses the targeted specificity of ZFNs which need to be expressed in the cell only transiently to have a permanent effect.
Building upon recent data on the regulation of fetal hemoglobin, Sangamo and Biogen have developed two related but distinct ZFN-mediated genome editing approaches to disrupt critical aspects of the regulatory pathway that, in early infancy, leads to the switch in production from fetal to adult globin.
Initially, Biogen and Sangamo developed a strategy for beta-thalassemia that specifically knocked out the gene encoding the BCL11A transcription factor, a critical regulator of the switch from fetal to adult globin production. A second approach was initiated for the SCD program, which involved the disruption of the more recently described erythroid-specific "Enhancer" of BCL11A expression, a regulatory DNA sequence in the genome that is essential for expression of BCL11A but that is functional exclusively in cells destined to become RBCs. Both ZFN-mediated approaches were found to be equally specific and efficient leading to similar increases in fetal globin production. However, the Enhancer approach was found to have certain advantages, including its specificity for RBC producing cells, making it a preferable therapeutic strategy for hemoglobinopathies. Thus, the determination was made that the beta-thalassemia program should follow BCL11A Enhancer approach, like the SCD program.
"Sangamo's design and selection process enables rapid optimization of highly specific lead ZFN therapeutics," commentedPhilip Gregory , D.Phil ., Sangamo's senior vice president of research and chief scientific officer. "While the discovery of the BCL11A Enhancer as a potential target was made relatively recently, the team generated critical data that supported the Enhancer approach in time for this strategy to be used for both the beta-thalassemia and the sickle cell disease programs."
Preclinical data supporting the Enhancer program will be presented at4:15 pm ET , today, Wednesday, May 13, 2015 at the 18th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Abstract #53 "From GWAS to the Clinic: Genome-Editing the Human Bcl11a Erythroid Enhancer for Fetal Globin Elevation in the Hemoglobinopathies
Biogen's experience in the development of novel therapeutics has been critical as we work to advance these ZFP Therapeutics into the clinic," said
"The quality of the clinical candidate and a focus on patient benefit drives our development decisions," said
Sangamo intends to file a new Investigational New Drug (IND) application for the "BCL11A Enhancer" approach for beta-thalassemia and anticipates initiating a Phase 1 clinical trial in 2016.
About the BCL11A Enhancer and BCL11A Knockout approachesBoth beta-thalassemia and SCD manifest several months after birth, when patients' cells switch from producing functional fetal globin to a mutant form of adult beta-globin, which causes their condition. Naturally occurring increased levels of fetal hemoglobin have been shown to reduce the severity of both SCD and beta-thalassemia disorders in adulthood. The collaborative development program uses ZFN-mediated genome editing of a patient's own hematopoietic stem and progenitor cells (HSPCs) to increase production of fetal globin in cells that will ultimately become red blood cells (RBCs). This novel approach uses the targeted specificity of ZFNs which need to be expressed in the cell only transiently to have a permanent effect.
Building upon recent data on the regulation of fetal hemoglobin, Sangamo and Biogen have developed two related but distinct ZFN-mediated genome editing approaches to disrupt critical aspects of the regulatory pathway that, in early infancy, leads to the switch in production from fetal to adult globin.
Initially, Biogen and Sangamo developed a strategy for beta-thalassemia that specifically knocked out the gene encoding the BCL11A transcription factor, a critical regulator of the switch from fetal to adult globin production. A second approach was initiated for the SCD program, which involved the disruption of the more recently described erythroid-specific "Enhancer" of BCL11A expression, a regulatory DNA sequence in the genome that is essential for expression of BCL11A but that is functional exclusively in cells destined to become RBCs. Both ZFN-mediated approaches were found to be equally specific and efficient leading to similar increases in fetal globin production. However, the Enhancer approach was found to have certain advantages, including its specificity for RBC producing cells, making it a preferable therapeutic strategy for hemoglobinopathies. Thus, the determination was made that the beta-thalassemia program should follow BCL11A Enhancer approach, like the SCD program.
"Sangamo's design and selection process enables rapid optimization of highly specific lead ZFN therapeutics," commented
Preclinical data supporting the Enhancer program will be presented at
Seeking Alpha article has high praise for Sangamo BioSciences
Fire Up The Immune System To Fight Cancers And Boost Portfolios: John McCamant
Excerpts:JM: I'd like to talk about Sangamo BioSciences Inc. (NASDAQ:SGMO). Why is bluebird bio Inc. (NASDAQ:BLUE) worth five times more than Sangamo? Why is bluebird a $5.4 billion stock while Sangamo is a $875 million [$875M] stock? It doesn't make any sense when I compare the two.
What are the two companies' lead programs? Beta thalassemia and sickle cell disease. It looks like both companies have effective approaches to these diseases, which have very large market opportunities, yet Sangamo has probably one of the best corporate partners in the business. Biogen Inc. [BIIB:NASDAQ] has fully validated the Sangamo program and chose it, over bluebird's, in both sickle cell and beta thalassemia.
TLSR: Sangamo's stock is basically flat versus one year ago. What moves the stock from here? Could it be the Phase 2 data in HIV/AIDS?
JM: It could. Sangamo has an extremely broad pipeline, but the bigger picture is this: Who's going to be advancing these indications in gene therapy? We think the safety profile of Sangamo's zinc finger technology is more proven.
A lot of companies have been talking up a gene editing technology called CRISPR [clustered regularly interspaced short palindromic repeats] for the last two years, but now there's a voluntary moratorium on CRISPR use in human studies. We're really making progress in changing genes inside of human cells. This is something that scares regulators-and it should-but that's why we like companies that have been around longer, created bigger safety databases, and that are coming out with multiple shots on goal. Gene therapy is cutting-edge science fiction, and it's becoming a reality.
TLSR: It is indeed. But I think the name Jesse Gelsinger will linger in the minds of regulators for quite some time to come.
JM: Yes, it will. It's important not to forget that young man's name; your readers will recall that a gene therapy experiment went wildly wrong and killed an 18-year-old patient who wasn't that sick to begin with. But I really think that with Sangamo, when investors add up the amount of years it's been developing its technology, the way it has built out its platform, how it's been validated, and its safety profile, they will understand that its valuation is quite low today. Not only does Sangamo have platform validation from Biogen, but it also has three preclinical programs with Shire Plc [SHPGY:NASDAQ; SHP:LSE] in hemophilia A, hemophilia B and Huntington's disease.
The other thing we know is that bluebird is not going to sail forward from being a $5.4B company to being a $20B company without bumps in the road. If nothing else, the risk/reward is unfavorable with bluebird versus Sangamo, which has a market valuation below $900M.
Big Pharma closing in on Gene Therapy
The blog, Clinical Leader offers a good review of the state of the gene therapy field.
Sangamo BioSciences (SGMO) CEO is quoted with regards to regulatory requirements, but no mention of the clinical programs Sangamo is fielding. This is disappointing.With regards to Biogen the article refers to its Hemophilia work.
http://www.clinicalleader.com/doc/keeping-tabs-on-gene-therapy-big-pharma-is-closing-in-0001
Juno Therapeutics: In a Phase 1 clinical trial, 91 percent of pediatric patients taking Juno’s JCAR017 for relapsed/refractory CD19-positive acute lymphoblastic leukemia (ALL) achieved complete remission. In even more recent news, the company launched a research collaboration with Fate Therapeutics to bolster the therapeutic potential of Juno’s T-cell immunotherapies, as well as acquired Stage Cell Therapeutics.
