New company to develop novel gene therapies

February 12th, 2014
UMass Medical School (UMMS) recognizes the launch today of Voyager Therapeutics, a gene therapy company founded by four world leaders in the fields of AAV gene therapy, RNA biology and neuroscience, to develop life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS).

UMMS researchers Phillip D. Zamore, PhD, co-director of the RNA Therapeutics Institute, and Guangping Gao, PhD, director of the Gene Therapy Center, are two of the scientific founders.

Voyager Therapeutics will focus on an adeno-associated virus (AAV) approach to gene therapy, which has the potential to transform treatment for a wide range of CNS diseases with one-time therapies that may dramatically improve patients' lives. The company is committed to advancing the field of AAV gene therapy by innovating and investing in areas such as vector optimization and engineering, dosing techniques, and process development and production.

Voyager's scientific and clinical founders are leaders in the fields of AAV gene therapy, expressed RNA interference (RNAi) and neuroscience and include:

  • Krystof Bankiewicz, MD, PhD, a translational neurosurgeon and leader in AAV gene therapy who has brought multiple new AAV therapies to the clinic; he is the Kinetics Foundation Chair in Translational Research and a Professor in Residence of Neurological Surgery and Neurology at the University of California, San Francisco (UCSF).
  • Guangping Gao, PhD, a leading AAV researcher who has played a key role in the discovery and characterization of new AAV serotypes; he is director of the UMMS Gene Therapy Center & Vector Core; scientific director of the UMMS-China Program Office; professor of microbiology & physiology systems and the Penelope Booth Rockwell Professor in Biomedical Research.
  • Mark Kay, MD, PhD, a leading researcher in the fields of gene therapy and AAV biology, including the identification of new AAV capsids; he is the head of the Division of Human Gene Therapy and professor in the departments of pediatrics and genetics at Stanford University School of Medicine; he is one of the founders of the American Society of Gene and Cell Therapy and is currently the editor of Human Gene Therapy.
  • Phillip D. Zamore, PhD, a leader and innovator in the understanding of RNAi and development of related therapeutic approaches, including expressed RNAi; he is a Howard Hughes Medical Institute Investigator, the Gretchen Stone Cook Chair of Biomedical Sciences, professor of biochemistry & molecular pharmacology and co-director of the RNA Therapeutics Institute at UMMS.

Voyager is launching with multiple clinical and preclinical product programs for CNS diseases in dire need of effective new therapies, including a Parkinson's disease program in an ongoing Phase 1b study with its collaborators at UCSF, as well as preclinical programs for a monogenic form of amyotrophic lateral sclerosis (ALS) and Friedreich's ataxia.

Depending on the disease, Voyager's gene therapy products will use either gene replacement or gene knockdown techniques. By significantly increasing or decreasing production of relevant proteins at targeted sites within the CNS, the goal is to address the underlying biology of the disease and make a meaningful difference for patients.

"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," said Mark Levin, interim chief executive officer of Voyager and partner at Third Rock. "We are extremely pleased to join forces with UMMS, a world leader in AAV gene therapy research and translation."

Voyager has entered into license and other agreements with UMMS, UCSF and Stanford University to access relevant technology and data, including certain rights to intellectual property related to expressed RNAi technology discovered at UMMS, clinical data related to the Parkinson's disease program from UCSF and certain rights to AAV intellectual property developed at Stanford.

In order to accelerate its research and development efforts, Voyager has also entered into a broad strategic collaboration with UMMS.

"A renaissance is underway in the field of gene therapy, as its long-held promise is fulfilled," said Terence R. Flotte, MD, executive deputy chancellor, dean of the school of medicine and the Celia and Isaac Haidak Professor in Medical Education at UMMS. "Voyager is poised to be a key player in bringing the power of this technology to patients suffering from tragic neurologic diseases."

Over the past 10 years, AAV has emerged as a highly promising and attractive approach to gene therapy. In clinical trials, AAV has been shown to be a safe and effective gene therapy delivery vehicle. Advances in AAV vector development and related dosing techniques that enable widespread gene delivery in the brain and spinal cord have made AAV particularly well-suited for the treatment of CNS diseases. In addition, improvements in related production technology and approaches have made AAV production more easily scalable and cost effective to meet commercial requirements.

Voyager's founding management team has deep expertise and a track record of building exceptional life science companies.

Provided by University of Massachusetts Medical School

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