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Entacingene turiparvovec - MeiraGTx

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Drug Profile

Entacingene turiparvovec - MeiraGTx

Alternative Names: A-002 - MeiraGTx; AAV-CNGB3; AAV2/8-hCARp.hCNGB3; Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene - MeiraGTx

Latest Information Update: 26 Jun 2025

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At a glance

  • Originator UCL Business
  • Developer MeiraGTx
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action CNBG3 gene 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 - Colour vision defects
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Colour vision defects

Most Recent Events

  • 11 Jun 2025 Discontinued - Phase-I/II for Colour vision defects (In adolescents, In children, In adults) in United Kingdom (Intraocular) prior to June 2025
  • 11 Jun 2025 Discontinued - Phase-I/II for Colour vision defects (In adults, In adolescents, In children) in USA (Intraocular) prior to June 2025
  • 04 Apr 2025 MeiraGTx terminates a long term follow-up phase I/II trial in Colour vision defects (In adolescents, In adults, In children) in USA and United Kingdom (Intraocular, Injection) due to strategic decision to not further develop AAV8-hCARp.hCNGB3 and AAV8-hG1.7p.coCNGA3. The decision is not due to safety concerns. (NCT03278873) (EudraCT2016-003856-59)

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