Novartis: One researcher from Novartis, in partnership with an expert from GenVec, is exploring a gene therapy treatment for patients with hearing loss, Bloomberg reported in February. These two researchers are attempting to regrow the sound-sensing hair cells within the ear that have been destroyed by excessive loud noise. A trial enrolling 45 U.S. patients received NIH approval and is currently underway, with results expected by 2017. In addition, the company made headlines at the beginning of 2015 for its deals with Intellia Therapeutics and Caribou Biosciences which will provide Novartis with CRISPR (clustered regularly interspaced short palindromic repeats) technology to edit the genes of targeted cells and boost drug discovery efforts. Novartis is also currently in a collaboration with the University of Pennsylvania, which led to the development of CTL019 for pediatric and adult patients with r/r ALL. This treatment earned a BTD from the FDA last summer for this indication, and, in a clinical trial, led to complete remission of the disease in 36 of the 39 patients receiving the therapy.
Sanofi: In February, Sanofi’s subsidiary Genzyme launched an $845 million partnership with Voyager to collaborate on gene therapy programs in Parkinson’s disease (VY-AADC01), Friedreich’s ataxia (VY-FXN01), and Huntington’s disease (VY-HTT01). In particular, the two are working to accelerate the Parkinson’s candidate through Phase 1, as it promises to be able to treat those who don’t respond to standard-of-care levodopa and carbidopa. Voyager, with Sanofi’s help, is also planning preclinical programs for the other candidates.
Spark Therapeutics: In January, the gene therapy company expanded its pipeline of inherited retinal dystrophy treatments by initiating a Phase 1/2 clinical trial of its SPK-CHM candidate for choroideremia, an inherited retinal dystrophy (IRD) affecting male children. The company’s lead candidate, SPK-RPE65, is currently in Phase 3 trials for IRDs.
Bristol-Myers Squibb (BMS): In April, BMS signed an agreement with UniQure for the rights to UniQure’s lead heart failure candidate, S100A1. According to the agreement, BMS will also gain the rights to three other UniQure candidates in the upcoming months.
Biogen Idec: Following in GSK’s footsteps, Biogen Idec has joined forces with Fondazione Telethon and Ospedale San Raffaele against hemophilia A and B. The partners will explore liver-targeting lentiviral gene transfer technology. This step followed the company’s decision to hire Olivier Danos as VP of its burgeoning gene therapy operation.
Pfizer: Adding to the pool of companies exploring hemophilia gene therapy treatments, Pfizer jumped aboard with Spark Therapeutics in December 2014 to accelerate development of the AAV vector SPK-FIX and launch Phase 1/2 clinical trials this year.
bluebird bio: In October 2014, the company launched a trial for its primary gene therapy candidate, LentiGlobin BB305, indicated for sickle cell disease. The first patient dosed was enrolled in the Phase 1/2 HGB-205 study being conducted in Paris, while a separate Phase 1 trial, HGB-206, enrolling upwards of eight sickle cell disease patients is currently underway in the U.S. LentiGlobin also recently demonstrated its ability to boost hemoglobin production in patients with beta-thalassemia major in the HGB-204 study — an indication for which the treatment recently was awarded an FDA BTD. The company has also been in a partnership with Celgene since 2013 to explore new and develop existing chimeric antigen receptor T-cell (CART) therapies in cancer.
UniQure: In a Phase 1 dose-escalation clinical trial, the company’s AAV5-PBGD candidate for Acute Intermittent Porphyria, AMT-021, was found to be a safe method of inserting the porphobilinogen deaminase gene (PBGD) into patients liver cells.
Bayer: Last summer, Bayer teamed up with Dimension Therapeutics to come up with a gene therapy treatment for hemophilia A.
Sangamo BioSciences (SGMO) CEO is quoted with regards to regulatory requirements, but no mention of the clinical programs Sangamo is fielding. This is disappointing.With regards to Biogen the article refers to its Hemophilia work.
http://www.clinicalleader.com/doc/keeping-tabs-on-gene-therapy-big-pharma-is-closing-in-0001
Tuesday, May 12, 2015
Gero Hutter: “I believe it’s possible to develop a mass-market single-shot treatment for HIV,”
In this article he offers hope that we are finally close to a cure.
Hutter offers,“If we can overcome a few problems, our approach is closer to a complete cure than anything in the last 30 years.”
See the article here:
http://www.theguardian.com/science/blog/2015/may/12/hiv-immunity-rare-gene-differences-offer-hope-for-treatment
Excerpts:
Hütter appeared to have the perfect solution. But after the accolades and the acclaim died down, reality has slowly set in. Since Brown, six more HIV patients have been treated with similar transplants around the world. None have survived longer than twelve months.
Instead of trapping and slowly eliminating the virus, some believe that disabling the CCR5 receptor simply provoked it to mutate and invade cells via alternative receptors. But why did this happen in those patients while Brown was cured? “If we can understand this, we may be able to translate his cure into something feasible for all patients,” Hütter says.
It was 16 years ago that Professor Michael Farzan discovered the beneficial qualities of the CCR5 gene mutation at Harvard University. Now he believes he’s close to developing an HIV vaccine based on this form of natural immunity.
While the CCR5 mutation has received the lion’s share of the spotlight, it’s also not the only form of natural immunity to HIV. At the University of Minnesota, Professor Reuben Harris is studying couples with mixed HIV status. “These instances are extremely interesting because you have an infected person and their partner who remains HIV-negative despite many opportunities for the virus to be transmitted,” he says. “By taking blood samples, we can play around with the virus and work out what changes would need to be made in order for it to infect cells from the partner. And from that we can work out what’s protecting them.”
There’s a particular family of genes called APOBEC3 which produces antiretroviral enzymes, one of the body’s main defences against viral infection. Harris has found that people with specific variations of the gene APOBEC3H produce stronger and more stable enzymes which can inhibit the replication of HIV. Having the right variant of this gene may make the likelihood of transmission much lower.
Hutter offers,“If we can overcome a few problems, our approach is closer to a complete cure than anything in the last 30 years.”
See the article here:
http://www.theguardian.com/science/blog/2015/may/12/hiv-immunity-rare-gene-differences-offer-hope-for-treatment
Excerpts:
Hütter appeared to have the perfect solution. But after the accolades and the acclaim died down, reality has slowly set in. Since Brown, six more HIV patients have been treated with similar transplants around the world. None have survived longer than twelve months.
Instead of trapping and slowly eliminating the virus, some believe that disabling the CCR5 receptor simply provoked it to mutate and invade cells via alternative receptors. But why did this happen in those patients while Brown was cured? “If we can understand this, we may be able to translate his cure into something feasible for all patients,” Hütter says.
It was 16 years ago that Professor Michael Farzan discovered the beneficial qualities of the CCR5 gene mutation at Harvard University. Now he believes he’s close to developing an HIV vaccine based on this form of natural immunity.
