The relevant section:
If the idea that a virus can improve health still seems a bit far-fetched, know that viruses aren’t the only solution, though the alternatives have yet to make it through clinical trials and some are experiencing setbacks. Scientists may also use tools such as zinc-finger nucleases or Crispr. Both methods can cut a strand of DNA in two, remove a problematic base pair, and insert a new section, but neither of these methods has passed clinical trials. Sangamo BioSciences in Richmond, California, is currently testing zinc-finger nucleases as a way to interrupt a protein that HIV relies on to attack immune cells.
http://www.ibtimes.com/how-live-longer-scientists-are-pushing-make-120-new-70-1900609
Thursday, April 30, 2015
PJ Analyst Schimmer " even modest penatration of sickle cell market will be worth over $1 Billion"
To flesh out the report that JBWIN posted on InvestorVillage this morning, I want to add a comment found on the analystratings.com website. This Website contains a quote from PJ analyst Joshua Schimmer that reiterates even a minor penetration of the sickle cell market is worth $1 Billion dollars. http://www.analystratings.com/2015/04/30/biogen-or-bluebird-which-will-lead-in-scd/402054/
"Schimmer also analyzes the pipeline drug developed in partnership between Biogen and Sangamo BioSciences (NASDAQ: SGMO), though he focuses his analysis on Biogen. The two companies are working on a ZFN approach to target BCL11a, meaning the use of artificial enzymes to target a specific protein. Schimmer notes that this approach offers “a viable alternative” to LentiGlobin. If the product is successful, Schimmer believes that “even modest penetration” in the market will deliver over $1 billion. "
I also wanted to reemphasize the point that the PJ analysts made yesterday, that Sangamo has a very good chance of its therapy succeeding.
Piper hosted a call on sickle cell. The focus was largely on what is likely to be technically required for a gene therapy approach to work. Regarding SGMO, the analysis seems to indicate that knock-out of Bcl11a is likely to succeed, with the ever-present caveats of impact of off-target DNA breaks as well as off-target effects of the desired genome modification. The above mentioned erythroid-specific-enhancer knockout for Bcl11a we think typifies the sophistication of Sangamo’s approach to gene therapy. In any case, the Piper analysts estimate that roughly 10-30% bone marrow chimerism could rescue sickle cell, and our discussions with SGMO suggest that level of rescue should be within reach.
"Schimmer also analyzes the pipeline drug developed in partnership between Biogen and Sangamo BioSciences (NASDAQ: SGMO), though he focuses his analysis on Biogen. The two companies are working on a ZFN approach to target BCL11a, meaning the use of artificial enzymes to target a specific protein. Schimmer notes that this approach offers “a viable alternative” to LentiGlobin. If the product is successful, Schimmer believes that “even modest penetration” in the market will deliver over $1 billion. "
I also wanted to reemphasize the point that the PJ analysts made yesterday, that Sangamo has a very good chance of its therapy succeeding.
Piper hosted a call on sickle cell. The focus was largely on what is likely to be technically required for a gene therapy approach to work. Regarding SGMO, the analysis seems to indicate that knock-out of Bcl11a is likely to succeed, with the ever-present caveats of impact of off-target DNA breaks as well as off-target effects of the desired genome modification. The above mentioned erythroid-specific-enhancer knockout for Bcl11a we think typifies the sophistication of Sangamo’s approach to gene therapy. In any case, the Piper analysts estimate that roughly 10-30% bone marrow chimerism could rescue sickle cell, and our discussions with SGMO suggest that level of rescue should be within reach.
Tuesday, April 28, 2015
ViroMed in Phase III Gene Therapy for DN
This should look familiar to long term investors in Sangamo BioSciences (SGMO) although SGMO did not get past phase II in Diabetic Neuropathy.
VM202-DPN is an innovative new biopharmaceutical that can induce the regeneration of damaged microvasculature and nerve cells
□ Differs from currently used pain killers by providing fundamental treatment for the disease
ViroMed Co., Ltd. (KOSDAQ: 084990:KS) today announced that VM202-DPN, the company's proprietary DNA medicine for the treatment of diabetic peripheral neuropathy (DPN), has received approval from the US FDA for a Phase III clinical trial.
VM202-DPN is an innovative new drug that induces regeneration of damaged microvasculature and nerve cells. When VM202-DPN is injected, a protein called Hepatocyte Growth Factor (HGF) is produced around the injection sites, which induces the formation of new blood vessels and regeneration of nerve cells. The HGF protein produced can treat the extreme pain experienced by diabetic peripheral neuropathy patients. This pain relieving effect has been confirmed through phase I and II clinical study conducted in both the US and Korea.
ViroMed submitted phase III IND based on these results and received approval from US FDA without any additional modification. This indicates that the US FDA, once again, confirms the superb safety and efficacy profile of VM202-DPN. VM202-DPN phase III will be a double-blind study targeting diabetic peripheral neuropathy patients distributed between placebo and VM202-DPN treated groups. The total number of patients will be 477 and the efficacy evaluation will be based on comparison with the placebo group.
Mr. Yong Soo Kim (CEO) commented “If VM202-DPN phase III is successfully completed, it will be the first Korean biopharmaceutical to challenge entry into global market. Currently, we are considering partnership with a global pharmaceutical company to enter the global market.”.
VM202-DPN is an innovative new biopharmaceutical that can induce the regeneration of damaged microvasculature and nerve cells
□ Differs from currently used pain killers by providing fundamental treatment for the disease
ViroMed Co., Ltd. (KOSDAQ: 084990:KS) today announced that VM202-DPN, the company's proprietary DNA medicine for the treatment of diabetic peripheral neuropathy (DPN), has received approval from the US FDA for a Phase III clinical trial.
VM202-DPN is an innovative new drug that induces regeneration of damaged microvasculature and nerve cells. When VM202-DPN is injected, a protein called Hepatocyte Growth Factor (HGF) is produced around the injection sites, which induces the formation of new blood vessels and regeneration of nerve cells. The HGF protein produced can treat the extreme pain experienced by diabetic peripheral neuropathy patients. This pain relieving effect has been confirmed through phase I and II clinical study conducted in both the US and Korea.
ViroMed submitted phase III IND based on these results and received approval from US FDA without any additional modification. This indicates that the US FDA, once again, confirms the superb safety and efficacy profile of VM202-DPN. VM202-DPN phase III will be a double-blind study targeting diabetic peripheral neuropathy patients distributed between placebo and VM202-DPN treated groups. The total number of patients will be 477 and the efficacy evaluation will be based on comparison with the placebo group.
Mr. Yong Soo Kim (CEO) commented “If VM202-DPN phase III is successfully completed, it will be the first Korean biopharmaceutical to challenge entry into global market. Currently, we are considering partnership with a global pharmaceutical company to enter the global market.”.
Sangamo BioSciences Dominates at ASGCT 2015
The American Society for Gene and Cell Therapy has released abstracts for ASGCT 2015.
Sangamo BioSciences (SGMO) has over 20 oral and poster presentations of their ZFN technology by the company or their collaborators.
The abstracts can be viewed here: http://www.abstracts2view.com/asgct/
JBWIN on the Investor Village Message Board has posted the abstracts to save you the search time. The IV board can be viewed here: http://www.investorvillage.com/smbd.asp?mb=1933
Start with message 72663.
Sangamo BioSciences (SGMO) has over 20 oral and poster presentations of their ZFN technology by the company or their collaborators.
The abstracts can be viewed here: http://www.abstracts2view.com/asgct/
JBWIN on the Investor Village Message Board has posted the abstracts to save you the search time. The IV board can be viewed here: http://www.investorvillage.com/smbd.asp?mb=1933
Start with message 72663.
Dow Agro Deal to Bolster Sangamo's Bottom Line
Sangamo Biosciences (SGMO) receives license fees and royalties from Dow Agro for licensing Sangamo's technology. Dow has named the product Exazct Precision Technology.
Dow AgroSciences LLC, a wholly owned subsidiary of The Dow Chemical Company (NYSE: DOW), Arcadia Biosciences, Inc. and Bioceres, S.A. announced today an agreement to develop and commercialize innovative traits in soybeans. The collaboration leverages Dow AgroSciences’ technology, regulatory expertise, and commercial seed capabilities with a leading soybean abiotic stress platform and unique grower relationships in South America represented by Verdeca LLC, a joint venture between Arcadia and Bioceres.
Under the collaboration, the companies will develop new soybean traits using Dow AgroSciences’ EXZACT™ Precision Technology platform to generate soybean trait stacks. These stacks will combine Verdeca’s agronomic performance and product quality traits with Dow AgroSciences’ herbicide-tolerant and insect-resistant traits. The EXZACT Precision Technology platform will facilitate the development of multiple trait stacks with greater degrees of precision and speed-to-market. This collaboration is expected to result in products that provide soybean growers around the world with unique and powerful options to achieve greater yields and improved on-farm economics. Dow AgroSciences has developed the EXZACT Precision Technology platform under an exclusive license and collaboration agreement in plants with Sangamo BioSciences, Inc
Dow AgroSciences LLC, a wholly owned subsidiary of The Dow Chemical Company (NYSE: DOW), Arcadia Biosciences, Inc. and Bioceres, S.A. announced today an agreement to develop and commercialize innovative traits in soybeans. The collaboration leverages Dow AgroSciences’ technology, regulatory expertise, and commercial seed capabilities with a leading soybean abiotic stress platform and unique grower relationships in South America represented by Verdeca LLC, a joint venture between Arcadia and Bioceres.
Under the collaboration, the companies will develop new soybean traits using Dow AgroSciences’ EXZACT™ Precision Technology platform to generate soybean trait stacks. These stacks will combine Verdeca’s agronomic performance and product quality traits with Dow AgroSciences’ herbicide-tolerant and insect-resistant traits. The EXZACT Precision Technology platform will facilitate the development of multiple trait stacks with greater degrees of precision and speed-to-market. This collaboration is expected to result in products that provide soybean growers around the world with unique and powerful options to achieve greater yields and improved on-farm economics. Dow AgroSciences has developed the EXZACT Precision Technology platform under an exclusive license and collaboration agreement in plants with Sangamo BioSciences, Inc
Sunday, April 26, 2015
Phil Gregory Speaking at MIT Synthetic Biology Workshop Today
Philip D. Gregory, D. Phil. , Chief Scientific Officer and Senior Vice President, Research of Sangamo BioSciences (SGMO) is presenting at mSBW2.0. This is the International Workshop on Mamilian Synthetic Biology 2.0 Recognizing the fast emergence and potential significance of this field, the aim of this workshop series is to bring practitioners of mammalian synthetic biology together with experts from other relevant fields. The general goals of the workshop are to nucleate the nascent mammalian synthetic biology community, reach out to experts from other fields that can benefit from and contribute to this field, and define the important challenges and future directions. The workshop provides a forum for exposition of the latest developments in the field and discussions of how experts from other fields can benefit from and contribute to mammalian synthetic biology.
