Research programme: gene therapy - MeiraGTx

Drug Profile

Research programme: gene therapy - MeiraGTx

Alternative Names: A-003 - MeiraGTx; A-004; A-005; A-006 - MeiraGTx; A-007 - MeiraGTx

Latest Information Update: 23 Dec 2016

Price : $50

At a glance

  • Originator MeiraGTx
  • Class Gene therapies
  • Mechanism of Action Gene transference; Platelet-derived growth factor beta receptor antagonists; Vascular endothelial growth factor receptor-2 antagonists
  • 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

  • Preclinical Amyotrophic lateral sclerosis; Colour vision defects; Dry age-related macular degeneration; Retinitis pigmentosa; Wet age-related macular degeneration

Most Recent Events

  • 22 Dec 2016 A 004 receives Orphan Drug status for Retinitis pigmentosa in USA
  • 20 Dec 2016 MeiraGTx plans a phase I/II trial for A 004 for retinitis pigmentosa in United Kingdom
  • 01 Sep 2016 Preclinical trials in Amyotrophic lateral sclerosis in USA (Parenteral) before September 2016
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