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Cevaretigene ritoparvovec - MeiraGTx

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Drug Profile

Cevaretigene ritoparvovec - MeiraGTx

Alternative Names: A-001 - MeiraGTx; AAV RPE65 - MeiraGTx; AAV2/5 OPTIRPE65; OPTIRPE65 - MeiraGTx; RPE65-gene-therapy-MeiraGTx

Latest Information Update: 23 Apr 2024

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At a glance

  • Originator MeiraGTx
  • Developer Medical Research Council; MeiraGTx; University College London
  • 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 - Leber congenital amaurosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Leber congenital amaurosis

Most Recent Events

  • 31 Dec 2023 MeiraGTx has patent protection related to compositions of matter and methods of use relating to RPE65 program, and the AAV-RPE65 product candidate in USA, Albania, Australia, Austria, Belgium, Bulgaria, China, Croatia, Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hong Kong, Hungary, Iceland, India, Ireland, Israel, Italy, Japan, Latvia, Lithuania, Luxembourg, Malta, Malaysia, Mexico, Monaco, Netherlands, North Macedonia, Norway, Philippines, Poland, Portugal, Romania, San Morino, Serbia, Singapore, Slovakia, Slovenia, Spain, Sweden, Switzerland/Liechtenstein, Turkey and the United Kingdom before December 2023
  • 31 Dec 2023 MeiraGTx has patents pending related to compositions of matter and methods of use relating to RPE65 program, and the AAV-RPE65 product candidate in USA, Brazil, Canada, Europe, Hong Kong, Mexico, New Zealand, Nigeria, Thailand before December 2023
  • 10 Nov 2021 MeiraGTx plans a phase III trial for Leber congenital amaurosis in early 2022 (Ophthalmic)

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