Agenda
| Saturday April 25, 2015 | |
| 8:00 | Registration |
| 8:15 | Welcome and Introduction |
| Ron Weiss, MIT | |
| 8:30 | Keynote Speaker |
| Professor Elaine Fuchs, The Rockefeller University: "Skin Stem Cells and Their Cancers" | |
| 9:30 – 10:30 | Session 1 - Foundational Technologies |
| George Church, Harvard University: "Engineering mammalian genetic systems, neuro-systems and ecosystems" | |
| Remus Wong, Stanford University: RNA Regulation of Expression | |
| 10:30 – 10:45 | Break |
| 10:45 – 12:15 | Session 2 - Directing Cell Phenotype |
| Charles Gersbach, Duke University: "Epigenome Engineering to Control Cell Phenotype" | |
| Wendell Lim, UCSF: "Redesigning the T Cell" | |
| Wilson Wong, Boston University: "Robust and scalable biocomputers in mammalian cells" (short talk) | |
| Michael Todhunter, UCSF: "Rapid Synthesis of 3D Tissues by Chemically Programmed Assembly" (short talk) | |
| 12:15 – 1:30 | Lunch |
| 1:30 – 3:00 | Session 3 - Design and Optimization of Genetic Circuits |
| Michael Elowitz, Caltech: "Design principles for signaling and memory circuits" | |
| Leonidas Bleris, University of Texas, Dallas: "Custom architectures for control in mammalian cells" | |
| Silvana Konermann, MIT: "An engineered CRISPR/Cas9 complex for genome-scale transcriptional activation" (short talk) | |
| Tasuku Kitada, MIT: "RNA encoded circuits with small molecular based regulation for 'smart vaccination'" (short talk) | |
| 3:00 – 3:15 | Break |
| 3:15 – 4:45 | Session 4 - Functions of Multicellular Systems |
| Ed Boyden, MIT: "Tools for Mapping and Controlling Complex Biological Systems" | |
| Geraldine Hamilton, Emulate: "Organs-on-Chips: A unique window into human biology and disease" | |
| Jonathan Brunger, Duke University: "Targeted genome engineering of iPSCs to produce auto-regulated inflammation resistance for regenerative medicine" (short talk) | |
| Daniel Woodsworth, Genome Sciences Centre: "A lymphocyte based cell-to-cell therapeutic delivery system" (short talk) | |
| 4:45 – 5:00 | Break |
| 5:00 – 6:00 | Session 5 - Gene Regulation and Epigenetic Control |
| Karmella Haynes, Arizona State University: "Regulating human cancer epigenomes with synthetic chromatin" | |
| Kobi Benenson, ETH: "Biological Computing Meets Synthetic Biology" | |
| 6:00 - 8:00 | Poster Session and Reception |
| Sunday, April 26, 2015 | |
| 8:00 – 8:30 | Breakfast |
| 8:30-9:30 | Closing Plenary |
| Professor David Baltimore, California Institute of Technology: "Engineering the Immune System" | |
| 9:30-10:30 | Session 1 - Circuits and Pathways |
| Joshua Leonard, Northwestern University: "Engineering Cell-based Devices that Interface Robustly with Host Physiology" | |
| Timothy Lu, MIT: "Massively Parallel Combinatorial Genetics" | |
| 10:30-10:45 | Break |
| 10:45-12:00 | Session 2: Industry Research |
| Sean Stevens, Synthetic Genomics, Inc.: "Synthetic Biology and Cell Engineering for Biomedical Use" | |
| Jon Chesnut, Thermo Fisher Scientific: "Democratizing cell engineering: New tools for simplifying and shortening the genome editing workflow" | |
| Jose-Carlos Gutierrez-Ramos, Pfizer BioTherapeutics | |
| Philip Gregory, Sangamo Biosciences: "Therapeutic Genome Editing - An Industry Perspective" | |
| Emily Leproust, Twist Biosciences | |
| 12:00-1:00 | Lunch |
| 1:00-2:30 | Session 3 - Towards cell-based therapies |
| Michel Sadelain, Memorial Sloan Kettering Cancer Center: "CAR therapy and the promise of T cell engineering" | |
| Yvonne Chen, University of California: "Engineering Smarter and Stronger T Cells for Cancer Therapy" | |
| 2:30-2:45 | Closing Remarks |
| Ron Weiss, MIT | |
| 2:45 | Adjourn |
Saturday, April 25, 2015
BMO Capital Markets Initiates Coverage of Bluebird Bio (BLUE) $160 Target !
Interesting.
Based on this analysis" We believe that BLUE has established the broadest clinical proof-of-concept in the gene therapy space, with its proprietary lentivirus vector platform
and phase 1/2 program in beta-thalassemia and phase 2/3 program in childhood cerebral adrenoleukodystrophy. With full economics retained for its key lead programs, we believe that benefit in transfusion independence seen in beta-thalassemia could translate into a $1.2 billion commercial opportunity following pivotal data and that benefit in stabilization of disease progression in CCALD, while representing a smaller $280 million peak opportunity, provides additional validation for its gene therapy platform.”
501 Canal Street, please take note.
In other BLUE news insider Mitchell H. Finer sold 15,000 shares of the company’s stock in a transaction dated Monday, April 20th. The stock was sold at an average price of $126.03, for a total value of $1,890,450.00.
Based on this analysis" We believe that BLUE has established the broadest clinical proof-of-concept in the gene therapy space, with its proprietary lentivirus vector platform
and phase 1/2 program in beta-thalassemia and phase 2/3 program in childhood cerebral adrenoleukodystrophy. With full economics retained for its key lead programs, we believe that benefit in transfusion independence seen in beta-thalassemia could translate into a $1.2 billion commercial opportunity following pivotal data and that benefit in stabilization of disease progression in CCALD, while representing a smaller $280 million peak opportunity, provides additional validation for its gene therapy platform.”501 Canal Street, please take note.
In other BLUE news insider Mitchell H. Finer sold 15,000 shares of the company’s stock in a transaction dated Monday, April 20th. The stock was sold at an average price of $126.03, for a total value of $1,890,450.00.
Happy DNA Day. Celebrate the Discovery of Watson and Crick
According to the Oxford University Press website today is DNA DAY 2015.
From the OUP Blog:
25 April is a joint celebration for geneticists, commemorating the discovery of the helix nature of DNA by James Watson and Francis Crick in 1953 and the completion of the human genome project fifty years later in 2003.
It may have taken half a century to map the human genome, but in the years since its completion the field of genetics has seen breakthroughs increase at an ever-accelerating rate. In 2015, gene therapy is fast becoming a viable option for the treatment of genetic diseases and will be a dominant focus for geneticists in the years to come.
- See more at: http://blog.oup.com/2015/04/dna-day-2015-genetics-gene-therapy-infographic/
From the OUP Blog:
25 April is a joint celebration for geneticists, commemorating the discovery of the helix nature of DNA by James Watson and Francis Crick in 1953 and the completion of the human genome project fifty years later in 2003.
It may have taken half a century to map the human genome, but in the years since its completion the field of genetics has seen breakthroughs increase at an ever-accelerating rate. In 2015, gene therapy is fast becoming a viable option for the treatment of genetic diseases and will be a dominant focus for geneticists in the years to come.
- See more at: http://blog.oup.com/2015/04/dna-day-2015-genetics-gene-therapy-infographic/
Friday, April 24, 2015
Cytotoxic Effects during Knock Out of Multiple Porcine Endogenous Retrovirus (PERV) Sequences in the Pig Genome by Zinc Finger Nucleases (ZFN)
Well it looks like this approach is not feasible.
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0122059
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0122059
Abstract
Xenotransplantation has been proposed as a solution to the shortage of suitable human donors for transplantation and pigs are currently favoured as donor animals. However, xenotransplantation may be associated with the transmission of zoonotic microorganisms. Whereas most porcine microorganisms representing a risk for the human recipient may be eliminated by designated pathogen free breeding, multiple copies of porcine endogenous retroviruses (PERVs) are integrated in the genome of all pigs and cannot be eliminated this way. PERVs are released as infectious particles and infect human cells. The zinc finger nuclease (ZFN) technology allows knocking out specifically cellular genes, however it was not yet used to eliminate multiple integrated proviral sequences with a strong conservation in the target sequence. To reduce the risk of horizontal PERV transmission and to knock out as many as possible proviruses, for the first time the powerful tool of the ZFN technology was used. ZFN were designed to bind specifically to sequences conserved in all known replication-competent proviruses. Expression and transport of the ZFN into the nucleus was shown by Western blot analysis, co-localisation analysis, PLA and FRET. Survival of transfected cells was analysed using fluorescent ZFN and cell counting. After transfection a strong expression of the ZFN proteins and a co-localisation of the expressed ZFN proteins were shown. However, expression of the ZFN was found to be extremely toxic for the transfected cells. The induced cytotoxicity was likely due to the specific cutting of the high copy number of the PERV proviruses, which is also commonly observed when ZFN with low specificity cleave numerous off-target sites in a genome. This is the first attempt to knock out multiple, nearly identical, genes in a cellular genome using ZFN. The attempt failed, and other strategies should be used to prevent PERVShort Interest continues slight decline
Sangamo BioSciences (SGMO) currently has over 8 million shares held short. That is out of 69.2 million shares outstanding. With average daily volume of 722k, that means it would take 11 days to cover.
DATE SHORT INTEREST
4/15/15 8,050,307
3/31/15 8,285,803
3/13/15 8,441,291
2/27/15 8,939,000
2/13/15 9,268,065
1/30/15 9,082,814
1/15/15 9,387,913
DATE SHORT INTEREST
4/15/15 8,050,307
3/31/15 8,285,803
3/13/15 8,441,291
2/27/15 8,939,000
2/13/15 9,268,065
1/30/15 9,082,814
1/15/15 9,387,913
In South Africa 40% fail to follow ARV protocols after 3 years: Resistence Looms
The success of HAART has had some individuals question the value of Sangamo's (SGMO) quest for a functional cure. David Sobek, Associate Professor of Political Science at LSU is one of these.
Adam Feuerstein, B.S. in Political Science from Emory is another. This 350 page report by the FDA contains discussion of actual HAART patients and their difficulties with the current therapy.
http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm363982.pdf
Here is the current article from South Africa that ties this together:
http://www.timeslive.co.za/thetimes/2015/01/11/aids-update-arv-resistance-looms-as-40-fail-to-follow-regimen
And dwindling funding threatens the advancements made last year that have brought down the infection rate.
"We have made progress but the battle's not won. A fifth of the new infections in the world are taking place in South Africa," said Fareed Abdullah, CEO of the SA National Aids Council.
Nearly 40% of people on ARVs don't take them as instructed after three years, amounting to hundreds of thousands of people at risk of drug resistance.
Adam Feuerstein, B.S. in Political Science from Emory is another. This 350 page report by the FDA contains discussion of actual HAART patients and their difficulties with the current therapy.
http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm363982.pdf
Here is the current article from South Africa that ties this together:
http://www.timeslive.co.za/thetimes/2015/01/11/aids-update-arv-resistance-looms-as-40-fail-to-follow-regimen
And dwindling funding threatens the advancements made last year that have brought down the infection rate.
"We have made progress but the battle's not won. A fifth of the new infections in the world are taking place in South Africa," said Fareed Abdullah, CEO of the SA National Aids Council.
Nearly 40% of people on ARVs don't take them as instructed after three years, amounting to hundreds of thousands of people at risk of drug resistance.
Nobel Laureate: " HIV Breakthrough imminent". Think: Elite Controllers.
It looks like the approach Sangamo BioSciences (SGMO) is taking with a gene therapy treatment for HIV is gaining credence. With the recent modification of treatment protocols to include the ZFN modification of CD8 cells Sangamo has seen 1 of three treated patients reduce his VL to below quantification . The second had delayed onset of viremia.
http://www.timeslive.co.za/scitech/2015/04/18/hiv-breakthrough-imminent-says-medicine-nobel-laureate
Nobel Laureate Francoise Barre-Sinoussi‚ who co-discovered the virus that causes Aids‚ believes it is possible that one day people will live with HIV‚ without taking medicine and yet remain healthy and un-infectious.
Barre-Sinoussi believes a cure that will completely eliminate HIV from the body is "impossible". But she says sending the virus into remission is possible.
The scientist explained that clinical remission is when the virus is still in the body‚ but it is not replicating‚ not attacking the cells and the person cannot transmit HIV.
The virus would also not be activating the carrier's immune system and causing inflammation in the body‚ which is linked to lifestyle disease such as heart disease and cancer.
The reason she believes remission is possible is because up to three in 1000 HIV-positive people naturally send the virus into remission and never require medicine. They are called elite controllers. These are people whose bodies stop the virus replicating and remain healthy 20 years after infection without antiretroviral medication.
http://www.timeslive.co.za/scitech/2015/04/18/hiv-breakthrough-imminent-says-medicine-nobel-laureate
Nobel Laureate Francoise Barre-Sinoussi‚ who co-discovered the virus that causes Aids‚ believes it is possible that one day people will live with HIV‚ without taking medicine and yet remain healthy and un-infectious.
