A 001 - MeiraGTx

Drug Profile

A 001 - MeiraGTx

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

Latest Information Update: 11 Dec 2017

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At a glance

  • Originator MeiraGTx
  • Developer Medical Research Council; MeiraGTx; University College London
  • Class 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

  • 07 Dec 2017 A 001 receives rare paediatric disease designation for Leber congenital amaurosis due to mutations in the RPE65 gene in USA
  • 17 Oct 2017 Pharmacodynamics data from a preclinical trial in Leber congenital amaurosis released by MeiraGTx
  • 01 Oct 2017 The US FDA accepts IND for A 001 for Leber congenital amaurosis (In children)
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