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Cotoretigene toliparvovec - Biogen

Drug Profile

Cotoretigene toliparvovec - Biogen

Alternative Names: AAV XLRPGR; AAV-RPGR; AAV-RPGR-Gene-Therapy-NightstaRx; AAV-XLRPGR gene therapy; BIIB-112; NSR AAV-RPGR; NSR-RPGR

Latest Information Update: 25 May 2021

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At a glance

  • Originator Isis Innovation; University of Oxford
  • Developer Biogen
  • 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.

    Yes - Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Phase II/III Retinitis pigmentosa

Most Recent Events

  • 14 May 2021 Interim efficacy and safety data from a phase II/III XIRIUS trial in Retinitis pigmentosa released by Biogen
  • 18 Nov 2020 Biogen completes the phase I/II XIRIUS trial in Retinitis pigmentosa (In adolescents, In adults, In children) in United Kingdom and USA (NCT03116113)
  • 12 Apr 2019 AAV RPGR gene therapy - Nightstar Therapeutics receives Fast Track designation for Retinitis pigmentosa [Intraocular,Injection] in USA
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