Barre-Sinoussi believes a cure that will completely eliminate HIV from the body is "impossible". But she says sending the virus into remission is possible.
The scientist explained that clinical remission is when the virus is still in the body‚ but it is not replicating‚ not attacking the cells and the person cannot transmit HIV.
The virus would also not be activating the carrier's immune system and causing inflammation in the body‚ which is linked to lifestyle disease such as heart disease and cancer.
The reason she believes remission is possible is because up to three in 1000 HIV-positive people naturally send the virus into remission and never require medicine. They are called elite controllers. These are people whose bodies stop the virus replicating and remain healthy 20 years after infection without antiretroviral medication.
Wednesday, April 22, 2015
1st quarter financial results
POSITIVE !
For the first quarter endedMarch 31, 2015 , Sangamo reported a
consolidated net loss of $5.3 million , or $0.08
per share, compared to a net loss of $7.6 million , or
$0.12 per share, for the same period in 2014. As of March
31, 2015 , the Company had cash, cash equivalents, marketable securities
and interest receivable of $226.1 million .
Revenues for the first quarter of 2015 were$13.5 million ,
compared to $8.1 million for the same period in 2014. First
quarter 2015 revenues were generated from the Company's collaboration agreements
with Shire International
GmbH (Shire), Biogen Inc. (Biogen), and Sigma-Aldrich Corporation (Sigma),
enabling technology agreements and research grants. The revenues recognized for
the first quarter of 2015 consisted of $12.7 million in
collaboration and enabling technology agreements and $0.8 million
in research grants, compared to $7.6 million and $0.5
million , respectively, for the same period in 2014. The increase in
collaboration agreement revenues was primarily due to increases in revenues
under the Company's collaboration agreements with Sigma and Biogen.
In the first quarter of 2015, Sangamo recognized$4.5 million
of revenues related to research services performed under the collaboration
agreement with Shire, and $1.5 million of revenues related to
research services performed under the collaboration agreement with Biogen. In
addition, pursuant to the agreements entered into with Shire in January
2012 and Biogen in January 2014 , Sangamo received upfront
payments of $13.0 million and $20.0 million ,
respectively. These payments are being recognized as revenue on a straight-line
basis over the initial six-year research term for Shire and approximately 40
months for Biogen. The Company recognized $0.5 million of the
Shire upfront payment and $1.5 million of the Biogen upfront
payment as revenue for the first quarter of 2015.
Research and development expenses were$15.0 million for the
first quarter of 2015, compared to $12.1 million for the same
period in 2014. The increase was primarily due to increases in manufacturing
expenses, external research expenses associated with our preclinical programs,
and personnel-related expenses, including stock-based compensation. General and
administrative expenses were $4.7 million for the first quarter
of 2015, compared to $3.6 million for the same period in 2014.
The increase was primarily due to increases in personnel-related expenses,
including stock-based compensation, as well as increases in legal and
professional services.
Total operating expenses for the first quarter of 2015 were$19.7
million , compared to $15.7 million for the same period in
2014.
Recent Events
Financial Guidance for 2015 The Company reiterates its earlier guidance as follows:
For the first quarter ended
Revenues for the first quarter of 2015 were
In the first quarter of 2015, Sangamo recognized
Research and development expenses were
Total operating expenses for the first quarter of 2015 were
Recent Events
- Presentation of New Clinical Data at CROI 2015 from Phase 1/2 SB-728-1101 clinical trial of ZFP Therapeutic Designed to Provide Functional Control of HIV. In February 2015 Sangamo presented new clinical data from its SB-728-T program for the potential functional cure of HIV/AIDS at the Conference on Retroviruses and Opportunistic Infections (CROI 2015). The data demonstrated that Cytoxan preconditioning combined with treatment with a CD4/CD8 zinc finger nuclease (ZFN)-modified T-cell product in (Cohort 3*) reduced viral load to below the limit of quantification in one of three subjects, and delayed onset of viremia for over 8 weeks, from the start of treatment interruption (TI), in another subject. Currently, all three patients from the cohort are undergoing TI.
- FDA Acceptance of IND to Initiate Clinical Trial of ZFP Therapeutic
Approach for Beta-thalassemia. In
February 2015 Sangamo announced that the Investigational New Drug (IND) application for SB-BCLmR-HSPC, designed to provide a one-time, long-lasting treatment for beta-thalassemia, and developed in collaboration with Biogen, was accepted by theU.S. Food and Drug Administration (FDA) and is active. The IND enables Sangamo to initiate a Phase 1/2 clinical trial of the ZFP Therapeutic in transfusion-dependent patients with beta-thalassemia major. The trial will assess the safety and tolerability, and measures of efficacy of this approach. InMay 2013 Sangamo was awarded a$6.4 million Strategic Partnership Award from CIRM, providing matching funds for preclinical work to support the IND application and Phase 1/2 clinical trial to develop this ZFP Therapeutic. The trial will be carried out at multiple centers, including UCSF Benioff Children's Hospital Oakland. - FDA Acceptance of IND Application to Initiate a Phase 1 Clinical Trial of
ZFP Therapeutic approach for HIV in Hematopoietic Stem Progenitor Cells (HSPCs).
In
February 2015 Sangamo announced that subsequent to theFDA's recent acceptance of the IND application, Sangamo and its collaborators atCity of Hope expect to initiate a Phase 1 clinical trial of ZFN-modified HSPCs. - Completion and closure of CERE-110 trial in Alzheimer's disease.
Data from a clinical trial of CERE-110 in subjects with Alzheimer's disease
(AD), acquired as part of the acquisition of
Ceregene in 2013, demonstrated that direct administration into the brain of AAV encoding the nerve growth factor gene (AAV-NGF) in subjects with AD was safe and well tolerated. As previously stated, Sangamo will not pursue this approach for potential treatment of AD and has terminated the CERE-110 program. - Presentation of Preclinical Data at 11th Annual
WORLDSymposium™ Meeting Supporting IVPRP Approach for the Treatment of LSDs.
In
February 2015 the Company presented preclinical data from its In Vivo Protein Replacement Platform (IVPRP) for the development of ZFP Therapeutics for the potential cure of lysosomal storage disorders (LSDs), at the WORLDSymposium™ 2015 Meeting. The data demonstrated that genes encoding functional human enzymes, defective in the LSDs Hunter, Hurler and Gaucher syndromes, could be inserted into the albumin locus of normal mice. Following ZFN-mediated genome editing, robust levels of protein expression were observed in the liver, blood plasma and spleen, consistent with the effective production, secretion and uptake of the functional therapeutic proteins by other cells and tissues of the body. Furthermore, elevated enzyme activity in the blood plasma was sustained over the course of the two month study. - In-Licensing of mRNA Delivery Technology and Expansion of Therapeutic
Pipeline Opportunities for In Vivo ZFN Platform. In
January 2015 Sangamo in-licensed nanoparticle technology enabling systemic mRNA delivery of ZFNs. Sangamo is developing applications of this technology to enable repeat-dosing of ZFNs in vivo, providing the opportunity to "dose to effect." The ability to re-dose could expand applications of ZFN-mediated in vivo genome editing to numerous, well-established therapeutic "knock-out" targets in the liver, such as PCSK9, a gene that regulates LDL cholesterol associated with cardiovascular disease.
Financial Guidance for 2015 The Company reiterates its earlier guidance as follows:
- Cash and Investments: Sangamo expects that its cash, cash equivalents
and marketable securities will be at least
$180 million at the end of 2015, inclusive of research funding and certain milestone payments from Shire and Biogen but exclusive of funds arising from any additional new collaborations or partnerships, equity financings or other new sources. - Revenues: Sangamo expects that revenues will be in the range of
$60 million to$70 million for 2015, inclusive of research funding and certain milestone payments from Shire and Biogen. - Operating Expenses: Sangamo expects that operating expenses will be
in the range of
$100 million to$110 million for 2015.
Positive results in Gene Therapy study
Sangamo has done work in this area and last May 21st presented the following work at 17th annual meeting of the ASCGT.
Genetic Correction of Induced Pluripotent Stem Cells from a Wiskott-Aldrich Syndrome Patient Normalizes the Immune Defects [Abstract #94]; Session: "Gene Targeting and Gene Correction,"Wednesday, May 21, 2014
Dr. Thrasher is also applying the same technique to Sickle Cell and Beta-Thalassemia. These are also areas that Sangamo BioSciences (SGMO) is active.
Here is the study reported in www.theguardian.com :
Children with a devastating immune condition appear to have been cured, after becoming the first to be given a new form of gene therapy.
Without the treatment, the patients who suffer from a rare condition called Wiskott-Aldrich syndrome, faced short life expectancies and prolonged periods in hospital.
Doctors at Great Ormond Street Hospital, who led the successful trial, said the findings marked a turning point for gene therapy and could pave the way for similar techniques being used for more common conditions, including sickle cell disease and thalassaemia.
Prof Adrian Thrasher, the immunologist who led the work, said: “This is a very powerful example of how gene therapy can offer highly effective treatment for patients with complex and serious genetic disease. It also excitingly demonstrates the potential for treatment of a large number of other diseases for which existing therapies are either unsatisfactory or unavailable.”
The findings follow several other positive trial results during the past year, which scientists say are putting to rest accusations that the promise of gene therapy has been over-hyped.
Emma Morris, an immunologist at University College London who is currently leading a gene therapy trial for leukaemia, said: “People have been working on it for ten years and it’s now becoming a reality that you can safely genetically modify cells and introduce them into people.”
In the latest study, seven children with Wiskott-Aldrich syndrome (WAS) were treated at Great Ormond Street Hospital and a separate clinic in Paris.
The genetic condition affects between one and 10 children in every million worldwide and reduces their ability to fight infection. Symptoms can include bleeding episodes, eczema and other recurrent skin infections and autoimmune disease, and in severe cases children can need to spend long periods in hospital.
The condition can be treated successfully with bone marrow transplants from a healthy donor, but this relies on finding a good match for the patient. “If you don’t have a good match, the treatment is complicated and requires lots of drugs,” said Thrasher.
All the children in the trial had recurrent skin infections, most experienced severe bleeding and one of the children was confined to a wheelchair.
For the new treatment, the scientists took the children’s own bone marrow and genetically modified it using a harmless virus to introduce a working version of the faulty gene into the patient’s immune cells.
The children were also given chemotherapy, which destroys some of the body’s existing bone marrow, before the genetically modified cells were replaced.
Following the treatment, one of the children died, from pre-existing problems unrelated to the treatment. The other six children’s immune systems showed a remarkable recovery and most of their symptoms were resolved. Over a period of two years, patients went from spending an average of 25 days in hospital before the gene therapy tozero days in hospital afterwards.
Daniel Wheeler, a 15-year-old from Bristol who took part in the trial, said the treatment has transformed his health. “I’m fine. I bruise a lot less easily, I’m not on anywhere near as many medicines and I’m getting more of an education,” he said.
“We don’t like to use the word ‘cure’, but given that all these patients had severe disease and symptoms, it’s pretty much resolved in all of them,” said Thrasher.
His team are now hoping to start new trials for patients with sickle cell disease and thalassaemia, which are among the most common inherited genetic disorders in Britain.
The scientists appear to have overcome a significant problem in some previous gene therapy trials, where the inserted gene had harmful “off target” effects, including switching on cancer-promoting genes. Previously, some children given gene therapy for other immune conditions, such as SCID (bubble baby syndrome), have gone on to develop leukaemia as a side-effect of the treatment. “The problem was they tended to turn on other genes indiscriminately, including cancer genes,” said Thrasher.
The latest technique appears to have overcome this by inserting a longer piece of DNA, meaning the gene is housed in a more natural environment that appears to control its action more effectively.
