Voyager Therapeutics announced the FDA placed a clinical hold on the company’s Investigational New Drug (IND) application for VY-HTT01 for the treatment of Huntington’s disease. Voyager submitted the IND to the FDA in September of 2020. The FDA placed the hold onto the program hold pending the resolution of certain chemistry, manufacturing and controls (CMC) matters. Voyager is expecting feedback from the FDA within the next 30 days and intends to resolve the issues in order to initiate clinical studies.
Voyager was partnered with Sanofi on the early development of VY-HTT01, but the two companies restructured their collaboration in 2019 and all the rights to the gene therapy reverted to Voyager.
VY-HTT01 is composed of an adeno-associated virus capsid (AAV1) and proprietary transgene that harnesses the RNA interference pathway to selectively knock down, or reduce, levels of HTT mRNA. The mechanism of action of VY-HTT01 is knockdown of HTT expression in neurons in the striatum and cortex, thereby reducing the level of toxicity associated with mutated protein in these brain regions, and slowing the progression of cognitive and motor symptoms.
About Huntington’s Disease
Huntington’s disease is a fatal, inherited neurodegenerative disease that results in the progressive decline of motor and cognitive functions and a range of behavioral and psychiatric disturbances. The average age of onset is 39 years, with patients typically dying approximately 15 to 20 years following diagnosis. According to the Huntington’s Disease Society of America, Huntington’s disease affects approximately 30,000 patients in the United States. Huntington’s disease is caused by mutations in the huntingtin, or HTT, gene.