Free 2013 Report: New HIV, HCV and TB Drugs, Diagnostics, and Vaccines in the Research Pipeline

HIV i-Base/Treatment Action Group 2013 Pipeline Report
calls on leaders to get the best HIV, hepatitis C virus (HCV), and tuberculosis drugs, diagnostics, and vaccines to the most people as quickly as possible
– Survey shows HIV pipeline healthy, HCV drug development surging,
while TB research moves forward much too slowly –

– Faster research, approval, and access demanded –

KUALA LUMPUR, Malaysia, 30 June 2013 – HIV i-Base and Treatment Action Group (TAG) called on global and national leaders, research sponsors, and regulatory authorities to work together to make the best HIV, HCV, and TB drugs, diagnostics, and vaccines accessible as fast as possible, according to a report released today at the 7th International AIDS Society (IAS) Conference on HIV Pathogenesis, Treatment and Prevention in Kuala Lumpur, Malaysia.

In the 2013 Pipeline Report, Polly Clayden of HIV i-Base (UK) and Mark Harrington of TAG (USA), set a seven-part agenda to speed the best medical products to all who need them everywhere. They call for research funders to continue investing in better products and to address global needs up front in their development. They demand that regulators expedite review of new medications in developing countries and close approval gaps between rich and poor countries and adult and pediatric indications. World Health Organization (WHO) and national guidelines groups must continue streamlining and simplifying treatment guidelines, they stress. Clayden and Harrington urge authorities in developing and developed countries to speed the rational use of the best possible drug combinations regardless of patent status or manufacturer and to expedite the transition to high-quality first-line generic products when preferred drugs go off patent.

Noting that the pharmaceutical industry has made billions from successful HIV therapies in the past two decades, Clayden and Harrington report that in the last decade alone, 47 new drugs and combinations were studied in phases II and III. An impressive 34 percent (16/49) were approved by the U.S. Food and Drug Administration (FDA), with 6.4 percent (3) awaiting approval and 21 percent (10) moving forward in phases II or III. “To get the best drugs to the most people as quickly as possible, global and national health leaders, regulators, and research sponsors must take concerted action to speed up the approval of pediatric, generic, and high-quality cross-sponsor fixed-dose combinations—as soon as they are available in rich countries—in those where they are needed most,” commented Clayden and Harrington.

Themes of the 2013 “Antiretroviral Pipeline” by Simon Collins (HIV i-Base) and Tim Horn (TAG) include the continuing wave of innovations bringing broader, and in some cases better, treatment options for people with HIV. They note “the possible conflicts these innovations will encounter due to global economic austerity.” Collins and Horn underscore “the potential for combining generic antiretrovirals as they move off-patent in many developed countries with innovator compounds to produce synergistic, often cross-sponsor, combinations and fixed-dose combinations (FDCs) that could offer people with HIV the best of the new and the old while saving cash-strapped health systems billions of dollars.”

Noting the current decade’s coming wave of patent expiries on first-line HIV drugs such as efavirenz and tenofovir, Collins and Horn note that, “savings from generics are essential if we are to retain public health services for those who remain uninsured or underinsured.” They call for generic manufacturers and governments to seize the opportunity posed by the coming wave of patent expiries to offer optimal combinations at prices much lower than for innovator compounds. It will be critical for rich and poor countries, to reinvest the savings generated by sensible use of generic-containing antiretroviral combinations into massively expanded HIV prevention and treatment programs to end HIV transmission and progression to AIDS and death and achieve an “AIDS-free generation” for everyone.
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In back-to-back chapters on pediatric HIV drugs and HIV treatment optimization, Polly Clayden calls for continued streamlining and rationalization of adult and pediatric HIV formulations and notes innovative partnerships such as those recently supported by the Drugs for Neglected Diseases Initiative (DNDi) and UNITAID to expedite production of optimized regimens for children. Clayden stresses the importance of developing generic combinations with the best target product profiles. “It’s time to move on from merely providing the world’s poor people with the cheapest medicines and time to give them the best – safest and most effective – treatments possible, and make them affordable” says Clayden.

