The FDA has cleared the investigational new drug (IND) application for HG202, a CRISPR/Cas13 RNA-editing therapy developed by HuidaGene Therapeutics, for the treatment of neovascular age-related macular degeneration (nAMD). This marks the first time the FDA has cleared a CRISPR/Cas13 therapy for clinical development. The decision follows promising preclinical data and initial results from the 'SIGHT-I' trial conducted in China.
HG202: A Novel Approach to nAMD Treatment
HG202 utilizes a CRISPR/Cas13 system to knockdown VEGF-A mRNA, offering a non-receptor binding pathway to address nAMD. This is particularly relevant as up to 46% of AMD patients show poor response or develop resistance to current anti-VEGF therapies. The therapy is designed to provide a safe and effective alternative for these patients.
Alvin Luk, PhD, MBA, CCRA, co-founder and CEO of HuidaGene, stated, "This open IND for HG202 by the US FDA...marks a significant milestone for HuidaGene and the CRISPR RNA-editing field...[It] underscored its potential to address AMD using a non-receptor binding pathway through the Cas13 RNA editor to knockdown VEGF-A mRNA."
The BRIGHT Trial: Evaluating Safety and Efficacy
The BRIGHT trial (NCT06623279) is a Phase 1, open-label, dose-escalation study designed to assess the safety and tolerability of HG202 in patients with neovascular AMD. Secondary endpoints include improvements in visual acuity, reduction in retinal thickness, and decreased need for anti-VEGF injections. The trial aims to enroll patients to evaluate HG202's potential to fill the unmet need for effective nAMD treatments.
Xin Zhang, MD, MSc, COO and CMO of HuidaGene, emphasized the urgency of developing new nAMD treatments, stating, "AMD patients deserve safe, effective treatment options. The BRIGHT trial will evaluate HG202’s safety and efficacy to address this gap. We look forward to enrolling patients soon."
Technological Foundation
HG202 is built upon HuidaGene's HG-PRECISE platform, which leverages AI/ML to discover the Cas13X/Y system. Further engineering has resulted in a high-fidelity Cas13Y with efficient editing capabilities and low off-target effects, providing a strong technical foundation for clinical applications.
Hui Yang, PhD, co-Founder and chief scientific advisor of the company, explained, "Our AI/ML-driven HG-PRECISE platform led us to discover the Cas13X/Y system...Building on this basis, my team engineered high-fidelity Cas13Y with efficient editing and low off-target effects, laying out the technical foundation for future clinical applications."
