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Voyager Therapeutics Selects Gene Therapy Candidate for Parkinson's, Other GBA1-Mediated Diseases

NEW YORK – Voyager Therapeutics on Tuesday announced that it has selected a gene therapy candidate it will develop as a potential treatment for Parkinson's disease and other conditions linked with the GBA1 gene.

The selection triggered a $3 million milestone payment from its development partner Neurocrine Biosciences.

Lexington, Massachusetts-based Voyager inked an R&D, manufacturing, and commercialization agreement with Neurocrine last year. Under the terms of that deal, Voyager is slated to receive a $3 million milestone payment in Q2 upon the selection of this gene therapy candidate by a joint steering committee comprising members from the partnered firms.

The candidate, for which the companies expect to file an investigational new drug application with the US Food and Drug Administration next year, combines a GBA1 gene replacement payload with an intravenously administered capsid derived from Voyager's adeno-associated virus TRACER capsid discovery platform that crosses the blood-brain barrier.

Voyager is eligible to receive additional development and commercialization milestone payments as the program advances as part of the agreement with San Diego-based Neurocrine.

The 2023 agreement covers certain gene therapy programs targeting Parkinson's disease and other GBA1-mediated diseases, alongside three other undisclosed programs. In addition to milestone payments, Voyager is also eligible to receive tiered royalties on net sales, program funding, and an option to elect 50/50 cost- and profit-sharing in the US for the GBA1 program.

"We now see the potential for three gene therapies leveraging our novel TRACER capsids to enter the clinic in 2025: the Neurocrine-partnered GBA1 and [Friedreich's ataxia] programs, and our wholly owned SOD1 [amyotrophic lateral sclerosis] program," Voyager CEO Alfred Sandrock said in a statement.