Gene Therapy Section Formed Within the Alliance for …

Edward Lanphier, President, Chief Executive Officer, Sangamo BioSciences; Jeffrey Walsh, Chief Operating Officer, bluebird bio; and Karen Kozarsky, Ph.D., Vice President, Research & Development, ReGenX Biosciences named as co-chairs

-- The Alliance for Regenerative Medicine and the American Society of Gene & Cell Therapy partner to support policies to advance novel gene therapies --

WASHINGTON, DC and SALT LAKE CITY, May 20, 2014 The Alliance for Regenerative Medicine (ARM) and the American Society of Gene & Cell Therapy (ASGCT) today announced their partnership. ARMs new Gene and GeneModified Cell Therapy Section (GTS) brings together the leading gene therapy companies and organizations in the U.S. and Europe to advocate for policies and programs to accelerate the development of new therapeutics to treat and cure a range of diseases for which no effective treatment options are available. ARMs focus on advocacy and clinical and commercial development will create a powerful alliance with the deep scientific and translational expertise resident in ASGCT.

The new ARM Gene Therapy Section is one of three technology sections that comprise the ARM membership. The other two sections focus on Cell Therapy and Tissue Engineering and Biomaterials. The Gene Therapy Section will dedicate its efforts to addressing regulatory, manufacturing, commercial and financial issues crucial to the success of the sector. In addition, the GTS will focus on building public awareness for this field of medicine and an appreciation for its potential to transform healthcare. The GTS will be led by Sarah Haecker, Ph.D., a member of ARMs senior staff. Sarah received her Ph.D. in Molecular Biology and Bioethics (with focus on gene transfer applications) and her postdoctoral scientific and business training in the Human Gene Therapy Program and the Center for Technology Transfer at the University of Pennsylvania. The three co-chairs of the group are Edward Lanphier, President, Chief Executive Officer, Sangamo BioSciences; Jeffrey Walsh, Chief Operating Officer, bluebird bio; and Karen Kozarsky, Ph.D., Vice President, Research & Development, ReGenX Biosciences.

As ARMs membership has grown, the organization has created specific technical sub-groups, such as the GTS, to focus on the unique development and commercialization needs of major sectors within advanced therapies said Lanphier. The addition of this new technology section is particularly exciting as the gene therapy field is making tremendous progress and holds great promise for transforming the lives of so many patients. It is our hope that gathering this group of technical, clinical and commercial experts in the field will help to accelerate product development and commercialization of these innovative technologies.

ASGCTs mission to bring together diverse stakeholders and advance the field of genetic and cellular therapies is closely aligned with ARMs goals, and we are looking forward to working with the organization, said Harry L. Malech, M.D., President-Elect of ASGCT. ARMs staff and members bring a wealth of knowledge involving regulatory and commercialization challenges in the field, and we feel this will nicely complement ASGCTs scientific and medical expertise.

ARM is the leading advocacy organization in the U.S. and Europe representing companies and organizations focused in the regenerative medicine and gene therapy field, and serves as an invaluable resource for all of its members. ASGCT, a nonprofit medical and scientific organization focused on genetic and cellular therapies, recently became a member of ARM and the two groups will work together to lead the advocacy and education efforts for the GTS of ARM.

Members of ARMs Gene Therapy Section: Abeona Therapeutics, AGTC, Alpha-1 Foundation, ALS Association, Association of Clinical Research Organizations (ACRO), American Society of Gene & Cell Therapy, Baxter/Chatham Therapeutics, Benitec Ltd., bluebird bio, Calimmune, Celgene Corporation, CIRM, Cornell University, Friends of Cancer Research, GenVec, Genzyme-Sanofi, Global Biotherapeutics, Juventas Therapeutics, MaxCyte, Memorial Sloan Kettering Cancer Center, National Multiple Sclerosis Society (NMSS), NeoStem, Oxford Biomedica, Parkinsons Action Network, Progenitor Cell Therapy (PCT), Pfizer, Prevent Cancer Foundation, ReGenX, Sangamo BioSciences, Shire, SironRX Therapeutics, Stop ALD, TissueGene, UniQure and Voyager Therapeutics

About The American Society of Gene & Cell Therapy: The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website at http://www.asgct.org.

About the Alliance for Regenerative Medicine: The Alliance for Regenerative Medicine (ARM) is a Washington, DC-based multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, DC to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today ARM has more than 150 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

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Gene Therapy Section Formed Within the Alliance for ...