SHANGHAI and CLINTON (NJ), December 19, 2023 – HuidaGene 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.

https://www.huidagene.com/new/news/51