While the CCR5 mutation has received the lion’s share of the spotlight, it’s also not the only form of natural immunity to HIV. At the University of Minnesota, Professor Reuben Harris is studying couples with mixed HIV status. “These instances are extremely interesting because you have an infected person and their partner who remains HIV-negative despite many opportunities for the virus to be transmitted,” he says. “By taking blood samples, we can play around with the virus and work out what changes would need to be made in order for it to infect cells from the partner. And from that we can work out what’s protecting them.”
There’s a particular family of genes called APOBEC3 which produces antiretroviral enzymes, one of the body’s main defences against viral infection. Harris has found that people with specific variations of the gene APOBEC3H produce stronger and more stable enzymes which can inhibit the replication of HIV. Having the right variant of this gene may make the likelihood of transmission much lower.
Sangamo partner Sigma-Aldrich expands gene therapy manufacturing facility
Sangamo BioSciences (SGMO) has a strategic partnership with Sigma-Aldrich to provide ZFP products and the CHOZN cell line production platform.
Sigma-Aldrich Corporation’s (NASDAQ: SIAL) custom manufacturing business unit, SAFC® Commercial (www.sigma-aldrich.com/safc), announced today the strategic expansion of its existing facility in Carlsbad, Calif. The investment will further enable SAFC to offer clinical and commercial bulk drug production, as well as fill/finish of viral products for its gene therapy, viral vaccine and immunotherapy customers. “Gene therapy is an emerging technology, and our SAFC and BioReliance® sites in Carlsbad, Rockville, and Glasgow have supported this growing industry for years,” said Gilles Cottier, President of SAFC. “This investment is pivotal to our customers and reflects SAFC’s continued dedication to providing the infrastructure customers need to bring their drugs to market.” The expanded Carlsbad facility complements a global footprint of GMP services in the virus and gene therapy segment, including cell banking in Rockville, Md., and manufacturing in the Glasgow location in Scotland, U.K. The investment was driven by continued interest in targeted gene therapies for indications such as hemophilia and cancer immunotherapies (CAR-T cells). Utilizing the biosafety testing expertise of BioReliance, the Carlsbad facility provides a full range of GMP manufacturing and testing services to its customers. To learn more about the capabilities at the Carlsbad location, please visit: www.sigmaaldrich.com/safc/carlsbad. - See more at: http://www.sigmaaldrich.com/safc/news-and-resource-center/press-releases/safc-expands-carlsbad-facility.html#sthash.isQp8vMw.dpuf
Sigma-Aldrich Corporation’s (NASDAQ: SIAL) custom manufacturing business unit, SAFC® Commercial (www.sigma-aldrich.com/safc), announced today the strategic expansion of its existing facility in Carlsbad, Calif. The investment will further enable SAFC to offer clinical and commercial bulk drug production, as well as fill/finish of viral products for its gene therapy, viral vaccine and immunotherapy customers. “Gene therapy is an emerging technology, and our SAFC and BioReliance® sites in Carlsbad, Rockville, and Glasgow have supported this growing industry for years,” said Gilles Cottier, President of SAFC. “This investment is pivotal to our customers and reflects SAFC’s continued dedication to providing the infrastructure customers need to bring their drugs to market.” The expanded Carlsbad facility complements a global footprint of GMP services in the virus and gene therapy segment, including cell banking in Rockville, Md., and manufacturing in the Glasgow location in Scotland, U.K. The investment was driven by continued interest in targeted gene therapies for indications such as hemophilia and cancer immunotherapies (CAR-T cells). Utilizing the biosafety testing expertise of BioReliance, the Carlsbad facility provides a full range of GMP manufacturing and testing services to its customers. To learn more about the capabilities at the Carlsbad location, please visit: www.sigmaaldrich.com/safc/carlsbad. - See more at: http://www.sigmaaldrich.com/safc/news-and-resource-center/press-releases/safc-expands-carlsbad-facility.html#sthash.isQp8vMw.dpuf
Monday, May 11, 2015
Former Sangamo research associate now Chief Scientific Officer of Gene Editing Startup
According to Fierce Biotech a new biotech has come into view. Precision BioSciences, a Duke spinout focused on competing in the fractious and rapidly growing field, has grabbed a $25.6 million A round with the help of some A-list investors.
http://www.fiercebiotech.com/story/amgen-fidelity-help-bankroll-another-upstart-gene-editing-player/2015-05-11
Combines CRISPR's and Talens, calls it ARCUS.
Precision is helmed by Duke graduate and CEO Kane and scientific officer Derek Jantz, who worked at Duke and once served a stint as a research associate at Sangamo, according to a short bio from Bloomberg. Jantz and his team won the $15,000 runner-up prize in Duke's 2006 startup challenge contest, boasting of the "only method to target and alter single DNA sequences in a genome."
http://www.fiercebiotech.com/story/amgen-fidelity-help-bankroll-another-upstart-gene-editing-player/2015-05-11
Combines CRISPR's and Talens, calls it ARCUS.
Precision is helmed by Duke graduate and CEO Kane and scientific officer Derek Jantz, who worked at Duke and once served a stint as a research associate at Sangamo, according to a short bio from Bloomberg. Jantz and his team won the $15,000 runner-up prize in Duke's 2006 startup challenge contest, boasting of the "only method to target and alter single DNA sequences in a genome."
Jay Levi M.D., Six questions about HIV/AIDS that deserve more attention
Dr. Jay Levi, one of the first researchers to isolate the AIDS virus and Director of the Laboratory for Tumor and AIDS Virus Research at the University of California, San Francisco calls on researchers not to lose sight of strategies that could lead to a sustainable, long-term solution to HIV infection.
Dr. Levi also has written that he is encouraged by the results of the Sangamo BioSciences (SGMO) HIV clinical trials.
1. Is HIV infection a universally fatal diagnosis?
A small percentage of people with HIV infection have been observed to possess immune systems that keep the virus at bay for at least 10 years, and some for more than 35 years. Although an HIV diagnosis was once considered a "death sentence," this is evidence that such long-time survivors or non-progressors can live a normal, asymptomatic life without intervention. "Importantly, we can learn a great deal about prevention of disease and infection by studying these exceptional people who have survived without AIDS or have warded off infection," Levy writes.
2. Is the body's innate immune response as important as the adaptive immune response?
Researchers have observed that some people who have been infected for many years do not develop disease. Others have been exposed on many occasions to HIV but do not become infected. In these cases, the innate immune system, the first line of defense against viruses, appears to play an important role. If the innate immune response fails, then adaptive immune activity—reflected by T and B cells—comes into action. Levy calls for increased attention to the innate immune system and its variety of immune cells and secreted factors. By focusing on this early activity against HIV, researchers have a better chance of discovering ways to prevent infection and disease.
3. How do CD8+ T cells combat HIV?
The immune system is a complex collection of cells with multiple mechanisms for taking down a pathogen. Even with 30+ years of HIV research, how immune cells behave when the virus enters the body is not fully understood. One immune cell of long-time interest to HIV/AIDS scientists is the CD8+ T lymphocyte, which is primarily thought to control HIV infection by killing infected cells. Levy discusses how this cell can employ an alternative mechanism for controlling HIV infection: it can secrete factors that suppress the virus without killing the cell, and then the infected cell can continue to function but without virus production and cell death. Because this immune response handles all HIV types, it would be important in approaches aimed at enhancing immune antiviral responses and in the development of a vaccine. Importantly, both activities of the CD8+ T cell need to be appreciated.
