Research programme: retinitis pigmentosa gene therapy - GenSight Biologics

Drug Profile

Research programme: retinitis pigmentosa gene therapy - GenSight Biologics

Alternative Names: AAV2.7m8-ChrimsonR-tdTomato; GS 030; GS030 DP; Optogenetics

Latest Information Update: 02 May 2018

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At a glance

  • Originator GenSight Biologics
  • Class 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 - Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Retinitis pigmentosa

Most Recent Events

  • 06 Mar 2018 GenSight Biologics obtains a license for AAV.7m8 vector technology from Adverum Biotechnologies before march 2018
  • 10 Jan 2018 GenSight Biologics initiates enrolment in the phase I/II PIONEER trial of GS030 in Retinitis pigmentosa in May 2018 (NCT03326336)
  • 10 Jan 2018 GenSight Biologics announces intention to submit IND and CTA applications for Retinitis pigmentosa to global regulatory authorities in 2018
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