HuidaGene Achieves Milestone in Global CRISPR Patent Strategy with hfCas12Max® and Files IPO Prospectus

The CRISPR-Cas system is an adaptive immune system in bacteria and archaea that resists viral invasion. The Cas9 system has been developed into a gene-editing tool and is widely used in basic research, crop breeding, nucleic acid detection, gene therapy, and other fields.

Compared with the Type II Cas9 system, the Type V Cas12 system has a more diverse range of endonuclease subtypes from V-A to V-K. Among them, Type V-I Cas12 (i.e., Cas12i) is characterized by its smaller size, self-processing into mature crRNA, and recognition of TTN PAM sequences, offering advantages for in vivo delivery. Meanwhile, the Cas12 system is a novel CRISPR system that is distinct from Cas9 in terms of both evolution and taxonomy, and its use is considered not restricted by the CRISPR-Cas9 patent.

In November 2022, the research team of Hui-Gene Therapeutics published a research article titled "An engineered xCas12i with high activity, high specificity and broad PAM range" in the journal Protein & Cell. The Hui-Gene team, based on its HG-PRECISE...^®(HuidaGene - Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise) technology platform, utilizing technical means such as bioinformatics analysis, protein engineering, flow cytometry, next-generation sequencing, PEM-seq, and in vivo/in vitro gene therapy efficacy evaluation, conducted protein engineering modifications, screening, and validation on its independently developed xCas12i. This led to the development of a high-fidelity xCas12i variant with highly efficient targeting editing activity and extremely low off-target editing activity, named hfCas12Max.

By combining electroporation-mediated RNP delivery, targeted next-generation sequencing, and flow cytometry, researchers delivered hfCas12Max RNP to CD3.⁺T cells and achieve up to 90% TRAC gene knockout efficiencyRate。

High-efficiency and High-fidelity hfCas12Max in CD3⁺Evaluation of Effectiveness in T Cells^[1]

With its highly efficient targeted editing activity, extremely low off-target editing activity, and smaller molecular weight, which allows for production packaging and in vivo delivery via a single AAV vector or LNP, hfCas12Max has been applied by Hui-Gene Therapeutics to the development of in vivo gene therapies for various indications, including liver, neural, and muscular fields. At the May ASGCT Annual Meeting this year, Hui-Gene Therapeutics presented to the world the research progress of hfCas12Max gene-editing therapy in diseases such as Huntington's disease, ALS, Angelman syndrome, Duchenne muscular dystrophy, and hepatitis B.

hfCas12Max Invention Patent US11649444B1^[2]In May 2023, through the patent fast-track examination channel, it officially received authorization from the United States Patent and Trademark Office (USPTO) within just 9 months, achieving a breakthrough in the overseas layout of China's first foundational patent for the CRISPR-Cas12i system. The patent covers several novel Cas12i proteins and their variants independently developed by Hui-Gene Therapeutics, as well as their application in DNA editing, with Hui-Gene Therapeutics holding exclusive global rights to this foundational patent. Notably, hfCas12Max is a type of CRISPR-Cas system completely distinct from the CRISPR-Cas9 system, avoiding the layered patent blockades set by multiple patent holders in CRISPR-Cas9 technology. On the basis of its technical advantages, it provides strong intellectual property protection for the commercialization and global expansion of gene editing and gene therapy products.

In August 2023, Hui-Gene Therapeutics and Kactus Biosystems reached a strategic cooperation. hfCas12Max is based on Kactus Biosystems' SAMS raw enzyme.^TMDevelop and GMP production platform to achieve commercialization in the Greater China region, providing gene editing tool enzymes with ultra-high editing efficiency and extremely low off-target activity for scientific research, cell and gene therapy customers, ensuring the safety and efficacy of gene therapy drugs. Fully support customer projects in preclinical research, IND submission, clinical research, BLA, and market launch, promoting the development of the cell and gene therapy industry.

Kactus Biosystems has established a quality management system in accordance with the relevant requirements of GMP for pharmaceuticals and ISO13485:2016, strictly controlling the quality of GMP products to meet regulatory requirements for raw materials and excipients used in cell therapy. It provides complete documentation to support clients' clinical and marketing applications for their drugs. Kactus Biosystems has collaborated with nearly 100 gene editing companies domestically and internationally on GMP-grade enzymatic raw materials, accumulating extensive experience in assisting clients with drug applications and clinical-stage applications.

Currently, hfCas12Max products are in stock and available for inquiry.

Catalog Number:CAS-EE128(Click on the item number to view product details)

References: