Two Boston-area biotech companies priced IPOs last week, raising millions of dollars for the development neurological disease therapies. WaVe Life Sciences produces “stereopure” RNA-based drugs proposed to confer improved safety and potency over other RNA-based therapies. The company is focusing on Huntington’s disease (HD) and Duchenne muscular dystrophy, for which clinical development may start as early as late 2016. Voyager Therapeutics is developing gene therapies for CNS diseases, including ALS, HD, and spinal muscular atrophy (see Feb 2014 news). The company is developing optimized adeno-associated viral vectors (AAV) for delivery into the brain or spinal cord, a challenging task fraught with safety risks. The company in clinical development stages of a Parkinson’s disease gene therapy, while other candidates will likely not enter clinical trials until 2017.
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