4. When should antiretroviral therapy be given?
There is no denying the success antiretroviral medications have had in lowering viral loads, reducing transmission, and helping people live a relatively normal life with HIV. Doctors often prescribe these drugs upon diagnosis, but Levy questions whether the long-term side effects, which include kidney and liver disorders, are worth it if a patient is asymptomatic. "Prescribing ART for a lifetime is like giving chemotherapy forever," he writes. "Future studies may very well indicate that toxicities will eventually result in many treated individuals."
There also exist concerns regarding the recent trend of "treatment as prevention," in which health-care professionals give antiretroviral drugs to HIV-negative people to substantially reduce their odds of infection. This treatment strategy is new, and it is unclear whether a lack of adherence because of drug side effects, including nausea or fatigue, will affect the prevention of transmission. It also could lead to the development of drug-resistant viral strains. While recognizing the important success of ART, Levy calls on his colleagues to give more attention to drug toxicities and the potential emergence of drug-resistant viruses. "It seems misguided to just look at the short-term benefit," he says.
5. What strategies should be considered for an HIV vaccine?
Levy argues that one of the greatest weaknesses of existing HIV-vaccine clinical trials—most reporting limited, if any, success—is that they are mainly looking to find a better antibody to neutralize the virus. On the basis of preclinical work, he believes that there are missed opportunities to invest in basic science and study other vaccine strategies, such as enhancing the antiviral responses of immune cells, particularly those of the innate immune system. He also advocates further studies of individuals who have been highly exposed to HIV but are not infected and the evaluation of killed-virus vaccines.
6. What approaches should be encouraged for an HIV cure?
A cure of HIV infection, which is exemplified in the "Berlin patient," has stimulated approaches at achieving this goal for all infected people. This person received a stem cell transplant from a donor who was genetically resistant to HIV. Levy argues that some of the directions chosen to affect a cure "are not based on well-established experimental facts" and eliminating a virus that has integrated itself into the genes of a cell requires a better understanding of the challenge.
Levy appreciates the pilot studies involving genetic editing that could mimic the treatment used for the "Berlin patient." And, he believes that the most immediate chance of success is one directed at enhancing the anti-HIV response of the immune system. "That strategy," he says, "could bring persistent control of HIV as seen in healthy infected long-term survivors. In this case, viral replication and its potential detrimental effects would be avoided—essentially the infected individual would be 'functionally cured.'"
In his review of these issues in HIV/AIDS, Levy says, "Researchers need to examine the basis of their observations and consider whether what they are seeing is really the answer or is part of what's really there. We have to ask whether we are disregarding or missing what might be a much better answer because we're grabbing at something that looks good today."
More information: Trends in Molecular Medicine, Levy, J.: "Dispelling myths and focusing on notable concepts in HIV pathogenesis" http://dx.doi.org/10.1016/j.molmed.2015.03.004
Dr. Levi also has written that he is encouraged by the results of the Sangamo BioSciences (SGMO) HIV clinical trials.
1. Is HIV infection a universally fatal diagnosis?
A small percentage of people with HIV infection have been observed to possess immune systems that keep the virus at bay for at least 10 years, and some for more than 35 years. Although an HIV diagnosis was once considered a "death sentence," this is evidence that such long-time survivors or non-progressors can live a normal, asymptomatic life without intervention. "Importantly, we can learn a great deal about prevention of disease and infection by studying these exceptional people who have survived without AIDS or have warded off infection," Levy writes.
2. Is the body's innate immune response as important as the adaptive immune response?
Researchers have observed that some people who have been infected for many years do not develop disease. Others have been exposed on many occasions to HIV but do not become infected. In these cases, the innate immune system, the first line of defense against viruses, appears to play an important role. If the innate immune response fails, then adaptive immune activity—reflected by T and B cells—comes into action. Levy calls for increased attention to the innate immune system and its variety of immune cells and secreted factors. By focusing on this early activity against HIV, researchers have a better chance of discovering ways to prevent infection and disease.
3. How do CD8+ T cells combat HIV?
The immune system is a complex collection of cells with multiple mechanisms for taking down a pathogen. Even with 30+ years of HIV research, how immune cells behave when the virus enters the body is not fully understood. One immune cell of long-time interest to HIV/AIDS scientists is the CD8+ T lymphocyte, which is primarily thought to control HIV infection by killing infected cells. Levy discusses how this cell can employ an alternative mechanism for controlling HIV infection: it can secrete factors that suppress the virus without killing the cell, and then the infected cell can continue to function but without virus production and cell death. Because this immune response handles all HIV types, it would be important in approaches aimed at enhancing immune antiviral responses and in the development of a vaccine. Importantly, both activities of the CD8+ T cell need to be appreciated.
4. When should antiretroviral therapy be given?
There is no denying the success antiretroviral medications have had in lowering viral loads, reducing transmission, and helping people live a relatively normal life with HIV. Doctors often prescribe these drugs upon diagnosis, but Levy questions whether the long-term side effects, which include kidney and liver disorders, are worth it if a patient is asymptomatic. "Prescribing ART for a lifetime is like giving chemotherapy forever," he writes. "Future studies may very well indicate that toxicities will eventually result in many treated individuals."
There also exist concerns regarding the recent trend of "treatment as prevention," in which health-care professionals give antiretroviral drugs to HIV-negative people to substantially reduce their odds of infection. This treatment strategy is new, and it is unclear whether a lack of adherence because of drug side effects, including nausea or fatigue, will affect the prevention of transmission. It also could lead to the development of drug-resistant viral strains. While recognizing the important success of ART, Levy calls on his colleagues to give more attention to drug toxicities and the potential emergence of drug-resistant viruses. "It seems misguided to just look at the short-term benefit," he says.
5. What strategies should be considered for an HIV vaccine?
Levy argues that one of the greatest weaknesses of existing HIV-vaccine clinical trials—most reporting limited, if any, success—is that they are mainly looking to find a better antibody to neutralize the virus. On the basis of preclinical work, he believes that there are missed opportunities to invest in basic science and study other vaccine strategies, such as enhancing the antiviral responses of immune cells, particularly those of the innate immune system. He also advocates further studies of individuals who have been highly exposed to HIV but are not infected and the evaluation of killed-virus vaccines.
6. What approaches should be encouraged for an HIV cure?
A cure of HIV infection, which is exemplified in the "Berlin patient," has stimulated approaches at achieving this goal for all infected people. This person received a stem cell transplant from a donor who was genetically resistant to HIV. Levy argues that some of the directions chosen to affect a cure "are not based on well-established experimental facts" and eliminating a virus that has integrated itself into the genes of a cell requires a better understanding of the challenge.
Levy appreciates the pilot studies involving genetic editing that could mimic the treatment used for the "Berlin patient." And, he believes that the most immediate chance of success is one directed at enhancing the anti-HIV response of the immune system. "That strategy," he says, "could bring persistent control of HIV as seen in healthy infected long-term survivors. In this case, viral replication and its potential detrimental effects would be avoided—essentially the infected individual would be 'functionally cured.'"
In his review of these issues in HIV/AIDS, Levy says, "Researchers need to examine the basis of their observations and consider whether what they are seeing is really the answer or is part of what's really there. We have to ask whether we are disregarding or missing what might be a much better answer because we're grabbing at something that looks good today."
