FDA Clears IND Application for HG202 Gene Therapy in Wet AMD

The U.S. FDA has granted investigational drug clearance for HG202, a pioneering CRISPR/Cas13Y RNA-editing therapy developed by HuidaGene to treat neovascular (wet) age-related macular degeneration (AMD). This milestone makes HG202 the first CRISPR/Cas13Y RNA-targeting therapy in clinical development and the only CRISPR RNA-editing therapy currently in the clinical stage for AMD.

About HG202: A Novel CRISPR/Cas13Y Therapy for Wet AMD

HG202 utilizes CRISPR/Cas13Y technology, an innovative RNA-editing approach that selectively targets and modifies RNA to inhibit VEGF-A expression, a key factor in the abnormal blood vessel growth associated with wet AMD. This mechanism is designed to partially knock down mRNA expression without receptor binding, offering a unique pathway for potential AMD treatment.

Phase 1 Clinical Trial to Begin After Promising Preclinical Data

Following encouraging preclinical results, HuidaGene is now initiating a phase 1 dose-finding trial to evaluate HG202’s safety and efficacy. This trial aims to establish optimal dosing parameters, marking the therapy's first entry into the clinical phase.

Milestone for RNA-Editing Therapy in Ophthalmology

Alvin Luk, PhD, MBA, CCRA, co-founder and CEO of HuidaGene, emphasized the significance of the FDA’s clearance:

“This open IND for HG202 by the U.S. FDA — the first regulator to have cleared CRISPR/Cas13 for clinical development — represents an important milestone for HuidaGene and the entire CRISPR gene-editing field of RNA editing. We chose to go to the FDA because HG202 demonstrated good results in the in vitro, in vivo preclinical studies and first-in-human SIGHT-I trial.”

In September 2023, HuidaGene dosed the world’s first patient with a CRISPR/Cas13 RNA-editing therapy, marking a critical advancement in gene therapy for ocular disease. Preliminary data from HG202 have already been shared at key scientific conferences, including ARVO, ASGCT, Euretina, and ESGCT in 2023.

Potential Impact on AMD Treatment

HuidaGene’s non-receptor binding approach with CRISPR/Cas13 RNA editing has the potential to offer AMD patients a new therapeutic option. By selectively reducing VEGF-A expression in retinal cells, HG202 aims to directly address the pathological neovascularization underlying wet AMD.

With FDA clearance for its investigational new drug application, HG202 advances as a novel, targeted therapy for wet AMD, setting a precedent in the RNA-editing and gene therapy landscape for retinal diseases.