The results of the trial are “very impressive”, according to Morris, who is now involved in an extension of the trial that will include adult patients for the first time. “It’s a very different way of delivering treatments to patients,” she aded. “When the problem is a cell that doesn’t work properly, gene therapy offers a form of treatment that drugs and medicine will never be able to achieve.”
Genetic Correction of Induced Pluripotent Stem Cells from a Wiskott-Aldrich Syndrome Patient Normalizes the Immune Defects [Abstract #94]; Session: "Gene Targeting and Gene Correction,"
Without the treatment, the patients who suffer from a rare condition called Wiskott-Aldrich syndrome, faced short life expectancies and prolonged periods in hospital.
Doctors at Great Ormond Street Hospital, who led the successful trial, said the findings marked a turning point for gene therapy and could pave the way for similar techniques being used for more common conditions, including sickle cell disease and thalassaemia.
Prof Adrian Thrasher, the immunologist who led the work, said: “This is a very powerful example of how gene therapy can offer highly effective treatment for patients with complex and serious genetic disease. It also excitingly demonstrates the potential for treatment of a large number of other diseases for which existing therapies are either unsatisfactory or unavailable.”
The findings follow several other positive trial results during the past year, which scientists say are putting to rest accusations that the promise of gene therapy has been over-hyped.
Emma Morris, an immunologist at University College London who is currently leading a gene therapy trial for leukaemia, said: “People have been working on it for ten years and it’s now becoming a reality that you can safely genetically modify cells and introduce them into people.”
In the latest study, seven children with Wiskott-Aldrich syndrome (WAS) were treated at Great Ormond Street Hospital and a separate clinic in Paris.
The genetic condition affects between one and 10 children in every million worldwide and reduces their ability to fight infection. Symptoms can include bleeding episodes, eczema and other recurrent skin infections and autoimmune disease, and in severe cases children can need to spend long periods in hospital.
The condition can be treated successfully with bone marrow transplants from a healthy donor, but this relies on finding a good match for the patient. “If you don’t have a good match, the treatment is complicated and requires lots of drugs,” said Thrasher.
All the children in the trial had recurrent skin infections, most experienced severe bleeding and one of the children was confined to a wheelchair.
For the new treatment, the scientists took the children’s own bone marrow and genetically modified it using a harmless virus to introduce a working version of the faulty gene into the patient’s immune cells.
The children were also given chemotherapy, which destroys some of the body’s existing bone marrow, before the genetically modified cells were replaced.
Following the treatment, one of the children died, from pre-existing problems unrelated to the treatment. The other six children’s immune systems showed a remarkable recovery and most of their symptoms were resolved. Over a period of two years, patients went from spending an average of 25 days in hospital before the gene therapy tozero days in hospital afterwards.
Daniel Wheeler, a 15-year-old from Bristol who took part in the trial, said the treatment has transformed his health. “I’m fine. I bruise a lot less easily, I’m not on anywhere near as many medicines and I’m getting more of an education,” he said.
“We don’t like to use the word ‘cure’, but given that all these patients had severe disease and symptoms, it’s pretty much resolved in all of them,” said Thrasher.
His team are now hoping to start new trials for patients with sickle cell disease and thalassaemia, which are among the most common inherited genetic disorders in Britain.
The scientists appear to have overcome a significant problem in some previous gene therapy trials, where the inserted gene had harmful “off target” effects, including switching on cancer-promoting genes. Previously, some children given gene therapy for other immune conditions, such as SCID (bubble baby syndrome), have gone on to develop leukaemia as a side-effect of the treatment. “The problem was they tended to turn on other genes indiscriminately, including cancer genes,” said Thrasher.
The latest technique appears to have overcome this by inserting a longer piece of DNA, meaning the gene is housed in a more natural environment that appears to control its action more effectively.
The results of the trial are “very impressive”, according to Morris, who is now involved in an extension of the trial that will include adult patients for the first time. “It’s a very different way of delivering treatments to patients,” she aded. “When the problem is a cell that doesn’t work properly, gene therapy offers a form of treatment that drugs and medicine will never be able to achieve.”
Tuesday, April 21, 2015
UPenn team receives prestigious award for Sangamo sponsored HIV breakthrough
Sangamo Biosciences (SGMO) collaborators Carl June, Bruce Levine and Pablo Tebas are recipients of the prestigious Clinical Research Achievement Award for their personalized gene therapy work in HIV.
The Clinical Research Forum recognized the year’s 10 most outstanding research papers written by teams from across the nation at its 4th annual awards ceremony in Washington, DC, on April 16. The winning papers were chosen based on their degree of innovation from a pool of more than 50 nominations from 30 research and academic health centers nationwide.
The Penn team’s work, published in the New England Journal of Medicine in March 2014, was the first successful clinical test of any gene editing approach in humans. In the phase I study, they engineered the immune cells of 12 HIV positive patients to resist the HIV infection, by mimicking a naturally occurring mutation occuring in a very small number of people that renders their cells resistant to HIV infection. Researchers used a zinc finger nuclease technology to induce the genetic CCR5 mutation in patients’ T cells to lock out HIV. The modified T cells persisted in all patients, they found, and reduced viral loads in some taken off treatment entirely.
“This study shows that we can safely and effectively engineer an HIV patient’s own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs,” said June, the study’s senior author. “This reinforces our belief that modified T cells are the key that could eliminate the need for lifelong antiretroviral drug therapy and potentially lead to functionally curative approaches for HIV/AIDS.” For more on the study, read the press release here.
The Forum and its supporters believe these papers represent the best and brightest work in the field, and will lead to advancements in medicine that will change lives and patient outcomes worldwide.
The Forum advocates for increased respect for the field, which members hope will translate to sustained financial support from the National Institutes of Health, academia, foundations and other donors. “The better our clinical research, the greater our ability to diagnose, treat and prevent illness,” said Herbert Pardes, Co-Chairman of the Clinical Research Forum Board of Directors.
The Clinical Research Forum recognized the year’s 10 most outstanding research papers written by teams from across the nation at its 4th annual awards ceremony in Washington, DC, on April 16. The winning papers were chosen based on their degree of innovation from a pool of more than 50 nominations from 30 research and academic health centers nationwide.
The Penn team’s work, published in the New England Journal of Medicine in March 2014, was the first successful clinical test of any gene editing approach in humans. In the phase I study, they engineered the immune cells of 12 HIV positive patients to resist the HIV infection, by mimicking a naturally occurring mutation occuring in a very small number of people that renders their cells resistant to HIV infection. Researchers used a zinc finger nuclease technology to induce the genetic CCR5 mutation in patients’ T cells to lock out HIV. The modified T cells persisted in all patients, they found, and reduced viral loads in some taken off treatment entirely.
“This study shows that we can safely and effectively engineer an HIV patient’s own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs,” said June, the study’s senior author. “This reinforces our belief that modified T cells are the key that could eliminate the need for lifelong antiretroviral drug therapy and potentially lead to functionally curative approaches for HIV/AIDS.” For more on the study, read the press release here.
The Forum and its supporters believe these papers represent the best and brightest work in the field, and will lead to advancements in medicine that will change lives and patient outcomes worldwide.
The Forum advocates for increased respect for the field, which members hope will translate to sustained financial support from the National Institutes of Health, academia, foundations and other donors. “The better our clinical research, the greater our ability to diagnose, treat and prevent illness,” said Herbert Pardes, Co-Chairman of the Clinical Research Forum Board of Directors.
Monday, April 20, 2015
Phil Gregory, Sangamo CSO speaking at symposium with masters of the gene editing universe
Phil Gregory, Sangamo Biosciences Chief Scientific Officer is the only industry representative speaking at the UC-Berkeley symposium "Re-writing Genomes: A New Era in Genome Engineering". The event will take place August 24, 2015.
Organizers:
Dirk Hockemeyer, MCB, UC Berkeley
Jennifer Doudna, MCB, UC Berkeley, HHMI
Confirmed speakers:
Emmanuelle Charpentier, Helmholtz Center for Infection Research, Umeå University
Eric A. Hendrickson, University of Minnesota
Jennifer Doudna, MCB, UC Berkeley, HHMI
Luciano Marraffini, The Rockefeller University
Rudolf Jaenisch, MIT
Danwei Huangfu, Memorial Sloan Kettering Cancer Center
Stanley Qi, Stanford University
Philip Gregory, Sangamo BioSciences, Inc.
Ophir Shalem, Broad Institute, UC Berkeley
In this third annual event, leading experts in genomics and molecular biology will explore how an integrated view of genome-editing technologies promises to transform basic research as well as biomedical engineering. The event is free and open to the public, but space is limited.
Organizers:
Dirk Hockemeyer, MCB, UC Berkeley
Jennifer Doudna, MCB, UC Berkeley, HHMI
Confirmed speakers:
Emmanuelle Charpentier, Helmholtz Center for Infection Research, Umeå University
Eric A. Hendrickson, University of Minnesota
Jennifer Doudna, MCB, UC Berkeley, HHMI
Luciano Marraffini, The Rockefeller University
Rudolf Jaenisch, MIT
Danwei Huangfu, Memorial Sloan Kettering Cancer Center
Stanley Qi, Stanford University
Philip Gregory, Sangamo BioSciences, Inc.
Ophir Shalem, Broad Institute, UC Berkeley
In this third annual event, leading experts in genomics and molecular biology will explore how an integrated view of genome-editing technologies promises to transform basic research as well as biomedical engineering. The event is free and open to the public, but space is limited.
Investopedia website mentions Sangamo as potential threat to GILD HIV franchise
Sangamo BioSciences currently has two major gene therapy programs in HIV. Both could be highly disruptive and hopefully provide functional control of the HIV virus, i.e. one treatment and your immune system would be able to control the virus without further medications.
http://www.investopedia.com/stock-analysis/042015/ignore-these-risks-gilead-your-peril-gild.aspx
Gilead Sciences (NASDAQ: GILD) has enjoyed remarkable success in the past year thanks to the launch of two instant billion-dollar blockbuster hepatitis C therapies, however, even best-in-breed biotech stocks like Gilead Sciences still face risks that could leave investors in the lurch. Read on to learn which three risks concern me most, and how they could impact Gilead Sciences down the road.
No. 1: The launch of a disruptive HIV medicineGilead Sciences is the leading maker of HIV medicines, and its long-standing dominance in that indication has provided it with the financial firepower to orchestrate game-changing investments, including its $11.2 billion acquisition of the company responsible for inventing its top-selling hepatitis C drug Sovaldi.
Gilead Sciences' success in HIV hasn't come overnight. Instead, it's due to the launch of a string of remarkable therapies that have changed HIV from a death sentence into a chronic disease, and from the development of multi-drug combination therapies that have significantly improved dosing regimens. However, if Gilead Sciences gets too comfortable in its position at the top, innovative research into an HIV vaccine could pose a big threat.
Although we're miles away from the commercialization of vaccine-like treatments that could control HIV without the need to take multiple pills daily in perpetuity, there are programs percolating at small biotech companies that investors ought to be tracking.
One of those research programs is under way at Sangamo Biosciences.
In March, the FDA gave the go-ahead to begin phase 1 studies of a gene-editing approach that could someday offer HIV patients a functional cure. The trial is being conducted by the City of Hope medical center using Sangamo technology to collect blood from HIV patients and reengineer it to eliminate the CCR5 protein used by the virus to infect cells. Also, Sangamo is already using that gene-editing technology in another phase 2 trial of SB-728-T for HIV, which is due to report results this year.
Admittedly, Sangamo's research is still in the early stages, but it's a great example of some of the game-changing work that's occurring in HIV treatment, and for that reason, Gilead Sciences investors need to keep an eye on it.
http://www.investopedia.com/stock-analysis/042015/ignore-these-risks-gilead-your-peril-gild.aspx
Gilead Sciences (NASDAQ: GILD) has enjoyed remarkable success in the past year thanks to the launch of two instant billion-dollar blockbuster hepatitis C therapies, however, even best-in-breed biotech stocks like Gilead Sciences still face risks that could leave investors in the lurch. Read on to learn which three risks concern me most, and how they could impact Gilead Sciences down the road.
