HuidaGene and Kactus Announce Strategic Collaboration and License Agreement to Promote Commercialization of Next-Generation Gene Editing Enzyme, hfCas12Max®

SHANGHAI and CLINTON (NJ), August 4, 2023 – HuidaGene Therapeutics (辉大基因, "HuidaGene"), a clinical-stage biotech company focused on developing CRISPR-based gene editing tools and genomic medicines, and Kactus Biotechnology (恺佧生物, "Kactus Bio"), a world-leading biotech company specialized in providing high-purity, high-activity, recombinant protein and enzyme products, today announced a strategic collaboration and license agreement for manufacturing and sales of the enzyme hfCas12Max^®. Under the terms of the agreement, Kactus Bio will obtain an exclusive license for manufacturing and sales of HuidaGene's hfCas12Max^® in Greater China, as well as the non-exclusive license for manufacturing and sales of GMP-grade products while HuidaGene will receive sales-based payments. This strategic cooperation with Kactus Bio is one of the important milestones of HuidaGene to accelerate the commercialization of hfCas12Max^® and continue to transform new gene editing technology bringing new breakthrough genome medicines to patients in Greater China.

High-fidelity Cas12 (hfCas12Max^®) is a new DNA gene editing system independently-developed through HuidaGene's HG-PRECISE^® (HuidaGene – Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise) platform. hfCas12Max^® has demonstrated superior on-target editing efficiency and lower off-target editing activity when compared to widely used CRISPR-Cas9, Cas12a, Cas12b and Cas12e. With its small size for packaging into a single AAV, hfCas12Max^® has potential in broad applications in biomedical research, agriculture, cell and gene therapy, and other fields. The publication entitled "An engineered xCas12i with high activity, high specificity and broad PAM range" was published in Protein & Cell journal in November 2022. In May 2023, the United States Patent and Trademark Office (USPTO) granted hfCas12Max^® invention patent US11,649,444B1 through the patent fast-track review channel in just 9 months. It is not only the first granted global patent on CRISPR-Cas12i system originally innovated from China, but it is also a breakthrough step on providing solid IP protection for the commercialization of novel gene editing therapies from China to the global. 

Kactus Bio has established the quality management system based on applicable GMP and ISO13485:2016 requirements, demonstrating strict controls in monitoring, maintaining, and controlling GMP manufacturing processes. The processes meet the applicable regulatory requirements of raw and excipient materials for gene and cell therapy. Kactus Bio has provided GMP-grade enzymes to nearly 100 gene editing companies both in China and aboard with accumulated rich experience in consistently assisting customers for clinical supply and regulatory applications ranging from IND to BLA. 

The GMP manufacturing and commercialization of hfCas12Max^® will rely on Kactus Bio's Structure Aided Design and Multiplex Screening (SAMS^TM), a proprietary R&D and GMP manufacturing platform. Kactus Bio and HuidaGene join forces to provide this novel gene editing tool enzymes with ultra-high editing efficiency and extremely low off-target activity for customers to support the development of gene and cell therapy.

"Kactus Bio has rich experience in providing GMP-grade enzymes for gene and cell therapy use. Kactus Bio has established a trusted GMP production and quality management system, which has been highly recognized by the industry. We are thrilled to have reached a strategic collaboration with Kactus Bio in Greater China, which is an important step of HuidaGene's hfCas12Max^® global commercialization." said Xuan Yao, Ph.D., Co-founder, President, and General Manager of Greater China of HuidaGene, "By leveraging each other's advantages, we believe that we can make hfCas12Max^® a better choice for partners who need gene-editing tools and bring more world-leading innovative gene editing technologies to meet unmet medical needs." 

"HuidaGene has a solid foundation and world-leading capabilities in the development of gene editing tools. We are more than happy to reach a commercial collaboration with HuidaGene in manufacturing and sales of hfCas12Max^®. This collaboration will technically rely on Kactus Bio's validated SAMS^TM R&D and GMP production platform." commented by Mr. Wang Gang, the Founder and CEO of Kactus Bio, "We are confident that we can work together to accelerate the research-use and commercial-use of hfCas12Max^® and contribute to novel gene and cell therapy development in China."

About HuidaGene -辉大基因  

HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patients' needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. We are currently advancing clinical programs in RPE65 mutation-associated inherited retinal dystrophies and our preclinical pipeline, including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy, and MECP2 duplication syndrome. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. HuidaGene is committed to transforming the future of genome-editing medicine. 

For more information, please visit http://www.huidagene.com  

or follow us on LinkedIn at http://www.linkedin.com/company/huidagene 

About Kactus Bio -恺佧生物

Founded in early 2018, Kactus Bio is an innovation-driven, protein raw material company focusing on the upstream process of biologics. Relying on SAMS^TM, a proprietary protein R&D and production platform, Kactus Bio provides GMP-grade Cas9 enzymes meeting the clinical needs of gene editing in China, GMP-grade totipotent nucleases for AAV and mRNA vaccine industries, and raw enzymes for cell therapy and mRNA vaccine manufacturing. Among them, Cas9 enzyme, universal nuclease, and a full range of mRNA related enzymes for in vitro synthesis which have completed drug master filing (DMF). The GMP products of Kactus Bio strictly follow the Pharmacopoeia of analytical procedures and methods validation, equipped with manufacturing execution systems (MES)-integrated digital manufacturing management system with good batch-to-batch product stability and consistency, ensuring to meet the release requirements.