AAV-REP1 - NightstaRx

Drug Profile

AAV-REP1 - NightstaRx

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

Latest Information Update: 26 Jul 2016

Price : $50

At a glance

  • Originator University of Oxford
  • Developer NightstaRx; University of Alberta; University of Miami; University of Oxford
  • Class 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 - Retinal dystrophies
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase III Retinal dystrophies

Most Recent Events

  • 13 Jul 2016 Phase-III clinical trials in Retinal dystrophies in Finland (Intraocular) (EudraCT2015-003958-41)
  • 12 Nov 2015 Phase-II clinical trials in Retinal dystrophies in USA (Intraocular)
  • 29 Jun 2015 Phase-II clinical trials in Retinal dystrophies in Canada (Intraocular)
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