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HG301

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

Overview

HG301 is an investigational gene therapy candidate developed by HuidaGene Therapeutics for the treatment of inherited retinal diseases (IRDs), specifically those associated with mutations in the rhodopsin (RHO) gene, such as autosomal dominant retinitis pigmentosa (adRP). The drug functions as a RHO gene modulator, utilizing gene-editing or gene-regulation technology to address the underlying genetic defects that lead to photoreceptor degeneration and vision loss. Currently in the preclinical development stage, HG301 aims to provide a durable therapeutic effect by correcting or compensating for the RHO mutation directly within the retinal cells.

Targets

RHO (Rhodopsin)

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