Richard Jefferys (TAG), in his broad survey of research developments on preventive technologies, research toward a cure, and immune-based and gene therapies, calls for intensified basic and translational science on potential HIV vaccines and continued research on antiretroviral (ART)-based pre- and postexposure prophylaxis (PrEP, PEP). He stresses the need for immune-based therapy research to raise CD4 T cell counts among immunologic nonresponders to HIV therapy, and new research to address ongoing residual dysfunction of the immune system that persists in people on successful ART. Finally, Jefferys emphasizes further investigation of the role of very early treatment with potent ART combinations in infants and adults infected with HIV; better lab measurements to quantify the cells and tissue sources of the latent HIV-1-infected cell reservoir, the target for HIV cure research; and exploration of the potential contribution of therapeutic vaccine approaches as part of combination curative therapy.

In the 2013 “HCV Treatment Pipeline,” Tracy Swan (TAG) notes that the “confluence of a robust HCV drug pipeline, shortened regimens, and [shorter] posttreatment follow-up are extraordinary. The new FDA breakthrough therapy designation may speed things up as well. By the end of 2014, [new HCV drugs] from four different classes and fixed-dose combinations (FDCs) are likely to be approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), offering the potential for off-label mixing and matching.” An impressive 26 new HCV drugs are being studied in phases II/III in at least 28 interferon-free regimens, which are bringing the potential of faster, all-oral HCV cures rapidly toward approval for the world’s 185 million people living with HCV.

Swan notes, however, that not all optimal combinations are being studied, with some sponsors preferring combinations of their own proprietary compounds, while many sponsors take too long to study their new drugs in people coinfected with HIV and HCV, and those with cirrhosis.

In her companion chapter, “Low- and Middle-Income Countries Defuse Hepatitis C, the ‘Viral Time Bomb,’” Karyn Kaplan (TAG) describes how a worldwide movement is forming to ensure that when new all-oral HCV cures are approved, that governments, health systems, and providers will be ready for them. Kaplan points to recent progress instigated by HCV activists in countries such as Egypt, Georgia, Thailand, and Ukraine.

In three chapters on tuberculosis (TB) diagnostics, treatment, and vaccine research, Colleen Daniels, Erica Lessem, and Mike Frick (TAG) find that lack of investment and political will make the TB pipeline the most anemic covered in the 2013 Pipeline Report.

Globally, one-third of active TB cases are never diagnosed, reported, or treated, meaning that 3 million people are walking around with undiagnosed disease, in danger of progression, death, and onward transmission.

Some progress has been made in rolling out new DNA tests, which can detect TB more rapidly. The tests still require high-tech laboratory equipment, electricity, and trained personnel though, meaning they are far from the majority of TB patients and those at risk. Manufacturers are trying out cheaper forms of DNA TB diagnostic testing with as yet little published data, and faster ways to detect TB mutations, which are associated with resistance to TB medications.

TB treatment is slowly moving forward following the first FDA approval of a new drug from a new class in over 40 years, and innovative combination TB studies under way for both drug-sensitive and drug-resistant disease. Most countries are not ready to review, approve, distribute, and assure rational use of new TB drugs and combinations, however, reinforcing HIV i-Base and TAG’s call for high-burden countries to modernize their regulatory research oversight and approval programs.

Research on TB vaccines remains slow and underfunded but there are some glimmers of hope, particularly on the basic science and early clinical front. Much more investment is needed in TB research and development overall, and in TB program scale-up to ensure access to the best diagnostics tests and preventive and curative therapies.
 

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The HIV i-Base/Treatment Action Group 2013 Pipeline Report: HIV, Hepatitis C Virus (HCV), and Tuberculosis (TB) Drugs, Diagnostics, Vaccines, Preventive Technologies, Research Toward a Cure, and Immune-Based and Gene Therapies in Development by Polly Clayden, Simon Collins, Colleen Daniels, Mike Frick, Mark Harrington, Tim Horn, Richard Jefferys, Karyn Kaplan, Erica Lessem, and Tracy Swan, edited by Andrea Benzacar, Tim Horn, and Scott Morgan, is online at:

            http://www.treatmentactiongroup.org/pipeline-report 

and as an interactive web report at:

            http://www.pipelinereport.org

About HIV i-Base – HIV i-Base is a London-based HIV treatment activist organization. HIV i-Base works in the United Kingdom and internationally to ensure that people living with HIV are actively engaged in their own treatment and medical care and are included in policy discussions about HIV treatment recommendations and access.

