Third Rock’s Voyager Hunts for CNS Disorders with Gene Therapy

Cambridge, MA-based Voyager Therapeutics has completed a $45M Series A round of equity financing for development of gene therapy and microRNA therapeutics for the treatment of central nervous system diseases. The company has its eyes set on ALS, Friedreich’s Ataxia and Parkinson’s disease. Voyager’s therapeutic strategy uses adeno-associated viral (AAV) vectors to introduce either a normal copy of a mutated gene or a microRNA to shut down a detrimental gene. AAV vectors are promising as vectors for gene therapy in humans, due to their relatively weak immunogenicity and their ability to generate widespread expression of the target gene in the brain. To treat ALS, Voyager plans to express micro RNAs that suppress mutant superoxide dismutase 1 (SOD1) expression. AAV-based therapies are already being developed by Dimension Therapeutics for rare diseases, by AveXis for treating spinal muscular atrophy, as well as Isis Pharmaceuticals has a major anti-sense program directed at knocking down SOD1. Voyager plans to build a rich AAV library to ultimately treat a variety of rare diseases. As for ALS – they hope to enter clinical trials in a couple of years.

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