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Timrepigene emparvovec - Biogen

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Drug Profile

Timrepigene emparvovec - Biogen

Alternative Names: AAV REP1 gene therapy - Biogen; AAV2 REP1; BIIB-111; NSR-REP1; rAAV2-REP1; rAAV2.REP1; Recombinant adeno-associated virus expressing REP1

Latest Information Update: 07 Mar 2022

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At a glance

  • Originator University of Oxford
  • Developer Biogen; University of Miami
  • 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

  • Suspended Choroideraemia

Most Recent Events

  • 31 Dec 2021 Suspended - Phase-III for Choroideraemia in France, United Kingdom, Canada, Denmark, Netherlands, USA, Germany, Finland (Intraocular) (Biogen SEC filing, Form 10-K, February 2022)
  • 01 Dec 2020 NightstaRx completes a phase III STAR trial in Choroideraemia in USA, Canada, Finland, France, Germany, Denmark, Netherlands and the United Kingdoms (NCT03496012)
  • 21 Nov 2019 Biogen completes enrolment in the phase III STAR trial for Choroideraemia in USA, Canada, Finland, Germany, Netherlands, United Kingdom and Denmark (NCT03496012) (EudraCT2015-003958-41)
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