GS 010

Drug Profile

GS 010

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

Latest Information Update: 13 Dec 2017

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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

  • 05 Dec 2017 Long term efficacy data from a phase I/IIa trial in Leber's hereditary optic neuropathy released by GenSight Biologics
  • 03 Oct 2017 GenSight Biologics plans a phase III trial for Leber's hereditary optic neuropathy (In children, In adolescents, In adults) (NCT03293524)
  • 01 Aug 2017 GenSight Biologics completes enrolment in the phase III RESCUE trial for Leber's hereditary optic atrophy in France, Germany, Italy, USA and United Kingdom
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