FDA Clears HuidaGene's CRISPR-Cas13 RNA-Editing Trial for Macular Degeneration
核心洞察
HuidaGene Therapeutics (搜索) received FDA clearance for its IND application for HG202, a Cas13-based RNA-editing therapy, to treat neovascular age-related macular degeneration (搜索) (nAMD).
HG202, delivered via AAV vector, aims to reduce VEGF (搜索)-A mRNA expression in the retina, addressing both anti-VEGF treatment-resistant and responsive nAMD patients.
The BRIGHT trial, a Phase 1 dose-finding study, will assess HG202's safety, tolerability, and efficacy in nAMD patients, with secondary endpoints including visual acuity and retinal thickness.
HuidaGene Therapeutics (搜索) 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 (搜索) (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 (搜索)), 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 (搜索) 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 (搜索) 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 (搜索) rescue injections. The trial aims to enroll patients with nAMD, a condition affecting nearly 190 million people over the age of 60 worldwide.