AAV-REP1 - Nightstar Therapeutics

Drug Profile

AAV-REP1 - Nightstar Therapeutics

Alternative Names: AAV2 REP1; NSR-REP1; rAAV2-REP1; rAAV2.REP1; Recombinant adeno-associated virus expressing REP1

Latest Information Update: 18 Jun 2018

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At a glance

  • Originator University of Oxford
  • Developer Nightstar Therapeutics; University of Alberta; University of Miami; University of Oxford
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action CHM protein expression stimulants; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Choroideraemia
  • New Molecular Entity No

Highest Development Phases

  • Phase III Choroideraemia

Most Recent Events

  • 14 Jun 2018 AAV-REP1 receives Regenerative Medicine Advanced Therapy (RMAT) Designation from the FDA for Choroideremia
  • 03 Apr 2018 Updated efficacy data from a phase I/II trial in Choroideraemia released by Nightstar Therapeutics
  • 05 Mar 2018 Pooled efficacy data from a phase I/II trials in Choroideraemia released by Nightstar Therapeutics
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