A 004

Drug Profile

A 004

Alternative Names: A004; AAV-RPGR - MeiraGTx; AAV2/5-hRKp.RPGR - MeiraGTx

Latest Information Update: 16 Aug 2018

Price : *
* Final gross price and currency may vary according to local VAT and billing address.
* Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase. A link to download a PDF version of the drug profile will be included in your email receipt. Adis is an information provider. We do not sell or distribute the pharmaceutical compounds written about in this database.

At a glance

  • Originator MeiraGTx
  • 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 - Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Retinitis pigmentosa

Most Recent Events

  • 13 Aug 2018 A 004 receives Orphan Drug status for Retinitis pigmentosa in European Union, prior to August 2018
  • 08 Aug 2018 The independent data monitoring committee (IDMC) recommends approval of third and highest dose cohort in phase I/II trial for X-linked Retinitis pigmentosa
  • 23 Apr 2018 A 004 receives Fast Track designation for Retinitis pigmentosa [Intraocular] in USA
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top