Drug Profile


Alternative Names: HORA-PDE6B

Latest Information Update: 25 Sep 2017

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At a glance

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

Highest Development Phases

  • Preclinical Retinitis pigmentosa

Most Recent Events

  • 19 Sep 2017 Preclinical trials in Retinitis pigmentosa in France (Intraocular) prior to September 2017
  • 19 Sep 2017 Horama plans a phase I/II trial for Retinitis pigmentosa (In children, In adolescents, In adults) in France in 2017 (EudraCT-2016-001429-16)
  • 19 Jun 2013 HORA PDE6B receives Orphan Drug status for Retinitis pigmentosa in European Union (Register of designated Orphan Medicinal Products by European Commission, September 2017)
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