US FDA clears investigational new drug application for CRISPR/Cas13 RNA-editing therapy

Strong preclinical data for HG202 and promising results from the first-in-human ‘SIGHT-I’ trial in China underscored its potential to address AMD. Image credit: ©kittyfly – stock.adobe.com

The US Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for HG202, by HuidaGene Therapeutics, a company with headquarters in Shanghai, China, and Clinton, New Jersey. According to a press release, the drug is the world’s first-ever CRISPR/Cas13 RNA-editing therapy for clinical use in treating neovascular age-related macular degeneration (nAMD).

Alvin Luk, PhD, MBA, CCRA, the co-founder and CEO of HuidaGene, commented on the milestone in the company's press release. He said, “This open IND for HG202 by the US FDA-the first regulator to clear CRISPR/Cas13 for clinical development–marks a significant milestone for HuidaGene and the CRISPR RNA-editing field. The strong preclinical data for HG202, combined with promising results from the first-in-human ‘SIGHT-I’ trial in China, underscored its potential to address AMD using a non-receptor binding pathway through the Cas13 RNA editor to knockdown VEGF-A mRNA.”

Xin Zhang, MD, MSc, COO and CMO of HuidaGene, highlighted the importance and urgency of developing new nAMD treatments to benefit patients worldwide, saying “Up to 46% of AMD patients respond poorly or develop resistance to anti-VEGF therapies. 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.”

Similarly, Hui Yang, PhD, the co-Founder and chief scientific advisor of the company shared his thoughts and explained the technical side of this candidate, saying, “Our AI/ML-driven HG-PRECISE platform led us to discover the Cas13X/Y system (Nature Methods 2021). Building on this basis, my team engineered high-fidelity Cas13Y with efficient editing and low off-target effects (Nature Biotechnology 2022), laying out the technical foundation for future clinical applications.”

BRIGHT Trial of HG202 (NCT06623279)

The BRIGHT trial is a phase 1, open-label, dose-escalation study for CRISPR/Cas13-RNA targeting therapy for the treatment of neovascular AMD. Primary endpoints include the safety and tolerability of HG202, with secondary endpoints assessing improvements in visual acuity, retinal thickness, and the need for anti-VEGF injections.

Reference:

1. HuidaGene Therapeutics Receives the First-Ever FDA Clearance of CRISPR/Cas13 RNA-Editing HG202 for Macular Degeneration. HuidaGene Therapeutics. November 4, 2024. Accessed November 12, 2024. https://www.biospace.com/press-releases/huidagene-therapeutics-receives-the-first-ever-fda-clearance-of-crispr-cas13-rna-editing-hg202-for-macular-degeneration