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HuidaGene Announces Rare Pediatric Drug Designation Granted to HG302, A Novel CRISPR DNA-editing Therapy, for the Treatment of Duchenne Muscular Dystrophy

2023.12.19 20:00
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·    HG302 effectively generates stable genome editing with minimal off-target effects in-vitro and in-vivo

·    A single intravenous injection of HG302 restores functional dystrophin protein expression

·    HG302 demonstrates therapeutic effects by improving muscle functions in humanized DMD mice


SHANGHAI and CLINTON (NJ), December 19, 2023HuidaGene Therapeutics (辉大基因“HuidaGene”) today announced the U.S. FDA granted Rare Pediatric Drug Designation (RPDD) to HG302, a novel CRISPR-Cas12 DNA-editing therapy, to treat Duchenne muscular dystrophy (DMD), affecting 1 in ~5,000 newborn boys.


Unlike the in-vivo use of Cas9 system with limited genome-editing efficiency, high occurrence of off-targeting modifications, viral delivery packaging limitations, and complicated CRISPR-Cas9 patent dispute, HuidaGene identified novel Cas12i proteins using AI and deep machine-learning of DNA sequencing through the proprietary HG-PRECISE® platform to develop high-fidelity Cas12i variant (hfCas12Max®). This hfCas12Max® is smaller in size than Cas9, thereby packaging it using one viral vector with the lowest off-targeting activity and highest editing efficiency in mammalian cells when compared to SpCas9 and LbCas12a. Furthermore, Cas12i was granted a patent by USPTO with a cleaner commercial landscape.


“This is Company’s third program receiving RPDD, suggesting the recognition of the importance of DMD and the preclinical safety and efficacy of hfCas12Max gene-editing therapy by FDA,” said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and CEO of HuidaGene. “DMD patients are often wheelchair-bound before the age of 12 facing a significant unmet need. HG302 is designed specifically to target the DMD gene and enable the production of functional dystrophin protein. Time is muscle; we look forward to advancing HG302 into the clinic as soon as possible.”


The RPDD is granted for rare medical diseases affecting fewer than 200,000 people in the US and primarily for individuals aged under 18. If the HG302 Biologic Licensing Application (BLA) for DMD is approved by the FDA, HuidaGene may be eligible to receive a priority review voucher (PRV) that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred this high-value PRV. PRVs have historically commanded prices over US$ 100 million.


About HG302

HG302 is a novel CRISPR DNA-editing therapy with high-fidelity Cas12 (hfCas12Max) and CRISPR RNA targeting human DMD exon 51 splice-donor (SD) site delivered by a single 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 - 辉大基因 

HuidaGene Therapeutics (辉大基因), a global clinical-stage biotechnology company, utilizes its CRISPR-based HG-PRECISE® platform to discover, engineer, and develop potentially curative genomic medicine. The Company is advancing clinical programs of HG004 in RPE65-associated inherited retinal disease (granted both ODD and RPDD), HG202 CRISPR RNA-editing in neovascular age-related macular degeneration, and the preclinical pipeline, including HG301 CRISPR DNA-editing in retinitis pigmentosa, HG204 CRISPR RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (granted both ODD and RPDD), and HG302 CRISPR DNA-editing for Duchenne muscular dystrophy (granted RPDD) etc. The Company’s extensive intellectual property portfolio positions it as a leader in unleashing the full potential of genome medicine. Learn more at huidagene.com or on LinkedIn.