Research programme: gene therapy - Spark Therapeutics Ireland

Drug Profile

Research programme: gene therapy - Spark Therapeutics Ireland

Alternative Names: GT 038; RHO-adRP; RhoNova; SPK- LHON

Latest Information Update: 04 Nov 2017

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 Genable Technologies
  • Developer Spark Therapeutics Ireland
  • Class Gene therapies
  • Mechanism of Action Gene transference; RNA interference
  • 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

  • No development reported Retinitis pigmentosa

Most Recent Events

  • 04 Nov 2017 No recent reports of development identified for preclinical development in Retinitis-pigmentosa in Ireland (Intraocular, Injection)
  • 07 Mar 2016 Genable Technologies has been acquired by Spark Therapeutics
  • 31 Oct 2014 GT 038 receives Advanced Therapy Medicinal Product designation for Retinitis pigmentosa in European Union
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