NEW YORK – The US Food and Drug Administration has granted HuidaGene Therapeutics permission to begin a clinical trial of a CRISPR RNA-editing therapy it is developing to treat neovascular age-related macular degeneration (nAMD).
Shanghai-based HuidaGene said Monday it plans to launch an open-label, dose-escalation Phase I trial, dubbed BRIGHT, to investigate the safety and efficacy of HG202 in patients with nAMD, a late-stage eye disorder. Researchers will monitor the safety and tolerability of the investigational candidate and assess whether it improves patients' visual acuity and retinal thickness.
Patients with nAMD experience vision loss due to abnormal growth of blood vessels in the retina, which is stimulated by an excess of vascular endothelial growth factor (VEGF) proteins.
In a statement, HuidaGene CEO and Cofounder Alvin Luk lauded the FDA's clearance of the firm's investigational new drug application for HG202 and allowing the use of the CRISPR-Cas13 system in a clinical trial as "a significant milestone for HuidaGene and the CRISPR RNA-editing field."
HG202 is already being tested in a first in-human clinical trial called SIGHT-I in China. The RNA-editing therapy is designed to knock down VEGF-A mRNA. 4D Molecular Therapeutics, Adverum Biotechnologies, and AbbVie through a collaboration with Regenxbio are also advancing new gene therapy candidates for nAMD, also known as wet AMD.