More information: Trends in Molecular Medicine, Levy, J.: "Dispelling myths and focusing on notable concepts in HIV pathogenesis" http://dx.doi.org/10.1016/j.molmed.2015.03.004
Friday, May 8, 2015
CBS 13 Sacramento news story on elite controllers
3 minute video here:
http://sacramento.cbslocal.com/video?autoStart=true&topVideoCatNo=default&clipId=11473606
A Sacramento woman is part of a rare population of people who are infected with HIV, but somehow keep the virus from damaging their immune system.
Scientists hope these HIV controllers hold the key to finding a cure.
In the world, 35 million people are living with HIV or AIDS. Most take a myriad of drugs to suppress the disease from wreaking havoc on their immune system.
Loreen Willenberg is one of those 35 million, but in the 23 years since she was diagnosed, she takes no drugs, has no symptoms, and has never been sick.
In a clinical sense, I’m not progressing towards AIDS,” she said. “I’m not progressing towards the disease stage.”
Dr. Richard Pollard says she’s part of a small number of HIV patients—less than 1 percent—who have the virus, but can control its invasion of their bodies.
“Their body has such an effective way of reacting to the virus that it’s hard to even detect that they’re virus positive,” he said.
Researchers think it works like this:
HIV attacks CD4 T-cells, which are basically white blood cells that help your immune system. In most patients, HIV kills those T-cells and replicates itself, while overpowering another type of T-cell called CD8 cells. These are meant to fight off viral infection.
But in people like Loreen, those CD8 cells are particularly strong. They act like soldiers and regulate the infected CD4 cells. This keeps HIV from replicating and diminishing her immune system.
I haven’t had a decline of CD4 cell count at all,and that’s pretty magnificent, and I’m very humbled by that,” she said.
Over the past decade, Loreen has participated in 13 studies. Researchers are trying to figure out why elite controllers are able to do what they do.
“Finding the answer to that might lead to other kinds of research to try to develop techniques to make someone mimic these elite controllers,” he said.
Pollard says studying elite controllers is just one avenue researchers are taking to stop the HIV-AIDS epidemic. Both he and Loreen hope they’ll see the day there’s a vaccine or even a cure for HIV.
“If that happens before I go, then i will have, then I know that I have lived a good life,” she said.
There are only 500 known elite controllers in the world.
At the age of 61, Loreen is going to college to major in bioethics. She’s stayed on top of the scientific studies she’s been a part of and served as an HIV advocate for years.
http://sacramento.cbslocal.com/video?autoStart=true&topVideoCatNo=default&clipId=11473606
A Sacramento woman is part of a rare population of people who are infected with HIV, but somehow keep the virus from damaging their immune system.
Scientists hope these HIV controllers hold the key to finding a cure.
In the world, 35 million people are living with HIV or AIDS. Most take a myriad of drugs to suppress the disease from wreaking havoc on their immune system.
Loreen Willenberg is one of those 35 million, but in the 23 years since she was diagnosed, she takes no drugs, has no symptoms, and has never been sick.
In a clinical sense, I’m not progressing towards AIDS,” she said. “I’m not progressing towards the disease stage.”
Dr. Richard Pollard says she’s part of a small number of HIV patients—less than 1 percent—who have the virus, but can control its invasion of their bodies.
“Their body has such an effective way of reacting to the virus that it’s hard to even detect that they’re virus positive,” he said.
Researchers think it works like this:
HIV attacks CD4 T-cells, which are basically white blood cells that help your immune system. In most patients, HIV kills those T-cells and replicates itself, while overpowering another type of T-cell called CD8 cells. These are meant to fight off viral infection.
But in people like Loreen, those CD8 cells are particularly strong. They act like soldiers and regulate the infected CD4 cells. This keeps HIV from replicating and diminishing her immune system.
I haven’t had a decline of CD4 cell count at all,and that’s pretty magnificent, and I’m very humbled by that,” she said.
Over the past decade, Loreen has participated in 13 studies. Researchers are trying to figure out why elite controllers are able to do what they do.
“Finding the answer to that might lead to other kinds of research to try to develop techniques to make someone mimic these elite controllers,” he said.
Pollard says studying elite controllers is just one avenue researchers are taking to stop the HIV-AIDS epidemic. Both he and Loreen hope they’ll see the day there’s a vaccine or even a cure for HIV.
“If that happens before I go, then i will have, then I know that I have lived a good life,” she said.
There are only 500 known elite controllers in the world.
At the age of 61, Loreen is going to college to major in bioethics. She’s stayed on top of the scientific studies she’s been a part of and served as an HIV advocate for years.
Paula Cannon speaking at Triwizard Tournament
Sorry, correction. She is not speaking at the Triwizard tournament of Harry Potter fame, she will be speaking about Zinc Finger Gene Therapy at the Tri-institutional Stem Cell Retreat.
USC, UCSF and UCLA, 3 day seminar begins on May 17th, 2015. Her presentation is titled
"Using targeted nucleases to modify HSC as an anti-HIV gene therapy"
https://stemcell.usc.edu/files/2015/05/CURRENT-Tri-retreat-program-May-6.pdf
USC, UCSF and UCLA, 3 day seminar begins on May 17th, 2015. Her presentation is titled
"Using targeted nucleases to modify HSC as an anti-HIV gene therapy"
https://stemcell.usc.edu/files/2015/05/CURRENT-Tri-retreat-program-May-6.pdf
Thursday, May 7, 2015
Adaptimmune trading (ADAP)
On May 5th:
Adaptimmune Therapeutics plc (Nasdaq: ADAP) today announced the pricing of its initial public offering of 11,250,000 American Depositary Shares (ADSs) at a price to the public of $17.00 per ADS. In addition, the underwriters have been granted a 30-day option to purchase up to an additional 1,687,500 ADSs. The ADSs are expected to begin trading on the NASDAQ Global Select Market under the symbol “ADAP” on May 6, 2015. The offering is expected to close on May 11, 2015, subject to customary closing conditions. Adaptimmune estimates net proceeds from the offering will be approximately $175.7 million.
On May 6th, from IBD:
ritish biotech Adaptimmune Therapeutics (NASDAQ:ADAP) fattened its IPO and priced at the high end of its range late Tuesday, but that seemed to be more than enough for investors as the stock dropped 5.9% to close at 16 in its debut Wednesday.
Adaptimmune priced 11.3 million shares, up from the previously announced 9.4 million, at 17 apiece. The company is the latest new issue developing cancer therapy using T cells, though it's using a different platform than the chimeric antigen receptor (CAR) T-cell therapies in the works from such recent hot IPOs as Juno Therapeutics (NASDAQ:JUNO), Kite Pharma (NASDAQ:KITE) and Bellicum Therapeutics (NASDAQ:BLCM).
Adaptimmune says that its engineered, increased affinity T-cell receptors can attack a wider range of targets than CAR T-cells. One such therapy is in early-stage clinical testing for several cancers in collaboration with GlaxoSmithKline (NYSE:GSK) .
The other T-cell therapy stocks have gotten hammered in the recent biotech selloff, which perhaps accounts for Adaptimmune's soft opening. In fact, the most recent CAR-T IPO, Cellectis (NASDAQ:CLLS), followed a similar pattern of enlarging its IPO but declining once it hit the market in late March.