No. 1: The launch of a disruptive HIV medicineGilead Sciences is the leading maker of HIV medicines, and its long-standing dominance in that indication has provided it with the financial firepower to orchestrate game-changing investments, including its $11.2 billion acquisition of the company responsible for inventing its top-selling hepatitis C drug Sovaldi.
Gilead Sciences' success in HIV hasn't come overnight. Instead, it's due to the launch of a string of remarkable therapies that have changed HIV from a death sentence into a chronic disease, and from the development of multi-drug combination therapies that have significantly improved dosing regimens. However, if Gilead Sciences gets too comfortable in its position at the top, innovative research into an HIV vaccine could pose a big threat.
Although we're miles away from the commercialization of vaccine-like treatments that could control HIV without the need to take multiple pills daily in perpetuity, there are programs percolating at small biotech companies that investors ought to be tracking.
One of those research programs is under way at Sangamo Biosciences.
In March, the FDA gave the go-ahead to begin phase 1 studies of a gene-editing approach that could someday offer HIV patients a functional cure. The trial is being conducted by the City of Hope medical center using Sangamo technology to collect blood from HIV patients and reengineer it to eliminate the CCR5 protein used by the virus to infect cells. Also, Sangamo is already using that gene-editing technology in another phase 2 trial of SB-728-T for HIV, which is due to report results this year.
Admittedly, Sangamo's research is still in the early stages, but it's a great example of some of the game-changing work that's occurring in HIV treatment, and for that reason, Gilead Sciences investors need to keep an eye on it.
Sigma expands ZFN modified cell line product
Sangamo BioSciences receives royalties from the sale of ZFN modified cell lines.
http://finance.yahoo.com/news/qualyst-transporter-solutions-announces-license-121000015.html
Qualyst Transporter Solutions (QTS) is pleased to announce the completion of a non-exclusive license agreement with BioReliance®, Sigma-Aldrich Corporation's biologics and early-development services business under SAFC® Commercial (www.sigmaaldrich.com/safc) to allow QTS' proprietary B-CLEAR® Technology to be used in their new transporter knock-out products and in HepaRG™-related contract research services. These products aim to exclude key drug transporters, such as BSEP, OATPs, and P-gp, from the hepatic HepaRG cell line in order to assess their impact in drug disposition and drug interactions. The B-CLEAR® Technology was used in the development of these cell line products to ensure proper transporter function and it can also be used by end-users after purchasing the products.
Sigma has generated transporter knock-out cell lines using the CompoZr® Zinc Finger Nuclease (ZFN) technology in HepaRG™ cells which will allow researchers to discern effects from a single transporter. This has historically been a challenge as primary hepatocytes, and HepaRG cells, express all of the hepatic transporters, which means that isolating effects from a single transporter has been very difficult. QTS has been able to provide the means, through B-CLEAR®, to measure compound uptake and efflux into the bile pocket. In addition, the method can evaluate the overall integrated effect of transporters and metabolism together by measuring the intracellular concentration of a compound. The intracellular concentration is the result of the balance of all of these processes and the driving force for everything that happens inside a hepatocyte.
http://finance.yahoo.com/news/qualyst-transporter-solutions-announces-license-121000015.html
Qualyst Transporter Solutions (QTS) is pleased to announce the completion of a non-exclusive license agreement with BioReliance®, Sigma-Aldrich Corporation's biologics and early-development services business under SAFC® Commercial (www.sigmaaldrich.com/safc) to allow QTS' proprietary B-CLEAR® Technology to be used in their new transporter knock-out products and in HepaRG™-related contract research services. These products aim to exclude key drug transporters, such as BSEP, OATPs, and P-gp, from the hepatic HepaRG cell line in order to assess their impact in drug disposition and drug interactions. The B-CLEAR® Technology was used in the development of these cell line products to ensure proper transporter function and it can also be used by end-users after purchasing the products.
Sigma has generated transporter knock-out cell lines using the CompoZr® Zinc Finger Nuclease (ZFN) technology in HepaRG™ cells which will allow researchers to discern effects from a single transporter. This has historically been a challenge as primary hepatocytes, and HepaRG cells, express all of the hepatic transporters, which means that isolating effects from a single transporter has been very difficult. QTS has been able to provide the means, through B-CLEAR®, to measure compound uptake and efflux into the bile pocket. In addition, the method can evaluate the overall integrated effect of transporters and metabolism together by measuring the intracellular concentration of a compound. The intracellular concentration is the result of the balance of all of these processes and the driving force for everything that happens inside a hepatocyte.
TALENS used to edit potato
Dan Voytas used ZFN's prior to Sangamo BioSciences license of the technology to Dow Agro.
Now using CRISPR's. Article also points out that Dow Agro's ZFN modified corn plant is not subject to US Dept of AG approval , BUT IT IS NOT BEING SOLD YET. Sangamo will receive royalties from the sale of ZFN modified products.
http://www.technologyreview.com/news/536756/a-potato-made-with-gene-editing/
Dan Voytas is a plant geneticist at the University of Minnesota. But two days a week he stops studying the fundamentals of DNA engineering and heads to a nearby company called Cellectis Plant Sciences, where he applies them.
His newest creation, described in a plant journal this month, is a Ranger Russet potato that doesn’t accumulate sweet sugars at typical cold storage temperatures. That will let it last longer, and when it’s fried it won’t produce as much acrylamide, a suspected carcinogen.
What’s different about the potato is that it was bred with the help of gene editing, a new kind of technique for altering DNA that plant scientists say is going to be revolutionary for its simplicity and power. The technology could also be a way to engineer plants that avoid the stigma, and the regulations, associated with genetically modified organisms (GMOs).
In the case of the Ranger Russet, Voytas’s gene-editing technique, known as TALENs, left behind no trace other than a few deleted letters of DNA. The edit disabled a single gene that turns sucrose into glucose and fructose. Without it, Voytas thinks, the potatoes can be stored far longer without loss of quality.
The potato is a prototype of what plant scientists say is a rapidly arriving new generation of genetically modified plants. With gene editing, small companies think they can very quickly develop new crops for a fraction of the typical cost—even in species so far mostly untouched by biotechnology, like avocados, sorghum, and decorative flowers.
Most genetically modified crops that have been grown commercially so far incorporate genes from bacteria to make them produce insecticides or resist weed killers. Public opposition and regulatory requirements make these transgenic plants expensive to develop. That is why nearly all biotech plants are lucrative, big-acreage crops like soy, corn, and cotton and are sold by just a few large companies, like Monsanto and DuPont.
In August, the U.S. Department of Agriculture told Cellectis that unlike transgenic plants, its potato wouldn’t be regulated. That means instead of being grown in fenced-in test plots and generating folder upon folder of safety data, the Ranger Russet may go quickly to the market. Two years ago the agency reached a similar conclusion when it considered a DNA-edited corn plant developed by Dow AgroSciences, although it isn’t being sold yet.
Scientists say products like the potato are just the start for gene-editing techniques in plants. The same technologies are going to allow far more sophisticated engineering, including manipulation of photosynthesis to make plants grow faster and yield more food. “It’s an enormous opportunity, an unfathomable opportunity,” says Martin Spalding, a plant researcher at Iowa State University.
For now, the techniques are being used to modify plants in more modest ways. “The first wave of this technology is just removing a few base pairs,” says Yinong Yang, a professor of plant pathology at Penn State University, referring to the combinations of DNA letters—A, G, C, and T—that make up a genome. By “knocking out” just the right gene, as researchers did with the potato, it’s possible to give a plant a few valuable properties.
The next step, Yang says, will be to change the DNA letters of plant genes, swapping one plant’s version of a gene for that of another known to offer, say, resistance to disease. Yang says there is a blight-resistant form of rice that differs from commercial species by only a few DNA letters. “I could just change that over to resistance,” he says. “It’s like gene therapy in humans.” He says he’s negotiating a contract to produce the gene-edited rice now.
As for Voytas, this isn’t the first time he has set out to gene-edit plants. A decade ago he started a company called Phytodyne based on an earlier technology, called zinc finger nucleases, but it folded after Dow AgroSciences paid more than $50 million for exclusive rights to use that type of gene editing in plants.
Voytas teamed up with the French biotechnology company Cellectis in 2010 after it offered to install him as science chief of a new plant engineering division. But initial efforts ran into difficulty when another gene-editing system, meganucleases, proved challenging to work with and also got tied up by patent disputes.
Eventually, Voytas returned to the lab and coinvented a new way to edit genes, using specially engineered proteins called TALENs. That technology was used to make Cellectis’s potato, as well as a soybean with improved oil. Since then, Voytas and Cellectis have also worked with a newer technique, called CRISPR (see “Genome Surgery”).
Voytas says the potato took only about a year to create. “If you did it via breeding it would take five to 10 years,” he says.
Altogether, says Luc Mathis, CEO of Cellectis Plant Sciences, developing the potato cost a tenth of what it does to create and bring to market a transgenic plant, like corn or soy. “We will still need to generate some data, but it will not be a huge process,” says Mathis, who continues to meet with regulators to determine what steps remain before the potato can be sold.
Cellectis will move ahead with preliminary planting as soon as warm weather arrives in Minnesota. The first crops will determine whether the potatoes have the commercial benefits seen in greenhouse tests. “We need to check that we can store the potato in the cold,” says Mathis. “Once we have the commercial proof of concept, we can discuss with farmers what the interest level is.”
Kevin Folta, a professor of horticultural sciences at the University of Florida, says about 50 experts, including scientists and lawyers, met in Arizona earlier this year to discuss gene editing and how to orchestrate the industry’s approach to regulators in the United States and abroad. “Anyone who works in any kind of plant engineering is vigorously pursuing these technologies, especially with crops that have complex genomes or that you can’t breed easily,” he says. “There are lots of plants that need solutions.” He says gene editing will allow citrus trees to be modified in ways that would take 150 years with conventional breeding.
Folta says opponents of GMOs were not included in the planning meeting. “To invite people who view things nonscientifically would clog the discussion,” says. “There is no technology they are happy with.”
Now using CRISPR's. Article also points out that Dow Agro's ZFN modified corn plant is not subject to US Dept of AG approval , BUT IT IS NOT BEING SOLD YET. Sangamo will receive royalties from the sale of ZFN modified products.
http://www.technologyreview.com/news/536756/a-potato-made-with-gene-editing/
Dan Voytas is a plant geneticist at the University of Minnesota. But two days a week he stops studying the fundamentals of DNA engineering and heads to a nearby company called Cellectis Plant Sciences, where he applies them.
His newest creation, described in a plant journal this month, is a Ranger Russet potato that doesn’t accumulate sweet sugars at typical cold storage temperatures. That will let it last longer, and when it’s fried it won’t produce as much acrylamide, a suspected carcinogen.
What’s different about the potato is that it was bred with the help of gene editing, a new kind of technique for altering DNA that plant scientists say is going to be revolutionary for its simplicity and power. The technology could also be a way to engineer plants that avoid the stigma, and the regulations, associated with genetically modified organisms (GMOs).
In the case of the Ranger Russet, Voytas’s gene-editing technique, known as TALENs, left behind no trace other than a few deleted letters of DNA. The edit disabled a single gene that turns sucrose into glucose and fructose. Without it, Voytas thinks, the potatoes can be stored far longer without loss of quality.
The potato is a prototype of what plant scientists say is a rapidly arriving new generation of genetically modified plants. With gene editing, small companies think they can very quickly develop new crops for a fraction of the typical cost—even in species so far mostly untouched by biotechnology, like avocados, sorghum, and decorative flowers.
