GS 010

Drug Profile

GS 010

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

Latest Information Update: 01 Aug 2017

$50 / €47 *
* $ price applies to USA, Canada and Latin America. € price applies to the rest of the world. Final gross prices may vary according to local VAT.

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

  • 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
  • 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)
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