Read More At Investor's Business Daily: http://news.investors.com/technology/050615-751336-adap-ipo-prices-high-but-falls-in-trading.htm#ixzz3ZT3I90Xu
Follow us: @IBDinvestors on Twitter | InvestorsBusinessDaily on Facebook
Adaptimmune Therapeutics plc (Nasdaq: ADAP) today announced the pricing of its initial public offering of 11,250,000 American Depositary Shares (ADSs) at a price to the public of $17.00 per ADS. In addition, the underwriters have been granted a 30-day option to purchase up to an additional 1,687,500 ADSs. The ADSs are expected to begin trading on the NASDAQ Global Select Market under the symbol “ADAP” on May 6, 2015. The offering is expected to close on May 11, 2015, subject to customary closing conditions. Adaptimmune estimates net proceeds from the offering will be approximately $175.7 million.
On May 6th, from IBD:
ritish biotech Adaptimmune Therapeutics (NASDAQ:ADAP) fattened its IPO and priced at the high end of its range late Tuesday, but that seemed to be more than enough for investors as the stock dropped 5.9% to close at 16 in its debut Wednesday.
Adaptimmune priced 11.3 million shares, up from the previously announced 9.4 million, at 17 apiece. The company is the latest new issue developing cancer therapy using T cells, though it's using a different platform than the chimeric antigen receptor (CAR) T-cell therapies in the works from such recent hot IPOs as Juno Therapeutics (NASDAQ:JUNO), Kite Pharma (NASDAQ:KITE) and Bellicum Therapeutics (NASDAQ:BLCM).
Adaptimmune says that its engineered, increased affinity T-cell receptors can attack a wider range of targets than CAR T-cells. One such therapy is in early-stage clinical testing for several cancers in collaboration with GlaxoSmithKline (NYSE:GSK) .
The other T-cell therapy stocks have gotten hammered in the recent biotech selloff, which perhaps accounts for Adaptimmune's soft opening. In fact, the most recent CAR-T IPO, Cellectis (NASDAQ:CLLS), followed a similar pattern of enlarging its IPO but declining once it hit the market in late March.
Read More At Investor's Business Daily: http://news.investors.com/technology/050615-751336-adap-ipo-prices-high-but-falls-in-trading.htm#ixzz3ZT3I90Xu
Follow us: @IBDinvestors on Twitter | InvestorsBusinessDaily on Facebook
Pre-Martket Trade this AM.
Interesting trade. Somebody had to have 1,000 shares before market open @ $13.18 up $.74
Wednesday, May 6, 2015
Another NIH Grant for Sangamo Collaborators !!
More positive news from the Fred Hutchinson Cancer Research Center.
Dr. Hans-Peter Kiem and Paula Cannon have received a $12.6 million Dollar grant from the NIH for additional work in stem cells. I have confirmed that Sangamo BioSciences (SGMO) is an industry partner in this project. Here is the Hutch press release:
A stem cell transplant researcher in the Clinical Research Division, Kiem is co-director of the Fred Hutch-based defeatHIV, a public-private consortium of researchers investigating the use of genetically modified stem cells to cure HIV.
Also co-principal investigator on the new project is Dr. Paula Cannon, an associate professor of molecular microbiology and immunology, pediatrics, biochemistry and molecular biology at University of Southern California’s Keck School of Medicine and a member of the defeatHIV consortium.
Dr. Hans-Peter Kiem and Paula Cannon have received a $12.6 million Dollar grant from the NIH for additional work in stem cells. I have confirmed that Sangamo BioSciences (SGMO) is an industry partner in this project. Here is the Hutch press release:
Dr. Hans-Peter Kiem co-PI on $12.6M grant to study stem cell therapy for HIV
Fred Hutchinson Cancer Research Center’s Dr. Hans-Peter Kiem is the co-principal investigator for a new, $12.6 million grant over five years from the National Heart, Lung and Blood Institute – a division of the National Institutes of Health – to research next-generation hematopoietic stem cell gene therapy for HIV control and eradication.A stem cell transplant researcher in the Clinical Research Division, Kiem is co-director of the Fred Hutch-based defeatHIV, a public-private consortium of researchers investigating the use of genetically modified stem cells to cure HIV.
Also co-principal investigator on the new project is Dr. Paula Cannon, an associate professor of molecular microbiology and immunology, pediatrics, biochemistry and molecular biology at University of Southern California’s Keck School of Medicine and a member of the defeatHIV consortium.
“As the title [of the new project] says, it really gets into the next-generation gene editing but also into studying the role and mechanism of allogeneic transplantation in HIV eradication,” Kiem said.
To date, only one person in the world has been cured of HIV – Timothy Ray Brown, also known as “the Berlin patient” after the city in which he was treated. The Seattle-born Brown received a stem cell transplant in Germany in 2007 to treat acute myeloid leukemia. His German doctor decided to try to also cure Brown’s HIV infection by finding a stem cell donor who carried two copies of a rare gene mutation that protects against HIV infection.
Using Brown’s case as a blueprint, defeatHIV plans to take an HIV-infected patient’s own stem cells and knock out or disable the gene that acts as the HIV doorway, and then return the modified cells to the patient. Researchers elsewhere are looking at similar therapies that involve genetically modified T cells rather than stem cells.
Researchers are still trying to tease out which of several factors, independently or in combination, contributed to Brown’s cure. Before his transplant, he underwent conditioning, in which his own bone marrow was destroyed by intensive chemotherapy and whole-body irradiation. His doctor then used a marrow donor with the protective gene mutation. Additionally, any remaining HIV-infected cells may have been attacked by his new immune system, a process known as graft-vs.-host disease that plays a role in curing leukemia.
These are the questions that Kiem and Cannon will address in their new project, which is funded until 2020.
To date, only one person in the world has been cured of HIV – Timothy Ray Brown, also known as “the Berlin patient” after the city in which he was treated. The Seattle-born Brown received a stem cell transplant in Germany in 2007 to treat acute myeloid leukemia. His German doctor decided to try to also cure Brown’s HIV infection by finding a stem cell donor who carried two copies of a rare gene mutation that protects against HIV infection.
Using Brown’s case as a blueprint, defeatHIV plans to take an HIV-infected patient’s own stem cells and knock out or disable the gene that acts as the HIV doorway, and then return the modified cells to the patient. Researchers elsewhere are looking at similar therapies that involve genetically modified T cells rather than stem cells.
Researchers are still trying to tease out which of several factors, independently or in combination, contributed to Brown’s cure. Before his transplant, he underwent conditioning, in which his own bone marrow was destroyed by intensive chemotherapy and whole-body irradiation. His doctor then used a marrow donor with the protective gene mutation. Additionally, any remaining HIV-infected cells may have been attacked by his new immune system, a process known as graft-vs.-host disease that plays a role in curing leukemia.
These are the questions that Kiem and Cannon will address in their new project, which is funded until 2020.
Synageva Biopharma to be acquired by Alexion Pharmaceuticals
From AP
Alexion Pharma to pay $8.4 billion for rare disease treatment developer Synageva BioPharma
Alexion Pharma to pay $8.4 billion for rare disease treatment developer Synageva BioPharma
Alexion Pharmaceuticals is spending about $8.4 billion - a huge premium — to buy Synageva BioPharma, a fellow rare disease treatment developer with no products on the market that booked a first-quarter loss of nearly $60 million.
