" class="no-js "lang="en-US"> HuidaGene Therapeutics Gene-Editing System Granted Patent
Monday, June 05, 2023

HuidaGene Therapeutics’ Novel DNA Gene-Editing System Cas12i Patent Granted by USPTO

HuidaGene Therapeutics, a clinical-stage company focusing on developing gene editing tools and gene therapies, today announced that the United States Patent and Trademark Office (USPTO) has granted to the Company patent US11,649,444B1 with respect to its independently-developed DNA Gene editing system, CRISPR-Cas12i (Cas12Max®), in just 9 months post-filing with the assistance of a reputable US law firm. Cas12Max® has the highest DNA editing efficiency in mammalian cells compared with Streptococcus pyogenes Cas9 (SpCas9) and Lachnospiraceae bacterium Cas12a (LbCas12a). The grant of this US patent represents the first oversea breakthrough of CRISPR-Cas12i systems developed in China. This fundamental patent covers multiple novel Cas12i proteins, and their variants and DNA editing uses thereof. HuidaGene exclusively owns its global rights.

“After granting the CRISPR-Cas13X/Y (13e/f) patents in both China and US, grant of the first overseas patent of Cas12i is another important milestone for HuidaGene, which further strengthens the Company’s intellectual property position,” said Xuan Yao, Ph.D., Co-Founder and Chief Executive Officer of HuidaGene. “With our commitment to developing novel CRISPR systems, the Company has carried out global patent layout of the core technology and established the Company’s dominant position in gene editing tool and gene therapy development. The strong intellectual property protection for our gene editing tools would allow more independent gene editing products to be commercialized globally.”

CRISPR RNA and CRISPR-associated (Cas) protein constitute an RNA-guided adaptive immune system found in bacteria and archaea for the defense of virus infection. Class 2, Type II (CRISPR-Cas9) system has been transformed into one of the most powerful gene-editing tools used globally for biomedical research that has therapeutic potential against incurable genetic disorders by modifying their DNA sequences. However, DNA double-strand breaks (DSBs)-related genotoxicities due to formation of DSBs by Cas9 and DNA repair, including apoptosis, chromosome deletions, and chromosomal rearrangements, remain to be the major concern in clinical development. The in-vivo delivery of the CRISPR-Cas9 system for efficient gene editing is also challenging due to its large size. Additionally, various Cas9 patents owned by multiple patentees and continuous litigations are likely to remain unresolved for years to come, suggesting many uncertainties to commercial development.

Class 2, Type V (CRISPR-Cas12) system is an emerging CRISPR system different from Class 2, Type II (CRISPR-Cas9) system in both evolution and classification, and thus it is believed that its use is not limited to Cas9 patents. The Company filed the patent based on the November 2022 publication describing the novel engineered xCas12i with high activity, high specificity, and board protospacer adjacent motif (PAM) range.

“On 15 November 2022, my team published a research paper online of the Protein & Cell titled ‘An engineered xCas12i with high activity, high specificity and board PAM range’,” said Hui Yang, Ph.D., Co-Founder, Chief Scientific Advisor, and Chairman of the SAB at HuidaGene. “My team has identified 10 novel Cas12i proteins through AI and deep ML of DNA sequencing and assembly prediction from metagenomic database followed by the identification of xCas12i through the fluorescent reporting system. Through HGPRECISE® (HuidaGene – Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise) platform, we further engineered xCas12i system to develop a high-fidelity xCas12i variant (Cas12Max®), which is smaller than Cas9 allowing it to be packaged and delivered in vivo through a single AAV vector and has the highest editing efficiency in mammalian cells with strong fluorescence intensity in compared with SpCas9 and LbCas12a.”

At present, HuidaGene has applied Cas12Max® to the development of in vivo gene therapy for various indications in neurology, neuromyology, and hepatology. HuidaGene will continue to improve the CRISPR-based gene-editing technology and bring safe gene-editing therapies a reality for millions of patients worldwide.

  1. Providence Therapeutics Announces Partnership with University Health Network (UHN) for mRNA Therapeutic Discovery and Development Read more
  2. Affini-T Therapeutics Appoints Industry Veteran Thaminda Ramanayake, M.S., MBA, as Chief Business Officer Read more
  3. Feinstein Institutes Bioelectronic Medicine Researchers Stimulate Vagus Nerve to Reduce Bleeding in Hemophilia Read more
  4. Kyverna Therapeutics Granted FDA Fast Track Designation for KYV-101 in Lupus Nephritis Read more
  5. Arcturus Therapeutics Receives U.S. FDA Fast Track Designation for ARCT-810, mRNA Therapeutic Candidate for Ornithine Transcarbamylase Deficiency Read more