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HORA-001

Development stage: Unknown
Lead developer: Coave Therapeutics
Modality: Gene Therapies
Administration: Subretinal Injection

Overview

HORA-001 is a gene therapy developed by Horama (now Coave Therapeutics) for the treatment of inherited retinal dystrophies caused by pathogenic mutations in the CRB1 gene. This therapy aims to deliver a functional copy of the CRB1 gene to retinal cells, thereby restoring the function of the CRB1 protein, which plays a crucial role in maintaining retinal structure and cell organization. Horama acquired global rights for HORA-001 from Leiden University Medical Center in 2020, and a Phase 1/2 clinical study for CRB1-associated inherited retinal dystrophies was initiated in 2023.

Targets

CRB1 (Crumbs homolog 1)

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