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NFS 02

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Drug Profile

NFS 02

Alternative Names: NFS-02; rAAV2-ND1

Latest Information Update: 22 Aug 2023

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At a glance

  • Originator Neurophth Therapeutics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; NADH dehydrogenase replacements
  • Orphan Drug Status

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

    Yes - Leber's hereditary optic atrophy
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Phase I/II Leber's hereditary optic atrophy

Most Recent Events

  • 17 Aug 2023 Phase-I/II clinical trials in Leber's hereditary optic atrophy in China (Intravitreous) (NCT05820152)
  • 17 Aug 2023 Phase-I/II clinical trials in Leber's hereditary optic atrophy in USA (Intravitreous) (NCT05820152)
  • 17 Apr 2023 NMPA approves IND application for NFS 02 in Leber's hereditary optic neuropathy

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