Alexion will pay $115 in cash and a portion of its stock for each Synageva share. That puts the total per-share price at about $226, based on Alexion's Tuesday closing price.
That's more than double the value Synageva BioPharma Corp.'s closing price of $95.87 on Tuesday. Shares of the drugmaker are soaring in premarket trading
Clinical scale zinc finger nuclease mediated gene editing of PD-1 in tumor infiltrating lymphocytes for the treatment of metastatic melanoma.
Several Sangamo BioSciences authors contributed to this important research.
Clinical scale zinc finger nuclease mediated gene editing of PD-1 in tumor infiltrating lymphocytes for the treatment of metastatic melanoma.
Beane JD1, Lee G2, Zheng Z3, Mendel M2, Abate-Daga D4, Bharathan M3, Black M3, Gandhi N2, Yu Z3, Chandran S3, Giedlin M2, Ando D2, Miller J2, Paschon D2, Guschin D2, Rebar EJ2, Reik A2, Holmes MC2, Gregory PD2, P Restifo N3, Rosenberg SA3, Morgan RA5, Feldman SA3.
Programmed cell death-1 (PD-1) is expressed on activated T cells and represents an attractive target for gene-editing of tumor targeted T cells prior to adoptive cell transfer (ACT). We used zinc finger nucleases (ZFNs) directed against the gene encoding human PD-1 (PDCD-1) to gene-edit melanoma TIL. We show that our clinical scale TIL production process yielded efficient modification of the PD-1 gene locus, with an average modification frequency of 74.8% (n=3, range 69.9 - 84.1%) of the alleles in a bulk TIL population, which resulted in a 76% reduction in PD-1 surface-expression. Forty to 48% of PD-1 gene-edited cells had biallelic PD-1 modification. Importantly, the PD-1 gene-edited TIL product showed improved in vitro effector function and a significantly increased polyfunctional cytokine profile (TNFα, GM-CSF and IFNγ) compared to unmodified TIL in two of the three donors tested. In addition, all donor cells displayed an effector memory phenotype and expanded approximately 500 - 2000 fold in vitro. Thus, further study to determine the efficiency and safety of adoptive cell transfer using PD-1 gene-edited TIL for the treatment of metastatic melanoma is warranted.Molecular Therapy (2015); doi:10.1038/mt.2015.71.
Clinical scale zinc finger nuclease mediated gene editing of PD-1 in tumor infiltrating lymphocytes for the treatment of metastatic melanoma.
Beane JD1, Lee G2, Zheng Z3, Mendel M2, Abate-Daga D4, Bharathan M3, Black M3, Gandhi N2, Yu Z3, Chandran S3, Giedlin M2, Ando D2, Miller J2, Paschon D2, Guschin D2, Rebar EJ2, Reik A2, Holmes MC2, Gregory PD2, P Restifo N3, Rosenberg SA3, Morgan RA5, Feldman SA3.
Abstract
Tuesday, May 5, 2015
Role of the macrophage in HIV-associated neurocognitive disorders and other comorbidities in patients on effective antiretroviral treatment
Abstract
Combination antiretroviral therapy (ART) has altered the outcomes of HIV infection in treated populations by greatly reducing the incidence of opportunistic infections, cancer, and HIV-associated dementia. Despite these benefits, treated patients remain at high risk of chronic diseases affecting the peripheral organs and brain. Generally, these morbidities are attributed to persistence of latent HIV in resting T cells, chronic inflammation, and metabolic effects of ART. This review makes the case that monocytes/macrophages warrant attention as persistent reservoirs of HIV under ART, source of systemic and brain inflammation, and important targets for HIV eradication to control chronic HIV diseases.
Date: 02 May 2015
GSK files for Gene Therapy approval in Europe
From Reuters:
http://www.reuters.com/article/2015/05/05/us-gsk-genetherapy-idUSKBN0NQ0TR20150505
GlaxoSmithKline said on Tuesday it had submitted a gene therapy for approval in Europe, becoming the first big drugmaker to seek marketing authorization for the technology to fix faulty genes.
Reuters reported last week that the move was imminent. It marks the latest sign of a renaissance in gene therapy after some disastrous clinical trial results in the late 1990s and early 2000s.
GSK's product, developed with Italian scientists, is designed to treat a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no suitable bone marrow donor can be found.
http://www.reuters.com/article/2015/05/05/us-gsk-genetherapy-idUSKBN0NQ0TR20150505
GlaxoSmithKline said on Tuesday it had submitted a gene therapy for approval in Europe, becoming the first big drugmaker to seek marketing authorization for the technology to fix faulty genes.
Reuters reported last week that the move was imminent. It marks the latest sign of a renaissance in gene therapy after some disastrous clinical trial results in the late 1990s and early 2000s.
GSK's product, developed with Italian scientists, is designed to treat a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no suitable bone marrow donor can be found.
Monday, May 4, 2015
U PENN Recieves $7.5 million grant from NIH for UPENN/Sangamo HIV research
Penn Medicine Researchers Receive $7.5 Million to Expand HIV Gene Therapy Work
Penn-led team will engineer T cells to be resistant to HIV-1 infection
PHILADELPHIA – Researchers from the Perelman School of Medicine and the Penn Center for AIDS Research (CFAR) have been awarded $7.5 million over five years from the National Institutes of Health to initiate a multi-project HIV study investigating a new gene therapy approach to render immune cells of HIV positive patients resistant to the virus.
The project, entitled “Engineering T cells to Provide Durable Control of HIV-1 Replication,” includes principal investigator James L. Riley, PhD, associate professor of Microbiology, Pablo Tebas, MD, director of the AIDS Clinical Trials Unit at the Penn CFAR, James Hoxie, MD, professor of Medicine at Penn and director of the Penn CFAR, Michael C. Holmes, PhD, VP Research at Sangamo BioSciences Inc., E. John Wherry, PhD, professor of Microbiology and director of the Institute for Immunology, and Frederick D. Bushman, PhD, professor of Microbiology.
The Penn-led team, in collaboration with Sangamo, will investigate the ability of a synthetic molecule consisting of a viral entry inhibitor called C34 fused to CXCR4, an HIV co-receptor used by the virus to enter and infect T cells. Building upon the success of past studies that utilized a zinc finger nuclease (ZFN) technology to disrupt the other major viral entry factor, CCR5, the new Penn project—in both preclinical and clinical studies—aims to safely build an army of modified, HIV-1 resistant T cells in HIV infected patients using a lentiviral technology to express the C34-CXCR4 molecule. This approach, researchers believe, will make more CD4 T cells resistant to the virus and thus may re-invigorate the immune response to control HIV-1 replication in the absence of antiretroviral drug therapy (ADT).
The grant is funded under NIH’s U19 Research Program, which funds collaborative projects involving multiple institutions, including an industry collaborator. The purpose of this funding opportunity, supported by the National Institute of Allergy and Infectious Disease, National Heart, Lung, and Blood Institute, and the National Institute of Mental Health, is to encourage investigators to work together on innovative approaches to eliminate HIV and leverage the expertise and resources of the participating institutes.
