HuidaGene Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for HG202, a novel CRISPR-Cas13 RNA-editing therapeutic candidate for neovascular age-related macular degeneration (nAMD). This clearance allows HuidaGene to proceed with clinical trials to evaluate the safety and efficacy of HG202 in patients with nAMD, a leading cause of blindness in older adults.
HG202: A Novel Approach to nAMD Treatment
HG202 represents a first-in-class therapeutic approach as a clinical-stage Cas13-based RNA-editing therapy and the only clinical-stage RNA-targeting therapy for nAMD. The therapy is designed to address the overexpression of vascular endothelial growth factor (VEGF), a key driver of nAMD pathology. By reducing VEGF-A mRNA expression within the retina, HG202 aims to inhibit abnormal blood vessel growth and development, characteristic features of nAMD.
Traditional treatments for nAMD often involve anti-VEGF therapies; however, their effectiveness can wane over time, and many patients experience vision loss despite treatment. Approximately half of nAMD patients respond poorly or weaken in response to anti-VEGF therapies, leaving a significant unmet medical need. HG202 is designed to treat patients who have become resistant to anti-VEGF treatment as well as patients who respond to standard therapies.
Preclinical and Clinical Evidence
HG202 leverages HuidaGene's proprietary HG-PRECISE® platform, delivering a Cas13-based RNA-editing candidate via a single adeno-associated viral (AAV) vector. Preclinical studies in laser-induced choroidal neovascularisation (CNV) mice, a model for nAMD, demonstrated that HG202 treatment reduced the CNV area by 87%, surpassing the efficacy of both anti-VEGF antibodies and AAV-anti-VEGF gene therapy.
HG202 was previously evaluated in the SIGHT-I Phase 1 clinical trial in China. According to Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and Chief Executive Officer of HuidaGene, HG202 demonstrated good results in the in-vitro, in-vivo preclinical studies and first-in-human ‘SIGHT-I’ trial. Preliminary data were presented at ARVO, ASGCT, EURETINA, and ESGCT this year.
BRIGHT Trial: Phase 1 Study Details
With the FDA's IND clearance, HG202 will now be evaluated in the BRIGHT trial, an open-label, multi-center, Phase 1, dose-finding study in patients with nAMD. The primary endpoint of the trial is to assess the safety and tolerability of HG202 at different doses following a single administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), central retinal thickness (CRT) measurement, and the need for anti-VEGF rescue injections. The trial aims to enroll patients with nAMD, a condition affecting nearly 190 million people over the age of 60 worldwide.
