HuidaGene's CRISPR/Cas13 RNA-Editing HG202 Cleared by FDA for Macular Degeneration

HuidaGene Therapeutics, a global biotechnology company, has achieved a significant milestone as the U.S. FDA has approved its investigational new drug (IND) application for HG202. This development marks the first CRISPR/Cas13 RNA-editing therapy to be cleared for clinical use in treating neovascular age-related macular degeneration (nAMD), a condition affecting millions worldwide.

Dr. Alvin Luk, Co-founder and CEO of HuidaGene, emphasized the importance of this approval, noting that it is the first time the U.S. FDA has cleared a CRISPR/Cas13 therapy for clinical development. He highlighted the promising preclinical data for HG202 and positive outcomes from the initial human trial in China. HG202 utilizes a unique approach by targeting and reducing VEGF-A mRNA through the Cas13 RNA editor, offering a new pathway for treating age-related macular degeneration (AMD).

Dr. Xin Zhang, COO and CMO of HuidaGene, pointed out the pressing need for new treatments for nAMD, as many patients either respond poorly or develop resistance to existing anti-VEGF therapies. He stated that the upcoming BRIGHT trial aims to assess the safety and efficacy of HG202, with the goal of providing a new, effective treatment option for AMD patients. The trial will soon begin enrolling participants.

Dr. Hui Yang, Co-Founder and Chief Scientific Advisor of HuidaGene, elaborated on the technology behind HG202. The company's AI/ML-driven HG-PRECISE® platform led to the discovery of the Cas13X/Y system. The team has since engineered the high-fidelity Cas13Y, which boasts efficient editing and minimal off-target effects. This innovation lays the groundwork for future clinical applications.

The BRIGHT trial of HG202, identified as NCT06623279, is an open-label, dose-escalation study designed to explore the safety and efficacy of HG202 in nAMD patients. Primary objectives include assessing the safety and tolerability of the therapy, while secondary objectives focus on improvements in visual acuity, retinal thickness, and the necessity for anti-VEGF injections.

HuidaGene Therapeutics is at the forefront of developing genome medicines through its proprietary CRISPR-based HG-PRECISE® platform. The company is advancing several clinical programs, including the LIGHT trial for HG004, the STAR trial for RPE65-associated retinal disease, and the HERO trial for MECP2 duplication syndrome. Additionally, HuidaGene is conducting the MUSCLE trial for HG302 DNA-editing therapy targeting Duchenne muscular dystrophy (DMD).

The preclinical pipeline at HuidaGene includes HG303 DNA-editing therapy for amyotrophic lateral sclerosis (ALS) and a CRISPR RNA-editing therapy aimed at Alzheimer's disease. With a strong intellectual property portfolio, HuidaGene is positioned as a leader in the development of genome medicines, particularly in the fields of neurology and ophthalmology.