http://www.i-base.info 
About TAG – Treatment Action Group (TAG) is an independent AIDS research and policy think tank fighting for better treatment, a vaccine, and a cure for AIDS. TAG works to ensure that all people with HIV receive lifesaving treatment, care, and information. We are science-based treatment activists working to expand and accelerate vital research and effective community engagement with research and policy institutions. TAG catalyzes open collective action by all affected communities, scientists, and policy makers to end AIDS. TAG is a nonprofit, tax-exempt 501(c)(3) organization.

http://www.treatmentactiongroup.org 

Supreme Court strikes down DOMA

The gay rights movement saw a significant victory at the Supreme Court Wednesday, where the justices struck down part of a law that prohibits federal recognition of same-sex marriages.

In a 5-4 ruling, the court struck down Section 3 of the 17-year-old Defense of Marriage Act (DOMA), a provision of the law that denies federal benefits -- like Social Security benefits or the ability to file joint tax returns -- to same-sex couples legally married.

"DOMA is unconstitutional as a deprivation of the equal liberty of persons that is protected by the Fifth Amendment," Justice Anthony Kennedy wrote for the majority.

http://www.cbsnews.com/8301-250_162-57591089/supreme-court-strikes-down-doma/

ResearchMatch Connects HIV Study Volunteers with Researchers

I highly encourage everyone to join our important attempt to accelerate the cure of HIV.  Research Match also helps connect patients with other diseases with private investigators. Please forward to your friends and families.  Anyone can participate.

Thank you in advance for supporting this platform!

Contact: Kathy Edson

(615) 343-2571/ kathleen.edson@vanderbilt.edu

ResearchMatch Connects HIV Study Volunteers with Researchers

Nashville, TN – June 1, 2013 -- ResearchMatch (RM), the country’s first nationwide, non-profit research volunteer recruitment platform for all health conditions is launching an HIV sub-registry in collaboration with Program for Wellness Restoration (PoWeR), a national grassroots HIV advocacy andeducation organization.

RM has a simple goal – to bring together two groups of people who are searching for each other: people who are trying to find research studies, and researchers who are looking for people to participate in their studies. It is a free, secure online registry that has been developed by major academicinstitutions across the country that want to engage others in the mission of today’s ethical, expedited research that will hopefully make a real difference for people with HIV/AIDS in the future.

The Centers for Disease Control (CDC) estimates that 1,148,200 persons are living with HIV infection in the United States, including 207,600 (18.1%) who are unaware of their HIV status. Although great strides have been made in developing medications that treat HIV, research volunteers are needed foremerging HIV cure research studies that may lead to a functional HIV cure in the future. Presently, Researchmatch.org is the only non-profit clinical trials registry that proactively connects potential volunteers and researchers.

“ResearchMatch.org will make a dramatic difference in accelerating HIV cure research by connecting researchers with volunteers who want to help but don’t know how to get involved or where to turn. The hope is that many people will register to volunteer and that academic and research institutions will takeadvantage of this free service that promotes faster study enrollment,” said Nelson Vergel, HIV activist/educator and director of PoWeR.

All volunteers who register with RM indicating HIV as one of their conditions will be informed of enrolling studies in their area and also be connected with investigators who register studies at sites in their area. New RM registered members will also be prompted to consider answering optional questionsabout their condition to enhance the precision and prescreening process for matching volunteers and research teams.

To register as a volunteer or a researcher, please visit researchmatch.org.

About ResearchMatch

ResearchMatch is a national partnership created as a centralized, web-based recruitment registry, connecting individuals who are interested in participating in research with researchers nationwide. The site is a funded in part by the National Institutes of Health (NIH) and Clinical and Translational ScienceAward (CTSA) program grants. The CTSA program is led by the NIH’s National Center for Advancing Translational Science (NCATS). The content of this website is solely the responsibility of ResearchMatch and Vanderbilt University and does not necessarily represent the official views of the NIH.

About Program for Wellness Restoration

Program for Wellness Restoration (PoWeR) is a national non-profit all-volunteer organization that provides patient-friendly educational information to HIV-positive people and their healthcare providers about ways to improve HIV treatment response, side effects and quality of life. More information can be found at www.powerusa.org.

