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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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