Voyager Therapeutics reports selection of gene therapy development candidate

Voyager Therapeutics (VYGR) announced that the joint steering committee with its collaborator Neurocrine Biosciences (NBIX) has selected a lead development candidate in the Friedreich’s ataxia program. The candidate combines a frataxin gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER capsid discovery platform. The companies expect the program to advance into first-in-human clinical trials in 2025. Selection of the development candidate triggered a $5M milestone payment to Voyager, which the company expects to receive in the first quarter of 2024. Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program. “The nomination of this development candidate in FA marks an important step in our strategic collaboration with Neurocrine, reflecting the power of combining Voyager’s TRACER AAV capsids and payload design capabilities with Neurocrine’s expertise in neuroscience and clinical development,” said Alfred W. Sandrock, Jr., M.D., Ph.D., CEO of Voyager. “While there has been encouraging recent progress in the treatment of FA, it remains a very challenging and eventually fatal disease for which new therapeutic approaches are needed. We believe our strategy to replace the defective frataxin gene could address the underlying disease etiology of FA. We look forward to progressing this and our other gene therapy programs, including our wholly-owned SOD1 ALS program and our Neurocrine-partnered GBA1 Parkinson’s program, towards clinical studies.”