SANGAMO BIOSCIENCES PRESENTS CLINICAL DATA DEMONSTRATING HIV RESERVOIR REDUCTION IN HIV-INFECTED SUBJECTS TREATED WITH MODIFIED IMMUNE CELLS WITH ZINC FINGER NUCLEASES

SANGAMO BIOSCIENCES PRESENTS CLINICAL DATA DEMONSTRATING HIV RESERVOIR REDUCTION IN HIV-INFECTED SUBJECTS TREATED WITH ZFP THERAPEUTIC®, SB-728-T


Unprecedented Immune Reconstitution Drives HIV Viral Reservoir Depletion
Encouraging Preliminary Anti-Viral HIV Data during Treatment Interruption in Ongoing SB-728-T Phase 2 Trials



Richmond, California, May 15, 2013 – Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the presentation of new clinical data from its program to develop a ZFP Therapeutic® for HIV/AIDS. The data, which demonstrate that SB-728-T treatment results in a reduction in the HIV reservoir in HIV-infected subjects, are being presented at the 16th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The meeting is being held in Salt Lake City from May 15-18, 2013.


HIV-infected subjects enrolled in Sangamo’s ongoing SB-728-902 clinical trial (Cohorts 1-3) received a single infusion of SB-728-T which resulted in a durable increase in total CD4 T-cells driven by increased ZFN-modified CD4 central memory T-cells. The extent of exposure of subjects to circulating zinc finger nuclease (ZFN) CCR5 protected CD4 T cells correlated with a long-term decrease in the peripheral blood mononuclear cell (PBMC) HIV reservoir as measured by proviral DNA. In addition, two of four evaluable subjects in Cohort 5 of this study showed a decrease of greater than one log in their viral load during a sixteen week treatment interruption (TI) with one of the subjects achieving a transiently undetectable viral load during the TI period. In subjects in which viral load decreased, a measureable anti-HIV response was observed, specifically a multi-functional response of CD8 T-cells to elements of HIV core proteins.

"These data are quite remarkable,” commented Dale Ando, M.D., Sangamo’s vice president therapeutic development and chief medical officer. “In previous clinical studies, a decline in the HIV reservoir has never been observed in subjects on long-term anti-retroviral therapy (ART) and any increase in the levels of CD4 cells in HIV-infected subjects is often associated with a concomitant increase in the size of the reservoir. In contrast, a single SB-728-T treatment of subjects on long-term ART produced a significant and durable improvement in CD4 count and, in the majority of subjects, a notable decrease in the HIV reservoir over time. An observed correlation with circulating ZFN CCR5 protected CD4 cells is extremely promising.”


Summary of Clinical Data


SB-728-0902 Cohorts 1-3 in Immunologic Non-Responders (INR)


Treatment of HIV subjects with a single infusion of SB-728-T leads to long-term increases in CD4 counts (up to 3 years in some subjects).


Long-term increases in CD4 counts correlate with increased CD4 central memory and increased


ZFN CCR5 protected central memory T-cells.


The extent of long-term exposure to circulating ZFN-CCR5 protected CD4 cells correlates with long- term decreases in the PBMC HIV reservoir.



SB-728-0902 Cohort 5 (CCR5 delta-32 Heterozygotes)


Post SB-728-T infusion, a 16-week ART TI can lead to viral load reduction from initial peak.


o Two out of four subjects showed reduction in viral load during TI


o One subject achieved transient undetectable viral load during TI


The best viral load reduction responses are seen in subjects with CD8 T-cell HIV gag immune responses that are polyfunctional (expression of multiple cytokines) and the highest levels of bi-allelic modification of the CCR5 gene.


SB-728-1101 Immunologic Responders with Cytoxan Conditioning

Accrual and treatment in progress with ten subjects infused to date.


Analysis of numbers of modified SB-728-T cells and viral load during TI is in progress.


Viral load decreases correlate with highest levels of estimated biallelic CCR5 modification


Decreases in viral load from peak to the end of TI correlated with mean circulating bi-allelic


ZFN CCR5 protected CD4 cells during the TI for all patients to date who fully completed TI per protocol in SB-728-Penn, 902 Cohort 5 (CCR5 delta-32 Heterozygotes) and 1101 studies.