Most genetically modified crops that have been grown commercially so far incorporate genes from bacteria to make them produce insecticides or resist weed killers. Public opposition and regulatory requirements make these transgenic plants expensive to develop. That is why nearly all biotech plants are lucrative, big-acreage crops like soy, corn, and cotton and are sold by just a few large companies, like Monsanto and DuPont.
In August, the U.S. Department of Agriculture told Cellectis that unlike transgenic plants, its potato wouldn’t be regulated. That means instead of being grown in fenced-in test plots and generating folder upon folder of safety data, the Ranger Russet may go quickly to the market. Two years ago the agency reached a similar conclusion when it considered a DNA-edited corn plant developed by Dow AgroSciences, although it isn’t being sold yet.
Scientists say products like the potato are just the start for gene-editing techniques in plants. The same technologies are going to allow far more sophisticated engineering, including manipulation of photosynthesis to make plants grow faster and yield more food. “It’s an enormous opportunity, an unfathomable opportunity,” says Martin Spalding, a plant researcher at Iowa State University.
For now, the techniques are being used to modify plants in more modest ways. “The first wave of this technology is just removing a few base pairs,” says Yinong Yang, a professor of plant pathology at Penn State University, referring to the combinations of DNA letters—A, G, C, and T—that make up a genome. By “knocking out” just the right gene, as researchers did with the potato, it’s possible to give a plant a few valuable properties.
The next step, Yang says, will be to change the DNA letters of plant genes, swapping one plant’s version of a gene for that of another known to offer, say, resistance to disease. Yang says there is a blight-resistant form of rice that differs from commercial species by only a few DNA letters. “I could just change that over to resistance,” he says. “It’s like gene therapy in humans.” He says he’s negotiating a contract to produce the gene-edited rice now.
As for Voytas, this isn’t the first time he has set out to gene-edit plants. A decade ago he started a company called Phytodyne based on an earlier technology, called zinc finger nucleases, but it folded after Dow AgroSciences paid more than $50 million for exclusive rights to use that type of gene editing in plants.
Voytas teamed up with the French biotechnology company Cellectis in 2010 after it offered to install him as science chief of a new plant engineering division. But initial efforts ran into difficulty when another gene-editing system, meganucleases, proved challenging to work with and also got tied up by patent disputes.
Eventually, Voytas returned to the lab and coinvented a new way to edit genes, using specially engineered proteins called TALENs. That technology was used to make Cellectis’s potato, as well as a soybean with improved oil. Since then, Voytas and Cellectis have also worked with a newer technique, called CRISPR (see “Genome Surgery”).
Voytas says the potato took only about a year to create. “If you did it via breeding it would take five to 10 years,” he says.
Altogether, says Luc Mathis, CEO of Cellectis Plant Sciences, developing the potato cost a tenth of what it does to create and bring to market a transgenic plant, like corn or soy. “We will still need to generate some data, but it will not be a huge process,” says Mathis, who continues to meet with regulators to determine what steps remain before the potato can be sold.
Cellectis will move ahead with preliminary planting as soon as warm weather arrives in Minnesota. The first crops will determine whether the potatoes have the commercial benefits seen in greenhouse tests. “We need to check that we can store the potato in the cold,” says Mathis. “Once we have the commercial proof of concept, we can discuss with farmers what the interest level is.”
Kevin Folta, a professor of horticultural sciences at the University of Florida, says about 50 experts, including scientists and lawyers, met in Arizona earlier this year to discuss gene editing and how to orchestrate the industry’s approach to regulators in the United States and abroad. “Anyone who works in any kind of plant engineering is vigorously pursuing these technologies, especially with crops that have complex genomes or that you can’t breed easily,” he says. “There are lots of plants that need solutions.” He says gene editing will allow citrus trees to be modified in ways that would take 150 years with conventional breeding.
Folta says opponents of GMOs were not included in the planning meeting. “To invite people who view things nonscientifically would clog the discussion,” says. “There is no technology they are happy with.”
Friday, April 17, 2015
Pablo Tebas Upenn MD speaking Today at the Clinical Research Forum - Sangamo collaborator
Pablo Tebas the first author of the NEJM paper describing the Sangamo Biosciences sponsored work at UPENN titled " Gene Editiing of CCR5 in Autologous CD4 T-cells of persons affected with HIV" will speak today. The presentation is at the Clinical Research Forum.
The Clinical Research Forum was established in 1996 to provide a forum for discussions of the unique and complex challenges to clinical research among the senior clinical research leadership of leading academic health and science systems (AHASs). Over the past decade, it has convened these senior thought leaders in clinical research annually and has continued to provide a unique forum for discussion of common issues across the full spectrum of clinical and translational research.
There are slides posted online at http://www.clinicalresearchforum.org/vertical/Sites/%7B6D77AD78-8385-49EE-9E9C-158592F03CED%7D/uploads/Tebas_presentation.pdf
Slide 14 shows "Sustained Functional Control of Viral Load for More than One YEAR" !!!!!
Viral Load controlled more than 59 Weeks (less than 500 VL copies / mL)
Subject remains off ART !!!!
DURABLE FUNCTIONAL CONTROL ACHIEVED
Slide 20 shows "What we have learned"
It is DOABLE
The Size of the Reservoir Matters
The dose of the product (SB-728) matters
Mechanism ??
The Clinical Research Forum was established in 1996 to provide a forum for discussions of the unique and complex challenges to clinical research among the senior clinical research leadership of leading academic health and science systems (AHASs). Over the past decade, it has convened these senior thought leaders in clinical research annually and has continued to provide a unique forum for discussion of common issues across the full spectrum of clinical and translational research.
There are slides posted online at http://www.clinicalresearchforum.org/vertical/Sites/%7B6D77AD78-8385-49EE-9E9C-158592F03CED%7D/uploads/Tebas_presentation.pdf
Slide 14 shows "Sustained Functional Control of Viral Load for More than One YEAR" !!!!!
Viral Load controlled more than 59 Weeks (less than 500 VL copies / mL)
Subject remains off ART !!!!
DURABLE FUNCTIONAL CONTROL ACHIEVED
Slide 20 shows "What we have learned"
It is DOABLE
The Size of the Reservoir Matters
The dose of the product (SB-728) matters
Mechanism ??
Wednesday, April 15, 2015
CRISPER Wars: Sangamo gets positive mention
Its fantastic to see Sangamo Biosciences credited for having the most advanced gene editing treatment in the world !
Doudna, Berkeley Gain Expert’s Backing In CRISPR Patent Fight
Author: Alex Lash www.xcomomy.com
Jennifer Doudna is urging caution in the use of genetic manipulation. But she and her colleagues are also fighting for ownership of the scientific tools that make it possible.
Through Doudna’s employer, the University of California, Berkeley, the biochemist and her colleagues are forging ahead with a patent fight, bolstered with documents published Monday, over gene-editing technology known in shorthand as CRISPR/Cas9.
I wrote about the fight in December. It heated up when the U.S. Patent and Trademark Office in April 2014 awarded the first CRISPR-related patent to the Broad Institute and MIT for work led by the Broad’s Feng Zhang. The Berkeley group wants the PTO to reconsider that award, claiming that it had the invention first.
The PTO has not said whether it would re-open the case—a procedure that’s called “interference,” in patent-speak—but the Doudna camp has submitted thousands of pages of documentation to press its case. The documents were made available on the PTO website Monday.
A key element of the Berkeley group’s argument why it, and not Zhang’s group, deserve CRISPR/Cas9 ownership is a long written declaration from Dana Carroll of the University of Utah, who is considered a pioneer in the field of gene editing.
One of Carroll’s key arguments is that he and other scientists were able to use the descriptions of the Berkeley group—in a seminal paper published in Science in 2012 and then in the group’s patent application—to carry out their own CRISPR/Cas9 experiments in several kinds of cells, including eukaryotic cells—that is, from life forms more advanced than bacteria. “Not only do the applications provide this clear disclosure of the use of the compositions in eukaryotic cells, they also provide detailed descriptions of numerous steps that could be taken to apply the system to a eukaryotic cell environment,” Carroll wrote. (He also disclosed that he was paid for his work in support of the Berkeley patent.)
His argument rebuts a main point of the Broad patent: that the work of Zhang’s group made CRISPR-Cas9 editing a reality in eukaryotic cells and is therefore the invention when it comes to real-world uses, such as human therapeutics.
Well before CRISPR-Cas9, originally discovered as a bacterial defense mechanism 25 years ago, became a gene editing system, Carroll was working on earlier systems known as zinc finger nucleases. Sangamo Biosciences (NASDAQ: SMGO) of Richmond, CA, is the exclusive developer of zinc finger nucleases for human therapeutics, and the company now has a potential treatment for HIV infection in Phase 2. It’s the most advanced gene editing treatment in the world.
But proponents of CRISPR-Cas9 believe in its therapeutic potential, too. Doudna’s work has been licensed to a Berkeley, CA startup, Caribou Biosciences, which in turn has granted exclusive use of its technology in human therapeutics to Intellia Therapeutics, based in Cambridge, MA. Doudna’s co-inventor, Emmanuelle Charpentier, who has posts at institutes in Sweden and Germany, assigned her portion of the patent rights not to Caribou but to London-based Crispr Therapeutics, which recently established an R&D presence in Cambridge, MA.
The competing Boston group has thrown its weight behind Editas Medicine, also in Cambridge.
Nothing CRISPR-related has reached human clinical trials, but the use of CRISPR-Cas9 in basic research has spread across multiple organisms, plant and animal. Making sure CRISPR-Cas9 makes DNA cuts in the right places is a big challenge to overcome before it can be used in humans.
What’s more, fears that manipulation of the human germline—eggs, sperm, and embryos—is coming soon, not to treat disease but to improve people’s intelligence, looks, and other traits, have driven Doudna and several other prominent scientists—including Dana Carroll—to call for a moratorium on the practice. They published an open letter last month in Science. (Click here for my Q&A with Doudna about those fears, the moratorium, and the new research institute she runs on the UC Berkeley campus.)
If the PTO accepts the Berkeley group’s challenge to the Broad patent—no sure thing, despite the truckload of new material the group has provided—the interference hearing could last a long time as a PTO board of appeals would consider each party’s claims side by side.
Had the patent applications been filed just a little later, however, none of this would be possible. Because of the 2011 U.S. patent reform law, the system is no longer based on who invented first, and interference hearings will no longer be held. All this means that a patent fight that could determine ownership of what could be a crucial 21st century technology is one of the last being fought under anachronistic 20th century rules.
Doudna, Berkeley Gain Expert’s Backing In CRISPR Patent Fight
Author: Alex Lash www.xcomomy.com
Jennifer Doudna is urging caution in the use of genetic manipulation. But she and her colleagues are also fighting for ownership of the scientific tools that make it possible.
Through Doudna’s employer, the University of California, Berkeley, the biochemist and her colleagues are forging ahead with a patent fight, bolstered with documents published Monday, over gene-editing technology known in shorthand as CRISPR/Cas9.
I wrote about the fight in December. It heated up when the U.S. Patent and Trademark Office in April 2014 awarded the first CRISPR-related patent to the Broad Institute and MIT for work led by the Broad’s Feng Zhang. The Berkeley group wants the PTO to reconsider that award, claiming that it had the invention first.
The PTO has not said whether it would re-open the case—a procedure that’s called “interference,” in patent-speak—but the Doudna camp has submitted thousands of pages of documentation to press its case. The documents were made available on the PTO website Monday.
A key element of the Berkeley group’s argument why it, and not Zhang’s group, deserve CRISPR/Cas9 ownership is a long written declaration from Dana Carroll of the University of Utah, who is considered a pioneer in the field of gene editing.
One of Carroll’s key arguments is that he and other scientists were able to use the descriptions of the Berkeley group—in a seminal paper published in Science in 2012 and then in the group’s patent application—to carry out their own CRISPR/Cas9 experiments in several kinds of cells, including eukaryotic cells—that is, from life forms more advanced than bacteria. “Not only do the applications provide this clear disclosure of the use of the compositions in eukaryotic cells, they also provide detailed descriptions of numerous steps that could be taken to apply the system to a eukaryotic cell environment,” Carroll wrote. (He also disclosed that he was paid for his work in support of the Berkeley patent.)
