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

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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
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