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.
 
By Gareth MacDonald+, 29-May-2015
CRISPR patent claimant Cellectis has declined to comment on rumours US drug giant Pfizer is in talks about a €1.5bn takeover.

 

Tuesday, May 26, 2015

Jefferies June 1st 1:30 EST

Sangamo BioSciences Announces Presentation At The Jefferies 2015 Global Healthcare Conference


RICHMOND, Calif., May 26, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced today that Edward Lanphier, Sangamo's president and CEO, will provide an update on the progress of Sangamo's ZFP Therapeutic® development programs and an overview of the company's business strategy at 1:30pm ET on Monday, June 1, 2015, at the Jefferies 2015 Global Healthcare Conference. The conference is being held in New York, NY.

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.

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.”

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.
uniQure CEO Jörn Aldag
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."

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)