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EMD-203

Development stage: Preclinical
Lead developer: EmendoBio
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration: Subretinal, Intravitreal

Overview

EMD-203 is a preclinical gene-editing therapy developed by Emendo Biotherapeutics for the treatment of macular dystrophy. The drug utilizes in vivo excision to target and correct mutations in the RPE65 gene, which are implicated in certain forms of retinal degeneration such as retinitis pigmentosa and macular dystrophy. The mechanism of action involves monoallelic excision of the mutated RPE65 gene, aiming to restore normal function in retinal cells. EMD-203 is part of Emendo's ophthalmic pipeline leveraging their OMNI technology platform for precise genome editing.

Targets

RPE65 (Retinal pigment epithelium-specific 65 kDa protein)

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