Voyager gets FDA IND clearance for first tau-targeted gene therapy

The FDA has cleared Voyager Therapeutics’ IND application for VY1706, the company’s investigational gene therapy targeting intracellular and extracellular tau for Alzheimer’s disease.

“The IND clearance for VY1706 is the first for a tau-targeted gene therapy and follows a comprehensive preclinical program demonstrating a compelling pharmacology and safety profile,” said Alfred W. Sandrock, Jr., MD, Ph.D., Voyager CEO. “Recent third-party data continue to suggest that tau is the next critical target in Alzheimer’s disease, and that reducing tau production holds promise.”

The IND clearance enables Voyager to start a clinical trial of VY1706 in adults with early Alzheimer’s, expected to begin in the second half of the year.

VY1706 is a potent, vectorized siRNA that targets MAPT mRNA (messenger RNA transcribed from the microtubule-associated protein tau gene) to decrease levels of both intracellular and extracellular tau in the brain. This core is encapsulated in Voyager’s TRACER™ AAV capsid that leverages ALPL, a well-conserved, novel receptor identified by Voyager, to deliver the siRNA into the brain following a one-time, intravenous dose.

In preclinical studies, VY1706 has been demonstrated to reduce tau protein up to 75% in key brain regions relevant to Alzheimer’s and to de-target the liver, a source of adverse events associated with other systemically administered gene therapies.