His argument rebuts a main point of the Broad patent: that the work of Zhang’s group made CRISPR-Cas9 editing a reality in eukaryotic cells and is therefore the invention when it comes to real-world uses, such as human therapeutics.
Well before CRISPR-Cas9, originally discovered as a bacterial defense mechanism 25 years ago, became a gene editing system, Carroll was working on earlier systems known as zinc finger nucleases. Sangamo Biosciences (NASDAQ: SMGO) of Richmond, CA, is the exclusive developer of zinc finger nucleases for human therapeutics, and the company now has a potential treatment for HIV infection in Phase 2. It’s the most advanced gene editing treatment in the world.
But proponents of CRISPR-Cas9 believe in its therapeutic potential, too. Doudna’s work has been licensed to a Berkeley, CA startup, Caribou Biosciences, which in turn has granted exclusive use of its technology in human therapeutics to Intellia Therapeutics, based in Cambridge, MA. Doudna’s co-inventor, Emmanuelle Charpentier, who has posts at institutes in Sweden and Germany, assigned her portion of the patent rights not to Caribou but to London-based Crispr Therapeutics, which recently established an R&D presence in Cambridge, MA.
The competing Boston group has thrown its weight behind Editas Medicine, also in Cambridge.
Nothing CRISPR-related has reached human clinical trials, but the use of CRISPR-Cas9 in basic research has spread across multiple organisms, plant and animal. Making sure CRISPR-Cas9 makes DNA cuts in the right places is a big challenge to overcome before it can be used in humans.
What’s more, fears that manipulation of the human germline—eggs, sperm, and embryos—is coming soon, not to treat disease but to improve people’s intelligence, looks, and other traits, have driven Doudna and several other prominent scientists—including Dana Carroll—to call for a moratorium on the practice. They published an open letter last month in Science. (Click here for my Q&A with Doudna about those fears, the moratorium, and the new research institute she runs on the UC Berkeley campus.)
If the PTO accepts the Berkeley group’s challenge to the Broad patent—no sure thing, despite the truckload of new material the group has provided—the interference hearing could last a long time as a PTO board of appeals would consider each party’s claims side by side.
Had the patent applications been filed just a little later, however, none of this would be possible. Because of the 2011 U.S. patent reform law, the system is no longer based on who invented first, and interference hearings will no longer be held. All this means that a patent fight that could determine ownership of what could be a crucial 21st century technology is one of the last being fought under anachronistic 20th century rules.
Sangamo BioSciences gets mention as competitor in Bayer expansion article
Of course we were told the hemophilia program would happen much faster than has actually transpired. As Ed Lanphier says, " in the next 12, 18, 24 months". Who can wait that long while management continues to exercise their very generous options?
The 80,000-square-foot structure on the southern edge of Bayer’s East Bay campus actually is part of a long-range strategy by the subgroup of Germany-based drug giant Bayer AG. That plan has focused on upgrading facilities for research, development, manufacturing and testing so-called Factor VIII replacement products that help hemophilia patients control the bleeding disorder.
Factor VIII is a blood-clotting protein, and about one in every 5,000 boys is born with a defect in a gene that encodes the protein, causing hemophilia A.
Bayer will break ground on the new testing facility this month, said Joerg Heidrich, Bayer’s senior vice president for biotech product supply and site head for Bayer in Berkeley. Construction is expected to be completed in 2017.
The testing building, which will replace an older facility, won’t mean more Bayer jobs at the company's Berkeley campus, but there will be an undisclosed number of construction-related jobs.
What's more, company officials stressed, the project shows Bayer’s commitment to the Bay Area and to developing new products for hemophilia A patients and other people with blood disorders.
“It’s a great story for the Bay Area,” Heidrich said. “The research is done in Mission Bay and the development and manufacturing are done on the Berkeley site.”
“It’s a great story for the Bay Area,” Heidrich said. “The research is done in Mission Bay and the development and manufacturing are done on the Berkeley site.”
In all, Bayer has invested more than a half-billion dollars into its Berkeley facility over the past decade, Heidrich said, including a $100 million manufacturing expansion started in 2009.
Bayer already makes and tests Kogenate FS, a Factor VIII product approved in 2000, at its Berkeley facility. Kogenate was the company's second-best-selling product last year at about $1.1 billion, following the blood thinner Xarelto.
Kogenate FS costs about $1,000 per dose.
But Bayer's two-decade-old Kogenate franchise is getting squeezed by Factor VIII treatments that allow patients to dose less often. What's more, experimental gene therapies, which could fix the genetic defect with a single treatment, are progressing toward in-human studies.
Those competitors include Richmond's Sangamo BioSciences Inc. (NASDAQ: SGMO) and partner Shire plc.
As a result, Bayer has been busy pushing its own next-generation Factor VIII products through the clinical trial process.
"This is really the beginning of a much broader story that starts in factor replacement," said Dr. David Weinreich, Bayer's head of global development, specialty medicine.
Bayer in December filed for Food and Drug Administration approval of its latest experimental hemophila drug, called Kovaltry. A long-acting treatment that could allow hemophilia A patients to be dosed once a week, rather than once every four days or so, also has hurdled a late-stage trial.
Bayer's Berkeley site has made batches of Kovaltry as well as damoctocog — the experimental long-acting drug — for clinical trials.
If Kovaltry is approved by the FDA, which could occur next year, Bayer will make and test commercial batches of it and Kogenate FS in Berkeley. Those products could be shipped to some 80 other countries.
"It's simply a brick-and-mortar thing," Weinreich said about the new testing facility. "When you fill it up, you have to put more bricks and mortar into it."
First Quarter Conference Call 4/22/15
RICHMOND, Calif., April 15, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO) today announced that the company will release its first quarter 2015 financial results after the market closes on Wednesday, April 22, 2015. The press release will be followed by a conference call at 5:00 p.m. ET, which will be open to the public via telephone and webcast. During the conference call, the company will review the quarter's financial results and discuss other business matters.
The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 20895051. Participants may access the live webcast via a link on the Sangamo BioSciences website in the Investors section under Events and Presentations. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on April 22, 2015 to 11:59 p.m. ET on April 29, 2015. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 20895051.
Monday, April 13, 2015
Sangamo Named one of Jefferies top ten buyout targets
Buyout Targets
Jefferies recommends buying the following 10 potential M&A targets:1. Arena Pharmaceuticals, Inc. ARNA 0.22%
2. Lexicon Pharmaceuticals, Inc. LXRX 0.56%
3. Seattle Genetics, Inc. SGEN 2.18%
4. Sangamo Biosciences, Inc. SGMO 0.97%
5. DHT Holdings Inc DHT 2.02%
6. BioScrip Inc BIOS 2.96%
7. OraSure Technologies, Inc. OSUR 0.15%
8. Sonus Networks, Inc. SONS 0.26%
9. NuVasive, Inc. NUVA 1.12%
10. Allscripts Healthcare Solutions Inc MDRX 0.65%
Sunday, April 12, 2015
Dr. Steven Deeks giving keynote,"Beyond Antiretroviral Therapy: The Future of HIV Therapeutics"
Noted Sangamo BioSciences (SGMO) collaborator Dr. Steven Deeks will present the keynote opening address, "Beyond Antiretroviral Therapy: The Future of HIV Therapeutics" on April 17,2015.
The Andy I. Choi Mentoring Program of the UCSF-GIVI Center for AIDS Research will offer Bay Area investigators a glimpse into the future of HIV research with a half-day symposium by CFAR Mentees on Friday, April 17, 2015 at the J. David Gladstone Institutes' Mahley Auditorium. Dr. Jonathan Fuchs, Dr. Monica Gandhi and CFAR Co-Directors Drs. Paul Volberding and Warner Greene, will provide a venue for promoting the work of the future leaders in HIV research. The speaker lineup will feature a group of highly respected HIV/AIDS investigators from our Mentoring Program. Research presentations will span basic, clinical, epidemiologic, preventive and translational aspects of HIV biology.
http://cfar.ucsf.edu/cfar?page=education-mentor-symposium-2015
The Andy I. Choi Mentoring Program of the UCSF-GIVI Center for AIDS Research will offer Bay Area investigators a glimpse into the future of HIV research with a half-day symposium by CFAR Mentees on Friday, April 17, 2015 at the J. David Gladstone Institutes' Mahley Auditorium. Dr. Jonathan Fuchs, Dr. Monica Gandhi and CFAR Co-Directors Drs. Paul Volberding and Warner Greene, will provide a venue for promoting the work of the future leaders in HIV research. The speaker lineup will feature a group of highly respected HIV/AIDS investigators from our Mentoring Program. Research presentations will span basic, clinical, epidemiologic, preventive and translational aspects of HIV biology.
http://cfar.ucsf.edu/cfar?page=education-mentor-symposium-2015
Saturday, April 11, 2015
Sangamo 22 days to cover short position
With last weeks low volume and substantial short position it would take 22 days of trading to cover the short position in Sangamo BioSciences (SGMO).
Shares short as of 3/31/15 - 8,285,803
Avg daily volume last week - 378,789
DATE SHORT INTEREST
3/31/15 8,285,803
3/13/15 8,441,291
2/27/15 8,939,000
2/13/15 9,268,065
1/30/15 9,082,814
1/15/15 9,387,913
SYMBOL % of SHARES OUTSTANDING SHORT
SGMO 12%
JUNO 5%
BLUE 7%
QURE 1%
CLLS 1%
Shares short as of 3/31/15 - 8,285,803
Avg daily volume last week - 378,789
DATE SHORT INTEREST
3/31/15 8,285,803
3/13/15 8,441,291
2/27/15 8,939,000
2/13/15 9,268,065
1/30/15 9,082,814
1/15/15 9,387,913
SYMBOL % of SHARES OUTSTANDING SHORT
SGMO 12%
JUNO 5%
BLUE 7%
QURE 1%
CLLS 1%
Friday, April 10, 2015
Gene Therapy company UniQure prices follow-on offering at $29.50
Sangamo competitor Uniqure announced the pricing of its follow-on public offering of 3,000,000 ordinary shares at price to the public of $29.50 per ordinary share. After deducting the underwriting discounts and other estimated offering expenses payable by uniQure, the net proceeds of the follow-on public offering are expected to be approximately $82.4 million (EURO76.5 million). In addition, uniQure has granted the underwriters a 30-day option to purchase up to an additional 450,000 ordinary shares from uniQure at the public offering price, less underwriting discounts. The offering is expected to close on or about April 15, 2015.
Leerink Partners LLC, Cowen and Company, LLC, and Piper Jaffray & Co. are acting as the joint book-running managers for the offering. Oppenheimer & Co. Inc. and H.C. Wainwright & Co., LLC, are acting as co-managers.
Tuesday, April 7, 2015
Sigma-Aldrich launches new product, EX-CELL, includes CHOZEN cells
Sangamo receives royalties from Sigma-Aldrich on the sale or licensing of ZFN modified cell lines.
Sigma-Aldrich Corporation (NASDAQ: SIAL) today announced that SAFC® Commercial (www.sigma-aldrich.com/safc), the Company’s business unit providing products and services for use in regulated pharmaceutical and biopharmaceutical applications, has launched the EX-CELL® Advanced™ product line. Designed to address the needs of an evolving industry where speed to market is paramount, the new product line provides for increased performance, streamlines regulatory compliance, and offers the supply chain security needed in today’s biopharmaceutical environment.
