FDA Grants First Clearance for CRISPR/Cas13 RNA-Editing Therapy HG202 for Macular Degeneration

SHANGHAI and CLINTON (NJ), November 4, 2024 – HuidaGene Therapeutics, a global clinical-stage biotechnology company focused on genome medicines, has achieved a groundbreaking milestone. The US FDA has cleared its investigational new drug (IND) application for HG202, marking the first-ever clinical-stage CRISPR/Cas13 RNA-editing therapy to target neovascular age-related macular degeneration (nAMD). This FDA clearance propels HG202 as the only RNA-editing therapy of its kind currently in clinical development for nAMD.
Alvin Luk, Ph.D., Co-founder and CEO of HuidaGene, emphasized the impact of this clearance: “This open IND for HG202 by the US FDA – the first regulator to have cleared CRISPR/Cas13 for clinical development – represents a significant advancement for HuidaGene and the entire CRISPR gene-editing field of RNA editing. We chose the FDA pathway due to HG202’s promising in-vitro and in-vivo preclinical results, as well as initial findings from the ‘SIGHT-I’ first-in-human trial.”

Preclinical studies underscore HG202’s effectiveness. In laser-induced choroidal neovascularization (CNV) mouse models, HG202 reduced CNV area by 87%, surpassing both anti-VEGF antibody and gene therapy alternatives, showcasing substantial potential in nAMD treatment.

Dr. Xin Zhang, Chief Medical Officer at HuidaGene, highlighted HG202’s potential for patients who have limited response to current anti-VEGF therapies. “With up to 46% of AMD patients showing poor or diminishing responses to anti-VEGF treatments, resulting in irreversible vision loss, our work with HG202 is a critical step forward. The BRIGHT trial will assess HG202’s safety and efficacy across doses, moving us closer to viable solutions for those who have exhausted standard treatment options.”

The BRIGHT Trial (NCT06623279)

The BRIGHT trial is a Phase 1, open-label, dose-escalation study designed to evaluate the safety and tolerability of HG202 in patients with nAMD. Key trial endpoints include safety, tolerability, changes in best-corrected visual acuity (BCVA), central retinal thickness (CRT), and a reduction in anti-VEGF rescue injections.

HuidaGene’s CRISPR Innovations

HuidaGene has developed its RNA-editing technology through its AI/ML-driven HG-PRECISE® platform. Co-Founder Dr. Hui Yang explained that the discovery of the Cas13X/Y system allowed HuidaGene to create the high-fidelity Cas13Y (hfCas13Y), characterized by efficient RNA editing with minimal off-target effects, providing a strong foundation for clinical applications in RNA therapy.

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