“These data continue to demonstrate the important link between SB-728-T-driven immune reconstitution and HIV viral load depletion,” said Rafick-Pierre Sékaly, Ph.D., Co-Director & Chief Scientific Officer, The Vaccine & Gene Therapy Institute of Florida (VGTI Florida), whose laboratory carried out the immunologic analyses. “Specifically, SB-728-T treatment protects long-term central memory CD4 T-cells from HIV-infection which is key for the successful development of an immunologic approach to HIV. ZFN-protected central memory cells are driving positive effects on total CD4 T-cell counts in treated subjects and appear to also play a role in breaking the cycle of HIV reservoir maintenance.”

“We are very encouraged by our data to date and by our continued progress in understanding the factors required to maximize the potential of this novel immunologic approach to a functional cure for HIV,” stated Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. “Sangamo has demonstrated that we have the necessary factors for success: SB-728-T is well-tolerated; the modified cells engraft and traffic throughout the body, appear to be immunologically active, and importantly, persist.

We observe an unprecedented increase in total CD T-cell levels which correlates with the levels of ZFN-protected CD4 central memory T-cells, and a related long-term decrease in the viral reservoir. We have also observed reduction in viral loads during TI, to undetectable levels transiently in one of four subjects, providing a second example of this observation. Viral load changes during TI continue to correlate with circulating bi-allelic ZFN CCR protected CD4 cells. In addition, we have identified key immunologic markers of inflammation that correlate with the degree of engraftment and can potentially aid in the selection of subjects for which SB-728-T may be most effective.”

Dr. Nichol continued, “In our ongoing studies, we will continue to investigate these parameters including the threshold engraftment levels of biallelically modified T-cells and the types of HIV- reactive cells necessary to mount an immune response to the virus. We look forward to presenting the results of these completed studies at the end of 2013.”

The data were presented today by Dale Ando, M.D., Sangamo’s vice president of therapeutic development and chief medical officer in a Scientific Symposium entitled “Challenges and Success of Gene Therapy Product Approval.” Dr. Ando also chaired the symposium.

Data will also be presented from Sangamo’s Phase 1 clinical trial SB-728-902 Cohorts 1-3 in a second presentation at 2:15 pm MT, on Thursday, May 16, 2013, Abst.#: 58 “Long-term CD4 Reconstitution in HIV Subjects Receiving ZFN CCR5 Modified CD4 T-Cells (SB-728-T) May Be Attributed to the Sustained Durability of the Central Memory T-Cell Subset.”


Summary of Clinical Trial Design


About SB-728-902 Cohorts 1-3


The study is an open-label Phase 1 clinical trial to evaluate the safety and tolerability of single infusions of an escalating dose of an autologous (a patient’s own) CD4+ T-cell product genetically modified at the CCR5 gene by CCR5-specific ZFNs (SB-728-T). The trial enrolled nine HIV-infected subjects (three cohorts of three subjects each) who have sub-optimal T-cell levels and no detectable viral load on long-term ART. Subjects remained on their existing antiviral therapy while receiving treatment with SB-728-T.

About SB-728-902 Cohort 5

Up to 20 HIV-infected subjects heterozygous for the CCR5 delta-32 mutation (i.e. with one CCR5 gene that is naturally modified) who are currently on ART are being enrolled and will receive a single intravenous infusion of SB-728-T (5 to 30 billion modified cells). Two months after SB-728-T treatment, subjects undergo a 16 week TI during which time their anti-retroviral therapy is discontinued. ART will be reinstituted in subjects whose CD4 T-cell counts drop to <350 br="" nbsp="">cells/ mm3 and/or whose HIV-RNA increases to 100,000 /mL for three consecutive weekly measurements. At the end of the TI, subjects with a sustained detectable HIV viral load are reinstituted on ART. Subjects with an undetectable viral load can remain off ART until HIV RNA levels are detectable or their CD4 T-cell count drops below 350 cell/mm3 for three consecutive weekly measurements.


