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Voyager: a new company tackling CNS disease via gene therapy
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A new biotechnology company called Voyager Therapeutics was launched this week with the goal of developing gene therapies for diseases of the central nervous system (CNS). The scientific founders of Voyager Therapeutics are experts in the fields of adeno-associated virus (AAV) gene therapy, RNA biology, including RNA interference, and neuroscience. They include the University of Massachusetts researchers Phillip D. Zamore, PhD and Guangping Gao, PhD, Mark Kay, MD, PhD of Stanford University and Krystof Bankiewicz, MD, PhD of the University of California.

Voyager Therapeutics will exploit the expertise of the founders by focusing on an AAV approach to gene therapy. This may transform treatment for a range of devastating CNS disease by allowing one-time therapies. The company will concentrate on sometimes problematic areas in AAV research including vector optimization and engineering, dosing techniques, and process development and production. The company already has multiple clinical and preclinical product programmes underway for CNS diseases, for example an on-going collaborative Parkinson’s disease Phase 1b study with researchers at the University of California and preclinical programmes for a monogenic form of amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.

AAV has emerged over the past decade as a promising gene therapy approach, with good safety profiles and effectiveness as a delivery vehicle. Development of AAV vectors that facilitate delivery to the brain and spinal cord means it is well placed for development of CNS disease therapies. AAV production has also become more commercially attractive in recent times, with developments in scalability and cost effectiveness of production.

Voyager’s proposed suite of gene therapies will include both gene replacement and gene knockdown techniques, depending on the disease to be targeted. These techniques can either increase or decrease production of relevant target proteins as appropriate. Thus, the therapy gets to the heart of the disease’s underlying biology.

Mark Levin, interim chief executive officer of Voyager and partner at Third Rock, is optimistic about the future of gene therapy in general. He says: “We believe the time is right for gene therapy, and we have assembled the expertise, technology and strategies to translate the promise of AAV gene therapy into breakthrough treatments for patients with these devastating CNS diseases.”

Voyager’s future will be bolstered by licencing agreements with the University of Massachusetts, the University of California and Stanford University, which allows the fledgling company to access relevant technology and data. This includes intellectual property on RNA interference technology discovered at the University of Massachusetts, Parkinson’s disease clinical data from the University of California and AAV intellectual property at Stanford.

Source
http://www.umassmed.edu/news/2014/resear...apies.aspx [UMass Medical School press release; accessed 19 February 2014].
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