CRISPR/Cas gene editing systems
Gene editing is increasingly applied across biomedicine. As a key technology of molecular biology, gene editing is not only a core means of drug development, but has also rapidly developed into a highly promising class of innovative medicines.
Foundational technology
CRISPR/Cas gene editing systems are a landmark innovative foundational technology in molecular and cellular biology. By pairing a nuclease protein with a matched guide RNA, researchers can efficiently and precisely modify target sequences in the genome. Cas9 and Cas12 achieve precise, permanent cut-based changes at target genomic sites through the cell’s own homologous recombination or non-homologous end joining mechanisms. Base editing can inactivate gene function by changing a single base in the target gene. Epigenetic editing can silence or inactivate an entire gene through epigenetic modification strategies, and can also activate gene expression; its durability depends on the design of such editors.
A paradigm shift and the editing toolbox
Compared with earlier gene augmentation approaches, CRISPR/Cas technology offers simplicity, efficiency, broad adaptability and strong scalability, and is driving a paradigm shift in therapies and medicines. CRISPR systems comprise multiple classes, and their clinical value is expanding from genetic diseases to prevalent major diseases, including Cas9, Cas12, Cas13 and more derived editing tools. CRISPR/Cas can edit at both the genomic and transcriptional levels. The gene editing toolbox has been greatly refined and extended, covering applications such as gene knockout or silencing, knock-in or fragment integration, and gene activation.
Curative potential and proprietary tools
CRISPR/Cas gene editing medicines hold immense potential and curative promise, applicable not only to genetic diseases such as thalassemia and inherited retinal diseases, but also to complex diseases including cardiovascular and metabolic diseases, infectious diseases, neurodegenerative diseases and malignant tumors. In terms of innovation in gene editing systems and related technology modules, we own a portfolio of proprietary CRISPR gene editing tools covering Cas12, Cas13, base editing and epigenetic editing, with independent drug development capabilities at both the DNA and RNA levels. We will continue to invest in developing new editing tools with more proprietary intellectual property and iterative optimization, ensuring excellent clinical efficacy and safety, guided by the world’s enormous unmet clinical needs, to develop highly competitive gene medicines for patients in urgent need of treatment.