Drug intelligence / Profile preview

HG202

Development stage
Unknown
Lead developer
辉大
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravitreal, Ophthalmic
01

Overview

HG202 is a first-in-class, clinical-stage gene therapy developed by HuidaGene Therapeutics. It utilizes a high-fidelity CRISPR/Cas13 RNA-editing system (specifically Cas13Y) delivered via a single adeno-associated virus (AAV) vector to target and knock down VEGF-A mRNA in the retina. The therapy is designed for the treatment of neovascular age-related macular degeneration (nAMD), including patients who have developed resistance or poor response to standard anti-VEGF therapies. Preclinical studies demonstrated that HG202 significantly reduced choroidal neovascularization compared to both anti-VEGF antibodies and AAV-based anti-VEGF gene therapies. The drug represents the first-ever clinical application of CRISPR/Cas13 RNA editing for any disease, with ongoing phase 1 trials evaluating its safety and efficacy in nAMD patients[1][2][3][4][5][6][7].

02

Targets

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