AAV RPGR gene therapy - Nightstar Therapeutics

Drug Profile

AAV RPGR gene therapy - Nightstar Therapeutics

Alternative Names: AAV XLRPGR; AAV-RPGR; AAV-XLRPGR gene therapy; NSR AAV-RPGR; NSR-RPGR

Latest Information Update: 05 Apr 2018

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At a glance

  • Originator Isis Innovation; University of Oxford
  • Developer Nightstar Therapeutics
  • 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.

  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Retinitis pigmentosa

Most Recent Events

  • 19 Mar 2017 Phase-I/II clinical trials in Retinitis pigmentosa in United Kingdom (Intraocular)
  • 18 Mar 2017 Preclinical trials in Retinitis pigmentosa in United Kingdom (Intraocular)
  • 01 Mar 2017 The UK Medicines and Healthcare Products Agency (MHRA) approves Clinical trial application for phase I/II trial of AAV RPGR gene therapy in Retinitis pigmentosa before March 2017 (NightstaRx pipeline, March 2017)
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