Clinical Hold on Voyager Therapeutics Gene Therapy

FDA has placed a clinical hold on a Voyager Therapeutics planned clinical trial for VY-HTT01 for treating Huntington’s disease. The company submitted an IND last month and the FDA hold was placed pending the resolution of certain chemistry, manufacturing and controls matters. Voyager says it expects to receive specific feedback from FDA on the CMC issues within 30 days and it plans to work closely with the agency to resolve them and promptly begin the clinical evaluation of VY-HTT01.

Huntington’s disease is a neurodegenerative disorder caused by mutations in the huntingtin (HTT) gene, which causes the production of an abnormally large amount of HTT protein that accumulates inside nerve cells and causes their death. VY-HTT01 is described as a type of gene silencing therapy aimed at addressing the underlying cause of the disease by significantly reducing the amount of damaging HTT protein produced.