The first EX-CELL Advanced product is the high-performing batch media system developed for a range of widely used industrial CHO cell lines, including SAFC’s proprietary CHOZN® cell line. During rigorous testing, the EX-CELL Advanced CHO Fed-batch System outperformed other commercially available equivalents by displaying significantly higher titers. Robust scale up and effortless adaptation offers those in biopharmaceutical development the opportunity to get their process up and running quickly and efficiently. Protein quality, another vital consideration in the development process, remains uncompromised
Sigma-Aldrich Corporation (NASDAQ: SIAL) today announced that SAFC® Commercial (www.sigma-aldrich.com/safc), the Company’s business unit providing products and services for use in regulated pharmaceutical and biopharmaceutical applications, has launched the EX-CELL® Advanced™ product line. Designed to address the needs of an evolving industry where speed to market is paramount, the new product line provides for increased performance, streamlines regulatory compliance, and offers the supply chain security needed in today’s biopharmaceutical environment.
The first EX-CELL Advanced product is the high-performing batch media system developed for a range of widely used industrial CHO cell lines, including SAFC’s proprietary CHOZN® cell line. During rigorous testing, the EX-CELL Advanced CHO Fed-batch System outperformed other commercially available equivalents by displaying significantly higher titers. Robust scale up and effortless adaptation offers those in biopharmaceutical development the opportunity to get their process up and running quickly and efficiently. Protein quality, another vital consideration in the development process, remains uncompromised
Monday, April 6, 2015
Uniqure to collaborate with Bristol-Myers Squib - Cardiovascular Diseases (QURE)
QURE up $10 per share in pre-market trading on this deal.
UP TO 10 TARGETS 50 mil upfront payments
Bristol-Myers Squibb and uniQure Enter into Exclusive Strategic Collaboration to Develop Gene Therapies for Cardiovascular Diseases.
The collaboration includes uniQure’s proprietary gene therapy program for congestive heart failure that is intended to restore the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction. Beyond cardiovascular diseases, the agreement also includes the potential for target-exclusive collaboration in other disease areas. In total, the companies may collaborate on ten targets, including S100A1.
uniQure will be eligible to receive research, development and regulatory milestone payments, including up to $254 million for the lead S100A1 therapeutic and up to $217 million for each other gene therapy product potentially developed under the collaboration. uniQure is also eligible to receive net sales based milestone payments and tiered single to double-digit royalties on product sales
UP TO 10 TARGETS 50 mil upfront payments
Bristol-Myers Squibb and uniQure Enter into Exclusive Strategic Collaboration to Develop Gene Therapies for Cardiovascular Diseases.
The collaboration includes uniQure’s proprietary gene therapy program for congestive heart failure that is intended to restore the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction. Beyond cardiovascular diseases, the agreement also includes the potential for target-exclusive collaboration in other disease areas. In total, the companies may collaborate on ten targets, including S100A1.
uniQure will lead discovery efforts and be responsible for manufacturing of clinical and commercial supplies using its vector technologies and its industrial, proprietary insect-cell based manufacturing platform. Bristol-Myers Squibb will lead development and regulatory activities across all programs and be responsible for all research and development costs. Bristol-Myers Squibb will be solely responsible for commercialization of all products from the collaboration.
Under the terms of the agreement, Bristol-Myers Squibb will make near-term payments of approximately $100 million, including an upfront payment of $50 million to be made at the closing of the transaction, a $15 million payment for the selection of three collaboration targets, in addition to S100A1, to be made within three months of the closing and an initial equity investment in uniQure for a number of shares that will equal 4.9% of the total number of shares outstanding following such issuance, at a purchase price of $33.84 per share, or at least $32 million in total. Bristol-Myers-Squibb will acquire an additional 5.0% ownership before December 31, 2015, at a 10% premium, and will be granted two warrants to acquire up to an additional 10% equity interest, at a premium, based on additional targets being introduced into the collaboration. The parties have also agreed to enter into a supply contract, under which uniQure will undertake manufacturing of all gene therapy products under the collaboration.uniQure will be eligible to receive research, development and regulatory milestone payments, including up to $254 million for the lead S100A1 therapeutic and up to $217 million for each other gene therapy product potentially developed under the collaboration. uniQure is also eligible to receive net sales based milestone payments and tiered single to double-digit royalties on product sales
Friday, April 3, 2015
Hockemeyer lab, UC-Berkeley, using zinc fingers to understand how the enzyme telomerase is transcriptionally regulated and how telomere shortening impacts tumor formation
Noted Sangamo collaborator Dr. Dirk Hockemeyer is adding staff to his lab.
Open Apr 2, 2015 through May 11, 2015
Description
The Hockemeyer lab in the Molecular Cell Biology Department at UC Berkeley seeks a Jr. Specialist. The goal of the Hockemeyer lab is to shed light on the key functions of telomeres and telomerase in tissue homeostasis, tumorigenesis and aging in human stem cell. To investigate these processes we developed the use of site-specific nucleases, specifically Zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), to genetically engineer human stem cells. Using these tools we seek to understand how the enzyme telomerase is transcriptionally regulated and how telomere shortening impacts tumor formation.
Responsibilities:
Additional Required Qualifications: The successful candidate will have at least 6 months of laboratory employment experience involving the techniques listed in the "Responsibilities" section. Excellent communication skills and careful attention to detail are also required.
Preferred Qualifications: Experience with animal care, genotyping, histology and immunohistochemistry
Open Apr 2, 2015 through May 11, 2015
Description
The Hockemeyer lab in the Molecular Cell Biology Department at UC Berkeley seeks a Jr. Specialist. The goal of the Hockemeyer lab is to shed light on the key functions of telomeres and telomerase in tissue homeostasis, tumorigenesis and aging in human stem cell. To investigate these processes we developed the use of site-specific nucleases, specifically Zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), to genetically engineer human stem cells. Using these tools we seek to understand how the enzyme telomerase is transcriptionally regulated and how telomere shortening impacts tumor formation.
Responsibilities:
- Animal colony maintenance and genotyping: including, animal breeding, weaning, culling, and genotyping by tissue biopsy and assay by PCR with a standard thermocycler. Additionally, regular record keeping of all animals in the colony, as well as daily recording the body weight and drinking volume of animals involved in behavioral studies, and providing appropriate water volumes to animals on restricted water diets.
- Histology and immunohistochemistry: including animal perfusion, tissue extraction, tissue slicing with a freezing microtome, immunostaining and processing and mounting of fixed and stained tissue slices.
- DNA amplification and purification: including bacterial retransformation, amplification, purification by standard Qiagen kit, and quantification with a spectrophotometer.
- Lab management duties will include ordering, filling reagent requests, coordinating food supplies, and stock keeping. Supervising compliances with all safety and animal regulations, radiation safety.
Additional Required Qualifications: The successful candidate will have at least 6 months of laboratory employment experience involving the techniques listed in the "Responsibilities" section. Excellent communication skills and careful attention to detail are also required.
Preferred Qualifications: Experience with animal care, genotyping, histology and immunohistochemistry
Thursday, April 2, 2015
Replay of E. Wolffe and E. Lanphier presentations at the Regen Med Investor Day
Elizabeth Wolffe, Ph.D., Sangamo's vice president, corporate communications, presented an overview of the Company's ZFP Therapeutic® programs at 1:55pm ET on Wednesday , March 25, 2015 , at the 3rd Annual Regen Med Investor Day
Here is her presentation:
https://www.youtube.com/watch?v=2uTUDyVwOVM
Edward Lanphier, Chairman, Alliance for Regenerative Medicine; President & CEO, Sangamo BioSciences. gave the welcoming remarks:
https://www.youtube.com/watch?v=2wQ9kyKEI54
Here is her presentation:
https://www.youtube.com/watch?v=2uTUDyVwOVM
Edward Lanphier, Chairman, Alliance for Regenerative Medicine; President & CEO, Sangamo BioSciences. gave the welcoming remarks:
https://www.youtube.com/watch?v=2wQ9kyKEI54
Sangamo Biosciences sponsored Keystone Symposia starts April 26th
Mechanisms of HIV Persistence: Implications for a Cure
Scientific Organizers: Olivier Lambotte, Steven G. Deeks and Guido Silvestri
April 26—May 1, 2015
Boston Park Plaza, Boston, Massachusetts, USA
Sponsored by Bristol-Myers Squibb Company, Gilead Sciences, Inc., Merck & Co., Inc.
and Sangamo BioSciences, Inc. Part of the Keystone Symposia Global Health Series,
supported by the Bill & Melinda Gates Foundation.
Steven G. Deeks, UCSF Medical School, provided advice for the Sangamo HIV CCR5 trials.
Paula M. Cannon, University of Southern California, Keck School of Medicine, USA
Targeted Nuclease Approaches for a Functional Cure (Sangamo Investigator)
Joumana Zeidan, Case Western Reserve University, USA
Short Talk: Decay of the HIV Reservoir Post Autologous Transfer of ZFN CCR5 Modified CD4 T Cells (SB-728-T) Correlates with Generation of a T Memory Stem Cell-Like Population and Enhanced HIV-Specific CD8 T Cell Polyfunctionality (Sangamo Investigator)
Wednesday, April 1, 2015
New Job Posting at Sangamo 3/31/15
There has been an unprecedented increase in the frequency of job opportunities at Sangamo in the past few months. This is the new posting:
We are seeking a Ph.D. level scientist to join our Technology group. The successful candidate will work with the group to engineer DNA binding proteins with unsurpassed biological specificity, and will develop new methods for characterizing and improving performance. The candidate will also extend Sangamo’s DNA-binding protein technology into novel applications. The position requires adaptability, creativity, and a capacity for constant innovation in pursuit of company goals. The environment is exciting and fast-paced, and offers the opportunity to work and publish at the cutting edge of genome editing research.
Requirements:
- Ph.D. with demonstrated success using protein engineering methods
- Excellent data analysis and problem solving skills
- Facility with general molecular biology techniques
- Familiarity with next-generation DNA sequencing methods and data
- Strong communication and presentation skills
- An ability to work in a team-oriented, multidisciplinary environment
Additional attractive qualities include:
- Programming skills
- Proficiency with the design and screening of combinatorial libraries
- Experience with the design and characterization of novel DNA-binding proteins
- Facility with liquid-handling robots such as the BioMek FX workstation
- Cell culture experience, including transgene delivery and analysis
The successful candidate will enjoy a competitive base salary and the opportunity to participate in incentive compensation programs, including bonus and stock option plans. Sangamo offers a comprehensive benefits program, including: medical, dental and vision care; paid vacation and holiday time; access to a voluntary 401(k) and Employee Stock Purchase Plan. Sangamo is an equal opportunity employer.
Please send your resume/CV and a cover letter that specifically addresses this job posting as email attachments to: jobs@sangamo.com
Scientist - Technology
Job Summary:We are seeking a Ph.D. level scientist to join our Technology group. The successful candidate will work with the group to engineer DNA binding proteins with unsurpassed biological specificity, and will develop new methods for characterizing and improving performance. The candidate will also extend Sangamo’s DNA-binding protein technology into novel applications. The position requires adaptability, creativity, and a capacity for constant innovation in pursuit of company goals. The environment is exciting and fast-paced, and offers the opportunity to work and publish at the cutting edge of genome editing research.
Requirements:
- Ph.D. with demonstrated success using protein engineering methods
- Excellent data analysis and problem solving skills
- Facility with general molecular biology techniques
- Familiarity with next-generation DNA sequencing methods and data
- Strong communication and presentation skills
- An ability to work in a team-oriented, multidisciplinary environment
Additional attractive qualities include:
- Programming skills
- Proficiency with the design and screening of combinatorial libraries
- Experience with the design and characterization of novel DNA-binding proteins
- Facility with liquid-handling robots such as the BioMek FX workstation
- Cell culture experience, including transgene delivery and analysis
The successful candidate will enjoy a competitive base salary and the opportunity to participate in incentive compensation programs, including bonus and stock option plans. Sangamo offers a comprehensive benefits program, including: medical, dental and vision care; paid vacation and holiday time; access to a voluntary 401(k) and Employee Stock Purchase Plan. Sangamo is an equal opportunity employer.
Please send your resume/CV and a cover letter that specifically addresses this job posting as email attachments to: jobs@sangamo.com
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