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 REP1Latest Information Update: 07 Mar 2022
Price :
$50
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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
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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
- 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)