About SB-728-1101
SB-728-1101 is an open-label, dose escalation, multi-center study designed primarily to evaluate the safety and tolerability of escalating doses of cyclophosphamide (Cytoxan®) administered one day prior to SB-728-T infusion. Cytoxan is a drug that is used to transiently reduce the numbers of T-cells in the body which then rapidly repopulate once the drug is discontinued. Such lymphodepletive treatment has been used to enhance engraftment of adoptively transferred T-cells in the treatment of cancer and as therapy for numerous autoimmune diseases. The drug has been previously used in HIV-infected individuals and studies demonstrate that, while the drug was transiently lymphodepleting, it did not significantly reduce total CD4 T-cell counts over the long-term and was adequately tolerated.

In addition to safety, the study is evaluating the effect of escalating doses of Cytoxan on SB-728-T engraftment, the effect of SB-728-T treatment on viral load following ART interruption, the change in CD4+ T-cell counts in peripheral blood and the long-term persistence of SB-728-T.

At least 9 HIV-infected subjects on ART are being enrolled into 3 dose-escalating cohorts (3 subjects/cohort), and will receive intravenous Cytoxan (200 mg, 500 mg/m2 or 1000 mg/m2). Within each cohort, treatment is staggered so that each subsequent subject cannot be infused with Cytoxan until at least 2 weeks after the preceding subject. One day after receiving Cytoxan, subjects are infused with SB-728-T (5 to 30 billion cells). Six weeks after SB-728-T infusion, subjects with CD4 cell counts ≥500 cells/ mm3 undergo a 16 week TI during which time their anti-retroviral therapy is discontinued. ART will be reinstituted in subjects whose CD4 T-cell counts drop to  t500 cells/mm3and/or whose HIV-RNA increases to >100,000 copies/ mL for three consecutive weekly measurements. At the end of the TI, subjects with a sustained detectable viral load or CD4 T-cell count <500 br="" nbsp="">cells/ mm3 are reinstituted on ART. Subjects with an undetectable viral load can remain off ART until HIV RNA levels are detectable or their CD4 T-cell count drops below 500 cells/mm3 for three consecutive weekly measurements.


Additional Presentations at ASGCT

Twelve additional presentations from Sangamo and its collaborators will be featured later in the week and include data from preclinical and research-stage human therapeutic programs.  Therapeutic areas include additional presentations on HIV/AIDS,  ZFP-based approaches for monogenic diseases such as hemophilia, including applications of Sangamo’s proprietary In Vivo Protein Replacement Platform, hemoglobinopathies, and oncology.  Developments in gene-editing technology applications will also be featured in presentations at the meeting.

All abstracts for the meeting are available online at  2013 ASGCT Meeting Abstracts.

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. It has ongoing Phase 2 and Phase 1/2 clinical trials to

evaluate  the  safety  and  efficacy  of  a  novel  ZFP  Therapeutic®  for  the  treatment  of  HIV/AIDS.

Sangamo’s other therapeutic programs are focused on monogenic diseases, including hemophilia and hemoglobinopathies  such  as  sickle cell anemia and beta-thalassemia,  and  a program in  Parkinson’s disease. Sangamo’s core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs).   Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription  factors  (ZFP  TFs)  that  can  control  gene  expression  and,  consequently,  cell  function. Sangamo has entered into a strategic collaboration with Shire to develop therapeutics for hemophilia, Huntington’s disease and other monogenic diseases and has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma- Aldrich Corporation. For more information about Sangamo, visit the company’s website at www.sangamo.com.

ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.

This press release may contain forward-looking statements based on Sangamo’s current expectations. These forward-looking statements include, without limitation, the research and development of novel ZFP  TFs  and  ZFNs  as  ZFP  Therapeutics  and  therapeutic  applications  and  the  scope  of  such applications of Sangamo’s ZFP technology platform to specific human diseases and unmet medical needs, including a potential functional cure of HIV/AIDS, the expansion of clinical studies of SB-728-T in HIV-infected individuals, expected timing for the presentation of clinical trial data, the development of ZFP Therapeutics for monogenic diseases and stem cell applications. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to whether clinical trials will validate and support tolerability and efficacy of ZFP Therapeutic approaches, technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. See Sangamo’s SEC filings, and in particular, the risk factors described in the Company’s Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.

Contact

Sangamo BioSciences, Inc. Elizabeth Wolffe, Ph.D.

510-970-6000, x271

ewolffe@sangamo.com

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