A 002 - MeiraGTx

Drug Profile

A 002 - MeiraGTx

Alternative Names: A002 - MeiraGTx; AAV-CNGB3; AAV2/8-hCARp.hCNGB3; Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene - MeiraGTx

Latest Information Update: 04 Jun 2018

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At a glance

  • Originator 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

  • Phase I/II Colour vision defects

Most Recent Events

  • 02 Mar 2018 MeiraGTx's A 002 receives Priority Medicine (PRIME) status for Colour vision defects in European Union
  • 01 Feb 2018 MeiraGTx receives Rare Pediatric Disease Designation for Colour vision defects (Achromatopsia) in USA
  • 17 Oct 2017 Pharmacodynamics data from a preclinical trial in Colour vision defects released by MeiraGTx
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