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NBIB 223

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Drug Profile

NBIB 223

Alternative Names: Friedreich's-ataxia gene therapy - Voyager Therapeutics; NBIB-‘223

Latest Information Update: 18 May 2026

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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.

    Yes - Friedreich's ataxia
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Friedreich's ataxia

Most Recent Events

  • 07 May 2026 NBIB 223 receives Orphan Drug status for Friedreich's ataxia in USA
  • 10 Mar 2026 Voyager Therapeutics in collaboration with Neurocrine Biosciences plans first in human clinical trial for Friedreich’s ataxia in second half of 2026
  • 12 Nov 2024 Voyager Therapeutics announces intention to file an IND application for Friedreich's ataxia in 2025

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