GS 010

Drug Profile

GS 010

Alternative Names: GS010; ND4 gene therapy; rAAV2/2-ND4; RAAV2/2-ND4 vector; Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene

Latest Information Update: 25 Jun 2017

Price : $50

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

  • 14 Jun 2017 Efficacy and adverse events data from a phase I/II trial in Leber’s Hereditary Optic Neuropathy released by GenSight Biologics
  • 21 Apr 2017 Adverse events, efficacy and immunogenecity data from a phase I/IIa trial in Leber's hereditary optic atrophy presented at the 69th annual meeting of the American Academy of Neurology (AAN-2017)
  • 21 Feb 2017 GenSight Biologics completes enrolment in the phase III REVERSE trial for Leber's Hereditary Optic Atrophy in USA, United Kingdom, France, Italy and Germany
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