From lab to global stage: Reforgene advances international patent portfolio for its novel CasRm gene editing system

Reforgene's proprietary, efficient, safe and compact novel CasRm gene editing system (derived from the CRISPR-Cas13 family), which holds independent intellectual property, has seen its PCT patent application (Application No. PCT/CN2023/115093) successfully enter China and the United States, with plans to expand to more than 10 countries or regions in the future. The patent family has global freedom-to-operate (FTO) value and covers CasRm and its mutants, homologous proteins, and various RNA editing applications.

Building on its original TIPBAIG general technology platform, Reforgene mines genomic big data through machine learning algorithms, and combines AI-driven protein structure analysis with downstream systematic molecular biology applications to enable high-throughput discovery and optimization of CRISPR-Cas nucleases and related functional proteins for gene editing. CasRm is a novel CRISPR-Cas13 gene editing tool that is highly efficient, has an extremely low off-target rate and a compact structure, with excellent RNA-targeting editing activity. In particular, its editing efficiency on multiple RNA targets surpasses that of CasRx (RfxCas13d), the high-activity tool currently used by mainstream U.S. research institutions. In addition, CasRm is about 70 amino acids smaller than CasRx, further improving flexibility in drug design and making it more suitable for AAV delivery. With significant advantages on multiple fronts, CasRm-based gene editing medicines show better potential in efficacy and safety.

Gene editing technology has broad application prospects in the life sciences. The CasRm system can be widely applied in human disease drug development, virus clearance, gene function research, transcriptomics research, RNA labeling and detection, disease diagnosis, crop improvement, veterinary drug development, synthetic biology and biosensor design, among other fields. Reforgene is currently applying the CasRm system to drug development for multiple diseases, including age-related macular degeneration, and multiple research results have been presented at the 27th ASGCT Annual Meeting and the IAPRD 2024 Congress.

Based on the development of the CasRm system, Reforgene has built an extensive patent portfolio around its underlying technology and drug targets, currently holding 9 patent families and a total of 19 patent applications, including 6 PCT applications, providing strong intellectual property protection for the commercialization of gene editing technology.

As one of the few innovative drug companies worldwide with underlying CRISPR patent technology, Reforgene has achieved notable results in gene editing tool innovation. The entry of the CasRm patent application into China and the United States is another major breakthrough in Reforgene's technological innovation, further expanding its intellectual property portfolio in the gene editing field. To date, Reforgene holds 69 invention patent families worldwide, involving 136 invention patent applications and 18 granted patents, covering Cas9, Cas12, Cas13 and base editing systems, forming a comprehensive and robust intellectual property network.

About Reforgene

Reforgene Medicine is one of China's leading innovative gene editing drug companies, driven by gene editing technology and dedicated to bringing novel medicines to life. Reforgene has a high-caliber team that applied gene editing to explore the treatment of genetic diseases at an early stage internationally, and has accumulated pioneering achievements in hematology, ophthalmology and other disease areas, with strong technical capabilities in gene editing tool innovation, drug development strategy, novel animal model construction, and multi-level efficacy and safety assessment, operating both in vivo and ex vivo drug development. Reforgene's mission is to deliver accessible, curative medicines for patients with serious diseases through innovative genetic technologies. The company currently has pipeline programs across genetic diseases, complex diseases and oncology. Its innovative beta-thalassemia medicine has achieved globally leading clinical progress, and it achieved the world's first cure of an alpha-thalassemia patient.