HuidaGene's First CRISPR RNA-Editing Therapy for nAMD Cleared by US FDA

HuidaGene Therapeutics (HuidaGene), a worldwide biotech company focused on genome medicines in clinical trials, revealed that the US FDA has approved its HG202 investigational new drug (IND) application for neovascular age-related macular degeneration (nAMD). HG202 is the initial clinical-stage CRISPR/Cas13 RNA-editing therapy and the sole clinical-stage therapy for nAMD that targets RNA.

“This open IND for HG202 by the US 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,” said Alvin Luk, Ph.D., M.B.A., C.C.R.A., co-founder and chief executive officer of HuidaGene.  “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, we dosed the world’s first novel CRISPR/Cas13 RNA-editing therapy in humans, and we recently presented preliminary data at the ARVO, ASGCT, EURETINA, and ESGCT this year. The rigor of our clinical data in China using a non-receptor binding pathway approach through Cas13 RNA editor to partially knock down the mRNA expression of VEGFA brings the potential to AMD patients.”

This phase 1 trial, called BRIGHT, aims to assess the safety and tolerability of CRISPR/Cas13-RNA therapy targeting VEGF-A mRNA in patients with nAMD, a condition impacting around 190 million individuals over 60 globally. The main focus of the clinical study is to assess the safety and tolerance of HG202 at various doses following a single dose administration. Additional outcomes measured include alterations in best-corrected visual acuity (BCVA), central retinal thickness (CRT), and the necessity for anti-VEGF rescue injections.