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ALS Therapy Development Institute and Oxford BioMedica Begin Second Phase of Collaboration to Develop Gene Therapies for Amyotrophic Lateral Sclerosis

Thursday, January 28, 2010 Genetics & Stem Cells News
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CAMBRIDGE, Mass. and OXFORD, England, Jan. 27 The ALS Therapy Development Institute and Oxford BioMedica (LSE: OXB) announced today the extension of their collaboration following successful completion of the first phase. The extended collaboration, which is funded by the Muscular Dystrophy Association, aims to advance the development of Oxford BioMedica's preclinical gene therapy candidate, MoNuDinŽ, and to evaluate other gene-based strategies for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
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"ALS is a debilitating disease that takes so many of our best and brightest from us without warning and without reason. This collaboration adds yet another important element to our burgeoning drug development pipeline. Gene therapy is a crucial, emerging therapeutic option for diseases such as ALS and Oxford BioMedica has the experience and commitment for excellence needed in a partner," said Steven Perrin, Ph.D., Chief Executive Officer and Chief Scientific Officer of the ALS Therapy Development Institute (ALS TDI).
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The collaboration combines Oxford BioMedica's advanced LentiVectorŽ gene delivery technology with the ALS TDI's extensive gene expression database and drug screening capabilities. The first phase of the collaboration included the development of new techniques to evaluate and identify gene therapy candidates at the ALS TDI's research facility in Cambridge.

In this second next phase, the ALS TDI will conduct preclinical efficacy studies of Oxford BioMedica's LentiVector-based product, MoNuDin, which is designed to protect the motor neurons susceptible to degeneration from ALS by targeted delivery of the neuroprotective gene, vascular endothelial growth factor. Furthermore, the joint team plans to evaluate other potentially therapeutic genes that inhibit or regulate specific genetic pathways associated with disease onset or progression.

Oxford BioMedica's Chief Executive Officer, John Dawson, commented: "The successful completion of the first phase of the collaboration is testament to the quality of the scientific teams at the ALS TDI and Oxford BioMedica. Our LentiVector technology has unique capabilities to deliver genes safely and efficiently to neuronal cells and our product candidate, MoNuDin, has shown promising results in preclinical models of ALS. The extension of our collaboration with the ALS TDI enables us to accelerate MoNuDin's development and to explore new disease pathways as potential targets for genetic intervention. We are delighted to have ongoing support from the Muscular Dystrophy Association for our collaboration with the ALS TDI, which underscores our shared goal to develop new treatment options for patients suffering from ALS."

The Muscular Dystrophy Association's Augie's Quest Initiative is a major donor to the ALS TDI and provided the critical funding needed to execute the collaboration between Oxford BioMedica and the ALS TDI.

About ALS TDI

The mission of the ALS Therapy Development Institute (ALS TDI) is to develop effective therapeutics that slow or stop amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease), as soon as possible. Focused on meeting this urgent unmet medical need, ALS TDI executes a robust discovery program, while running the world's largest efforts to pre-clinically validate potential therapeutics; including small molecules, protein biologics, gene therapies and cell-based constructs. The world's first non-profit biotech, ALS TDI has developed an industrial-scale platform that allows for the development and testing of dozens of potential therapeutics each year. Built by and for patients, the Institute is the world's only non-profit biotechnology company with more than 30 professional scientists. In addition, the Cambridge, Massachusetts based research Institute collaborates with leaders in both academia and industry. For more information, please visit us online at www.als.net.

About Oxford BioMedica

Oxford BioMedica (LSE: OXB) is a biopharmaceutical company developing innovative gene-based medicines and therapeutic vaccines that aim to improve the lives of patients with high unmet medical needs. The Company's technology platform includes a highly efficient gene delivery system (LentiVectorŽ), which has specific advantages for targeting diseases of the central nervous system and the eye; and a unique tumour antigen (5T4), which is an ideal target for anti-cancer therapy. Through in-house and collaborative research, Oxford BioMedica has a broad pipeline. Partners include sanofi-aventis, Sigma-Aldrich and Pfizer. Technology licensees include Biogen-Idec, GlaxoSmithKline, Merck & Co and Pfizer. Further information is available at www.oxfordbiomedica.co.uk.

About MDA

MDA (www.mda.org) is the world's largest nonprofit provider of ALS services and funder of ALS research. Over the years, MDA has invested $250 million specifically fighting ALS. MDA operates more than 200 clinics at hospitals across the country, 36 of which are ALS-specific research and care centers. The Association's unparalleled health care services, research, advocacy and education programs provide help and hope to more than one million Americans affected by ALS and 42 other neuromuscular diseases.

About Augie's Quest

Fitness pioneer Augie Nieto started Augie's Quest (www.augiesquest.org) in conjunction with MDA's ALS Division. Nieto is co-founder and former president of Life Fitness of Chicago, and chairman of Octane Fitness. He and his wife, Lynne, serve as co-chairpersons of MDA's ALS Division. Nieto received a diagnosis of ALS in March 2005, and Augie's Quest raises money primarily through a variety of special events, including "Fight Night" in Tustin, CA; Augie's Quest Bash in San Diego, CA; "Tradition of Hope" in Los Angeles, CA; "Celebrity Golf Classic" in Phoenix, AZ; "Gift of Time: St. Patty's Day Bash" in Denver, CO; "Field of Hope Gala" in New Jersey; "Secure A Cure Golf Classic" in Purchase, NY; "Big Canyon Golf" in Newport Beach, CA; The ClubCorp Charity Classic at golf clubs nationwide; plus "Clubs for A Cure" special visitation benefits for Augie's Quest at LA Fitness and Bally Total Fitness locations across the country, as well as at scores of other independent and regional health clubs.

SOURCE ALS Therapy Development Foundation
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