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Voyager Therapeutics, Neurocrine Biosciences Select Gene Therapy Candidate for Friedreich's Ataxia

NEW YORK – Voyager Therapeutics on Monday said it is developing a gene therapy candidate for Friedreich's ataxia through a collaboration with Neurocrine Biosciences, which the companies expect to advance into clinical trials next year.

Friedreich's ataxia is an inherited disorder characterized by movement and muscle coordination issues and is caused by mutations in FXN, a gene that encodes for the frataxin protein. Voyager and Neurocrine's gene therapy contains an FXN gene replacement payload with an intravenously administered capsid that penetrates the blood-brain barrier. The companies developed the novel capsid using Voyager's adeno-associated virus (AAV) capsid discovery platform, called Tropism Redirection of AAV by Cell-type-specific Expression of RNA, or Tracer for short.

Now that Voyager and Neurocrine have selected the Friedreich's ataxia gene therapy candidate, it will receive a $5 million milestone payment from Neurocrine in Q1.

Lexington, Massachusetts-based Voyager and San Diego-based Neurocrine entered into a collaboration agreement in 2019 to develop and commercialize AAV gene therapies for Friedreich's ataxia and two undisclosed targets. As part of the deal, Voyager is eligible to receive up to $1.3 billion in possible research, development, and commercialization milestone payments and tiered royalties on net sales of commercialized products. The deal includes an option for 60/40 cost- and profit-sharing for Neurocrine and Voyager, respectively, in the US for the Friedreich's ataxia program.