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On July 22, Sequence Biosciences (Shanghai), an innovative biomedical technology company focusing on new gene editing technology, announced that the clinical study of the base-editing drug "CS-101" for severe β-Eastern Mediterranean anemia carried out in cooperation with the First Affiliated Hospital of Guangxi Medical University successfully cured the first foreign patient, achieving continuous freedom from blood transfusion dependence for more than two months, and the total hemoglobin concentration stabilized to more than 120 g/L, and has returned to normal life, realizing the first gene editing cure of foreign patients in China.
Up to now, a number of patients with β-thalassemia have been free of transfusion dependence after CS-101 treatment, two of them have been transfusion-dependent for more than 6 months, and the longest has been more than 8 months. The therapy uses the base editing technology with independent intellectual property rights, and it only takes a few weeks to prepare, which can achieve the effect of "one treatment, lifelong cure", bringing hope for a complete cure for patients with β-hemoglobinopathies around the world.
Founded on December 27, 2021, with a registered capital of RMB 130 million, and the legal representative Wang Lijie, the company's business scope includes: general projects: technical services, technology development, technical consultation, technology exchange, technology transfer, technology promotion; natural science research and experimental development; medical research and experimental development; technology import and export; import and export of goods; import and export agency; biochemical product technology research and development; R&D and application of cell technology; software development; artificial intelligence application software development; software outsourcing services, etc.
The Lao patients who received positive albamine editing were transfusion-dependent patients with β-thalassemia and had a blood transfusion frequency of 2 units of red blood cells per month before receiving CS-101 treatment. The total hemoglobin concentration of the patient increased to more than 120 g/L and remained stable within two months after receiving CS-101 treatment, successfully achieving the goal of blood transfusion dependence.
The principle of CS-101 therapy is to collect patients' autologous hematopoietic stem cells and use the high-precision transformer base editor (TBE) independently developed by ShanghaiTech University (Wang et al., Nat Cell Biol, 2021) to perform precise base editing of the HBG1/2 promoter region in the patient's autologous hematopoietic stem cells, simulating the naturally occurring beneficial base mutations in healthy people. Reactivates γ-globin expression and reconstructs oxygen-carrying function of hemoglobin (Han et al., Cell Stem Cell, 2023). Further, the edited hematopoietic stem cells are infused back into the patient's body, so that the patient's own hemoglobin concentration reaches the level of a healthy person, so as to completely get rid of blood transfusion dependence.
Looking back at the development of gene editing technology:
Comparison of three gene editing technologies: CRISPR/Cas has obvious advantages
As a revolutionary gene editing technology, CRISPR/Cas has obvious advantages, compared with ZFNs and TALENs, CRISPR/Cas is much less difficult to design and construct, lower cost, shorter development cycle, and higher target modification efficiency.
-- Application of gene editing
Gene editing technology is mainly used in agriculture and disease treatment, among which, the application in agriculture is mainly crop breeding and livestock and poultry breed improvement, and in the field of disease treatment, it is mainly through the research and development of related drugs to achieve the purpose of treating patients.
Specifically, the application of gene editing technology in agriculture is relatively early, mainly because in the agricultural field, the regulatory environment is relatively relaxed, and does not involve ethical and moral risks, therefore, gene editing technology in the agricultural field has entered the stage of industrial application, and has been successfully applied to the breeding of new varieties with high yield and high resistance of tobacco, rice, wheat and other crops, as well as the improvement of economic livestock and poultry breeds such as pigs resistant to lethal viruses and cattle producing milk containing growth factors. In the field of disease treatment, gene editing technology has also shown great application potential. China has carried out a lot of research on the application of gene editing technology in biomedical research and development and disease treatment, and has made important breakthroughs in the treatment of reproduction, hereditary diseases, cancer, AIDS and other diseases, and applied CRISPR technology to gene editing in human embryos for the first time, successfully repairing the genes that cause β Eastern Mediterranean anemia in human embryos. At the same time, the mainland has also achieved certain results in the test of modifying pathogenic point mutations in human embryos.
-- Research and development of gene-editing drugs
According to the data of Yaorongyun, as of 2023, the research and development of gene editing drugs in mainland China includes 3 drugs under review and 4 projects in the clinical trial stage. Among them, the drugs in the review stage are PD-1 gene edited T cell injection, CD19 autologous gene editing T cell injection targeting interleukin 6 secretion function knockdown, and CD19 autologous gene editing T cell injection targeting interleukin 6 secretion function knockdown, and the projects in the clinical trial stage are the safety and efficacy study of multi-gene edited pig skin for deep burn wounds, and the targeted delivery of new hybrid exosomes CRISPR/ Cas9 gene editing MMP-13 in the treatment of cartilage defects, safety and efficacy evaluation of CCR5 gene in gene-edited human embryos, and safety evaluation of gene editing in human discarded embryos, mouse and monkey embryos.
According to the analysis of the Prospective Industry Research Institute, considering the downstream needs of the gene editing industry, the demand for updating the industry's technical means, and the development direction of the industry's hot technologies, the development trend of the gene editing industry in mainland China in the future is mainly reflected in the diversification and precision development of gene editing tools. Gene editing technology accelerates the efficiency of drug research and development, and the integrated development of machine learning and gene editing.
Prospective Economist APP Information Group
For more research and analysis of this industry, please refer to the "Market Prospect and Investment Strategic Planning Analysis Report of China's Genetic Modification Industry" by Qianzhan Industry Research Institute.
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