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HG205
- Development stage
- Discontinued
- Lead developer
- 辉大
- Modality
- Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, CRISPR-Cas13 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intracochlear, Otic
01
Overview
HG205 is a CRISPR/dCas13X-based gene therapy developed by HuidaGene Therapeutics for the treatment of congenital sensorineural hearing loss resulting from OTOF (otoferlin) gene mutations, specifically the p.Q829X mutation. The drug utilizes RNA base-editing technology to modulate expression of the OTOF protein and is designed for direct intracochlear injection. HG205 aims to restore or enhance otoferlin function, which is critical for normal hearing, by correcting pathogenic mutations at the RNA level. Its mechanism of action is classified as an "OTOF protein expression modulator." Clinical development has primarily focused on pediatric populations.
- Other names
- HG205HG-205HG 205
02
Targets
Otoferlin (OTOF) p.Q829X mutant messenger RNA
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