February 12, 2014
Third Rock Ventures, LLC announced the formation of Voyager Therapeutics, a gene therapy company developing life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS), with a $45 million Series A financing. Voyager’s adeno-associated virus (AAV) approach to gene therapy 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, as well as process development and production.
Voyager was founded by world leaders in the fields of AAV gene therapy, expressed RNA interference (RNAi) and neuroscience:
- Krystof Bankiewicz, M.D., Ph.D., is 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, Ph.D., is a leading AAV researcher who has played a key role in the discovery and characterization of new AAV serotypes; he is Director of the University of Massachusetts Medical School (UMMS) Gene Therapy Center & Vector Core, Scientific Director of the UMMS-China Program Office and Professor of Microbiology and Physiology Systems and the Penelope Booth Rockwell Professor in Biomedical Research at UMMS.
- Mark Kay, M.D., Ph.D., is 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 Zamore, Ph.D., is 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 at UMMS, Professor of Biochemistry and 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 our 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, the company’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.
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 leader in AAV research and translation.
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.