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Friedreich's-ataxia gene therapy - Voyager Therapeutics

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Drug Profile

Friedreich's-ataxia gene therapy - Voyager Therapeutics

Latest Information Update: 14 May 2025

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At a glance

  • Originator Voyager Therapeutics
  • Developer Neurocrine Biosciences; Voyager Therapeutics
  • Class Gene therapies; Small interfering RNA
  • Mechanism of Action Frataxin protein replacements; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Friedreich's ataxia

Most Recent Events

  • 06 May 2025 Voyager Therapeutics in collaboration with Neurocrine Biosciences plans first in human clinical trial for Friedreich’s ataxia in 2026
  • 12 Nov 2024 Voyager Therapeutics announces intention to file an IND application for Friedreich's ataxia in 2025
  • 26 Feb 2024 Voyager Therapeutics enters into strategic collaboration agreement with Neurocrine Biosciences for research, development, and commercialization of AAV gene therapy products for programs targeting Friedreich’s ataxia and two other undisclosed targets

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