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HG303
- Development stage
- Unknown
- Lead developer
- 辉大
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intrathecal
01
Overview
HG303 is an investigational AAV-delivered gene therapy developed by HuidaGene Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS), specifically targeting patients with FUS mutations (FUS-ALS). The therapy utilizes HuidaGene's proprietary CRISPR/Cas12 system (hfCas12Max) to target and reduce the expression of the Fused in Sarcoma (FUS) protein, which forms toxic aggregates in the motor neurons of affected individuals. By lowering FUS levels, HG303 aims to slow or halt disease progression. It is administered via a single intrathecal injection to ensure delivery to the central nervous system. Clinical evaluation is currently focused on safety, tolerability, and preliminary efficacy in patients with early-onset ALS.
- Other names
- HG303HG-303HG 303
02
Targets
FUS (Fused in sarcoma)
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