CAMBRIDGE, Mass., Nov. 16, 2017 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today at its R&D Day highlighted recent progress and plans for VY-AADC for advanced Parkinson’s disease, and progress with testing novel adeno-associated virus (AAV) capsids and delivery optimization efforts, along with its ALS, Huntington’s disease, Friedreich’s ataxia, anti-Tau antibody and severe chronic pain preclinical programs.
"Preclinical data from its Friedreich’s ataxia (FA) program in a transgenic mouse model of FA, with a one-time intravenous (IV) dosing of an AAV vector composed of a novel capsid and a frataxin transgene, together with intracerebral dosing of an AAV vector with the same transgene, that led to a rapid halting and reduction of FA disease progression in multiple functional tests of motor behavior. Additional preclinical studies are underway at Voyager including steps to identify a lead clinical candidate for the treatment of FA during 2018."
Voyager Therapeutics Highlights Lead Program VY-AADC for Advanced Parkinson’s Disease, AAV Novel Capsid and Delivery Optimization Efforts, and Pipeline Programs at its R&D Day