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HuidaGene Receives Orphan Drug Designation from FDA for HG302 for the Potential Treatment of Duchenne Muscular Dystrophy after Receiving Rare Pediatric Drug Designation

2024.01.23 09:00
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SHANGHAI and CLINTON (NJ), January 23, 2023 – HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company developing potentially curative genomic medicines, has been granted Orphan Drug Designation (ODD) by the U.S. FDA for HG302 to treat Duchenne muscular dystrophy (DMD). The ODD supplements the rare pediatric disease designation (RPDD) previously granted by the FDA for HG302.

HuidaGene identified novel Cas12i proteins through the HG-PRECISE® platform to develop the high-fidelity Cas12i variant (hfCas12Max®). This hfCas12Max® exhibits the highest on-target editing efficiency and lowest off-targeting activity in mammalian cells compared to widely used SpCas9 or other Cas12a/b/e systems while packaging into a single viral vector. Cas12i was granted a patent by the USPTO with strong intellectual property protection.

“While HG302 is the Company’s third program receiving both ODD and RPDD, these designations are important milestones in advancing our CRISPR-based therapeutic approach for patients suffering from rare, serious, or life-threatening disorders,” said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and CEO of HuidaGene.

The FDA grants ODD to support the development of medicines for rare diseases affecting fewer than 200,000 people in the U.S. Potential benefits of the ODD include accelerated approval review, 7-year post-approval market exclusivity, the federal tax credit for qualified clinical research expenses incurred in the U.S., and a waiver of Prescription Drug User Free Act (PDUFA) fees (currently worth over $4 million).

About HG302

HG302 is a novel, first-in-class CRISPR DNA-editing therapy with hfCas12Max® and CRISPR RNA targeting human DMD exon 51 splice-donor (SD) site delivered by one viral vector into the muscles. The human DMD exon 51 SD site will be disrupted by HG302, thereby allowing exon 51 skipping to form the correct open reading frame from exons 50 to 53, restoring functional dystrophin protein expression and improving muscle functions.

About HuidaGene

HuidaGene utilizes its proprietary HG-PRECISE® platform to discover, engineer, and develop CRISPR-based genomic medicine. The Company is advancing clinical programs of HG004 in RPE65-associated inherited retinal disease (granted ODD and RPDD), HG202 CRISPR RNA-editing in neovascular age-related macular degeneration, and the preclinical pipeline, including CRISPR RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (granted ODD and RPDD), CRISPR DNA-editing for Duchenne muscular dystrophy (granted ODD and RPDD), Amyotrophic Lateral Sclerosis (ALS), and Huntington’s Disease (HD). HuidaGene’s extensive intellectual property portfolio positions it as a leader in unleashing the full potential of genome medicines for neurology and ophthalmology. Learn more at or on LinkedIn.