For the preclinical work on the Penn project, researchers will learn more about how the mechanism by C34-CXCR4 provides such robust protection of CD4 T cells and apply chimeric antigen receptor (CAR) technology to re-direct the HIV-1 immune response, among other laboratory efforts. As part of the phase I study, researchers will manufacture the engineered T cells (taken from a patients’ own immune system) and then infuse them back into HIV infected patients taken off ADT to determine their resistance and survival abilities and anti-viral effects.
The work is an important extension of the HIV gene therapy work conducted by researchers at the Penn CFAR and the Perelman School of Medicine over the last decade.
Most recently, reporting in the New England Journal of Medicine in 2014, Penn researchers successfully genetically engineered the immune cells of 12 HIV positive patients to resist infection, and decreased the viral loads of some patients taken off ADT entirely—including one patient whose levels became undetectable. The group used the ZFN technology developed by Sangamo to modify the T cells in the patients—a “molecular scissors,” of sorts, to mimic the CCR5-delta-32 mutation. That rare mutation is of interest because it provides a natural resistance to the virus, but in only 1 percent of the general population. By inducing the mutations, the scientists reduced the expression of CCR5 surface proteins. Without those, HIV cannot enter, rendering the patients’ cells resistant to infection. In April 2015, the researchers received the National Clinical Research Achievement Award for the paper.
“In this next trial, we hypothesize that we will observe a significant loss in viral load for a longer period of time because we are able to protect CD4 T cells from different classes of HIV with the C34-CXCR4 approach,” Riley said. “This is an important component of the HIV gene therapy work here at Penn, as it will tell us how these approaches stack up against each other. We are ultimately trying to find effective, lasting ways to eliminate the need for lifelong ADT for HIV infected patients, and we believe these projects will help find the answers to some of the hard questions surrounding that goal.”
The NIH grant number for this project is U19 AI117950.
The project, entitled “Engineering T cells to Provide Durable Control of HIV-1 Replication,” includes principal investigator James L. Riley, PhD, associate professor of Microbiology, Pablo Tebas, MD, director of the AIDS Clinical Trials Unit at the Penn CFAR, James Hoxie, MD, professor of Medicine at Penn and director of the Penn CFAR, Michael C. Holmes, PhD, VP Research at Sangamo BioSciences Inc., E. John Wherry, PhD, professor of Microbiology and director of the Institute for Immunology, and Frederick D. Bushman, PhD, professor of Microbiology.
The Penn-led team, in collaboration with Sangamo, will investigate the ability of a synthetic molecule consisting of a viral entry inhibitor called C34 fused to CXCR4, an HIV co-receptor used by the virus to enter and infect T cells. Building upon the success of past studies that utilized a zinc finger nuclease (ZFN) technology to disrupt the other major viral entry factor, CCR5, the new Penn project—in both preclinical and clinical studies—aims to safely build an army of modified, HIV-1 resistant T cells in HIV infected patients using a lentiviral technology to express the C34-CXCR4 molecule. This approach, researchers believe, will make more CD4 T cells resistant to the virus and thus may re-invigorate the immune response to control HIV-1 replication in the absence of antiretroviral drug therapy (ADT).
The grant is funded under NIH’s U19 Research Program, which funds collaborative projects involving multiple institutions, including an industry collaborator. The purpose of this funding opportunity, supported by the National Institute of Allergy and Infectious Disease, National Heart, Lung, and Blood Institute, and the National Institute of Mental Health, is to encourage investigators to work together on innovative approaches to eliminate HIV and leverage the expertise and resources of the participating institutes.
For the preclinical work on the Penn project, researchers will learn more about how the mechanism by C34-CXCR4 provides such robust protection of CD4 T cells and apply chimeric antigen receptor (CAR) technology to re-direct the HIV-1 immune response, among other laboratory efforts. As part of the phase I study, researchers will manufacture the engineered T cells (taken from a patients’ own immune system) and then infuse them back into HIV infected patients taken off ADT to determine their resistance and survival abilities and anti-viral effects.
The work is an important extension of the HIV gene therapy work conducted by researchers at the Penn CFAR and the Perelman School of Medicine over the last decade.
Most recently, reporting in the New England Journal of Medicine in 2014, Penn researchers successfully genetically engineered the immune cells of 12 HIV positive patients to resist infection, and decreased the viral loads of some patients taken off ADT entirely—including one patient whose levels became undetectable. The group used the ZFN technology developed by Sangamo to modify the T cells in the patients—a “molecular scissors,” of sorts, to mimic the CCR5-delta-32 mutation. That rare mutation is of interest because it provides a natural resistance to the virus, but in only 1 percent of the general population. By inducing the mutations, the scientists reduced the expression of CCR5 surface proteins. Without those, HIV cannot enter, rendering the patients’ cells resistant to infection. In April 2015, the researchers received the National Clinical Research Achievement Award for the paper.
“In this next trial, we hypothesize that we will observe a significant loss in viral load for a longer period of time because we are able to protect CD4 T cells from different classes of HIV with the C34-CXCR4 approach,” Riley said. “This is an important component of the HIV gene therapy work here at Penn, as it will tell us how these approaches stack up against each other. We are ultimately trying to find effective, lasting ways to eliminate the need for lifelong ADT for HIV infected patients, and we believe these projects will help find the answers to some of the hard questions surrounding that goal.”
The NIH grant number for this project is U19 AI117950.
Sunday, May 3, 2015
Dale Ando earns prestigious FDA appointment
Dale Ando M.D., Vice President Therapeutic Development and Chief Medical Officer of Sangamo BioSciences (SGMO) has been appointed to the Cellular, Tissue and Gene Therapies Advisory Committee of the FDA. His term runs from 4/1/2015 to 3/31/2019. His area of expertise is Industry Representative.
Saturday, May 2, 2015
John Zaia, "The Goal is a Functional Cure"
In an interview in the New Scientist Magazine, principal investigator for the soon to begin Sangamo BioSciences (SGMO) stem cell HIV trial John Zaia states " The Goal is a Functional Cure".
John Zaia, M.D. is the Institutional Official for Research, City of Hope, Duarte CA.
It is great to hear him speak with such confidence.
The article also touches on the upcoming sickle cell trial:
"The trial blazes a path for using the approach to treat other diseases. For example, another trial set to start soon will focus on sickle cell disease, in which the oxygen-carrying haemoglobin molecules in red blood cells are abnormal. The technique would switch on a protein that can be used instead of the haemoglobin."
http://www.newscientist.com/article/mg22630194.200-human-gene-editing-has-arrived--heres-why-it-matters.html?full=true#.VUS-YZ0o7vZ
John Zaia, M.D. is the Institutional Official for Research, City of Hope, Duarte CA.
It is great to hear him speak with such confidence.
The article also touches on the upcoming sickle cell trial:
"The trial blazes a path for using the approach to treat other diseases. For example, another trial set to start soon will focus on sickle cell disease, in which the oxygen-carrying haemoglobin molecules in red blood cells are abnormal. The technique would switch on a protein that can be used instead of the haemoglobin."
http://www.newscientist.com/article/mg22630194.200-human-gene-editing-has-arrived--heres-why-it-matters.html?full=true#.VUS-YZ0o7vZ
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