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HORA RPE65

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Drug Profile

HORA RPE65

Alternative Names: HORA-RPE65

Latest Information Update: 23 Jul 2021

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At a glance

  • Originator French Muscular Dystrophy Association; INSERM; Nantes University Hospital; University Hospital of Montpellier
  • Developer Coave Therapeutics
  • 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 - Leber congenital amaurosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Leber congenital amaurosis

Most Recent Events

  • 21 Jul 2021 Horama is now called Coave Therapeutics
  • 08 Jan 2020 HORA RPE65 is still in phase I/II trials for Leber congenital amaurosis in France (Horama pipeline, January 2020)
  • 21 Sep 2017 Horama plans for extended phase I/II trial in Leber congenital amaurosis for 2017

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