New data for Friedreich's ataxia with a novel capsid demonstrate reversal of disease phenotype in a preclinical disease model.

Press Release: CAMBRIDGE, Mass., Oct. 17, 2017 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases announced today multiple data presentations at the Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 17-20, 2017, in Berlin, Germany. The data include an oral presentation related to recent results from Voyager's ongoing Phase 1b trial of VY-AADC01 in advanced Parkinson's disease, as well as six poster presentations related to Voyager's novel adeno-associated virus (AAV) capsid optimization efforts, gene therapy manufacturing, and preclinical pipeline programs.

Rescue of Central and Peripheral Neurological Phenotype in a Novel Mouse Model of Friedreich's Ataxia by Intravenous Delivery of AAV Frataxin." Poster P107.

Friedreich's ataxia is a severe, inherited neurological disease caused by mutations in the frataxin gene leading to decreased expression of frataxin (FXN), which results in severe sensory impairment, progressive loss of the ability to walk, generalized weakness, loss of sensation, as well as severe and potentially fatal cardiomyopathy. In a transgenic mouse model of FA, one-time intravenous post-symptomatic dosing of an AAV vector composed of a novel AAV capsid and a frataxin transgene, together with intracerebral dosing also delivering a frataxin transgene, rapidly halted and reduced FA disease progression in multiple tests including three functional tests of motor behavior and one electrophysiological test. In addition, increasing intravenous vector doses with the same novel capsid together with a fixed dose of the intracerebral vector led to a dose-dependent rescue of the FA phenotype. This novel AAV capsid provided at least 20-fold greater delivery of the vector to sensory ganglia as measured by vector genomes, and approximately a three-fold greater expression of frataxin in the cerebellum, as compared to an AAV9 vector at a similar dose. Additional preclinical studies are underway at Voyager including steps to optimize a lead clinical candidate for the treatment of FA.

New data for Friedreich's ataxia with a novel capsid demonstrate reversal of disease phenotype in a preclinical disease model.

Voyager Therapeutics Announces New Data Presentations at the Congress of the European Society of Gene and Cell Therapy