GS 010

Drug Profile

GS 010

Alternative Names: GS010; ND4 gene therapy; rAAV2/2-ND4; RAAV2/2-ND4 vector

Latest Information Update: 16 Apr 2018

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At a glance

  • Originator GenSight Biologics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference
  • 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

Highest Development Phases

  • Phase III Leber's hereditary optic atrophy

Most Recent Events

  • 03 Apr 2018 Top-line efficacy and safety data from the phase III REVERSE trial in Leber's hereditary optic atrophy released by GenSight Biologics
  • 12 Mar 2018 Phase-III clinical trials in Leber's hereditary optic atrophy (In adults, In adolescents, In the elderly) in Belgium, USA (Intravitreous) (NCT03293524) (EudraCT2017-002187-40)
  • 20 Feb 2018 Updated efficacy and adverse events data from a phase I/II trial in Leber's hereditary optic atrophy released by GenSight Biologics
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