Huidagene Therapeutics
SHANGHAI and CLINTON, NJ, April 20 /PRNewswire/
HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage company focused on the development of gene editing tools and gene therapies, announced today that the United States Patent and Trademark Office (USPTO) has granted the Company has granted patent US11,649,444B1 for its independently developed CRISPR-Cas12i (Cas12Max®) DNA editing system within just nine months of filing with a reputable US law firm. Cas12Max® has the highest DNA editing efficiency in mammalian cells compared to Streptococcus pyogenes Cas9 (SpCas9) and the Lachnospiraceae-Bakterium Cas12a (LbCas12a). The issuance of this US patent is the first overseas breakthrough of CRISPR Cas12i systems developed in China. This fundamental patent covers several novel Cas12i proteins and their variants and their use for DNA editing. HuidaGene exclusively owns the worldwide rights.
“Following the issuance of the CRISPR-Cas13X/Y (13e/f) patents in China and the US, the issuance of the first overseas patent for Cas12i is another important milestone for HuidaGene, which further reinforces the company’s intellectual property position strengthens,” said Xuan Yao, Ph.D., Co-Founder and Chief Executive Officer of HuidaGene. “With our commitment to the development of novel CRISPR systems, the company has patented the core technology worldwide and has a dominant position in the development of gene-editing tools and gene therapies. Strong intellectual property protection for our gene editing tools would allow more independent gene editing products to be commercialized worldwide.”
The protein associated with CRISPR RNA and CRISPR-associated (CAS) forms an RNA-directed adaptive immune system that is used in bacteria and archaea to fight off viral infections. The Class 2, Type II (CRISPR-Cas9) system has emerged as one of the most powerful gene-editing tools used in biomedical research worldwide, with therapeutic potential to treat incurable genetic disorders by altering DNA sequences owns. However, the main concern in clinical development is the genotoxicities associated with DNA double-strand breaks (DSBs), which result from the formation of DSBs by Cas9 and DNA repair, including apoptosis, chromosomal deletions and chromosomal rearrangements. The in-vivo Deploying the CRISPR-Cas9 system for efficient gene editing also poses a challenge due to its large size. In addition, various Cas9 patents are held by multiple patent holders and ongoing litigation is likely to remain unresolved for years to come, posing many uncertainties for commercial development brings.
The Class 2 Type V (CRISPR-Cas12) is an emerging CRISPR system that differs from the Type 2 II (CRISPR-Cas9) system in both development and classification. Therefore, it is believed that its use is not limited to Cas9 patents.
The company applied for the patent based on the November 2022 publication in which the novel engineered xCas12i will be high activity, high fidelity and in the Protospacer Adjacent Motif (PAM) range. “On November 15, 2022, my team published a research paper online in the journal Protein & Cell titled, “An engineered xCas12i with high activity, high specificity and board PAM range,” explained Hui Yang, Ph.D., co-founder, scientific director and chair of the SAB at HuidaGene. “My team has gone through 10 new Cas12i proteins AI and Deep ML of DNA sequencing and assembler prediction identified from the metagenomic database, followed by identification of xCas12i by the fluorescence reporting system.Using the HGPRECISE® (HswimGene – Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Epertise) platform, we have advanced the xCas12i system to create a high-fidelity xCas12i variant (Cas12Max®) that is smaller than Cas9, allowing it to be packaged in a single AAV vector and live can be administered and which has the highest editing efficiency in mammalian cells with strong fluorescence intensity compared to SpCas9 and LbCas12a.”
Currently, HuidaGene has Cas12Max® to develop a in-vivo -Gene therapy used for various indications in neurology, neuromyology and hepatology. HuidaGene will continue to advance CRISPR-based gene editing technology and make safe gene editing therapies a reality for millions of patients worldwide.
Information on the HGPRECISE® platform
HuidaGenes HGPRECISE H-swimGene – (P lattform for R ational Engineering of CRISPR-Cas Identification by Synergic Expertise) by HuidaGene enables rapid discovery of Cas proteins using artificial intelligence (AI) and deep machine learning (ML) of DNA sequencing and assembly prediction from the metagenomic database. HGPRECISE® Generated Cas12i protein (Cas12Max®) has demonstrated superior on-target editing efficiency and lower off-target editing activity compared to widely distributed CRISPR-Cas9 as well as Cas12a, Cas12b, and Cas12e. In addition, Cas12Max® is relatively small compared to SpCas9 and Cas12a (1033-1093 aa) and has a broad preference for the 5′ TN protospacer adjacent motif (PAM). With its high on-target editing efficiency, low off-target editing activity, and small packaging size in a single AAV vector, Cas12Max® offers many opportunities and perspectives for biomedical research, agriculture, gene therapy, and other fields .
About HuidaGene – Huida Gene
HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focused on the discovery, engineering and development of gene editing tools and gene therapies to reshape the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to serving the needs of patients around the world with various preclinical therapy programs in the fields of ophthalmology, otology, myology and neurology. HuidaGene is currently driving clinical programs for RPE65 mutation-associated inherited retinal dystrophies and various preclinical pipelines including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy and MECP2 duplication syndrome. These CRISPR-based therapeutics offer the potential to heal patients with life-threatening diseases by addressing the root cause of their disease. HuidaGene aims to change the future of genome editing in medicine.
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