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An angel round of financing of RMB 40 million has been completed.

In recent years, with the advent of the era of gene therapy, gene editing technology has shown positive effects in clinical phase I.

Then, existing gene editing technologies, such as CRISPR, ZFN, TALEN, generate targeted double-strand breaks in DNA, and then rely on the cell’s own repair mechanism to complete the editing process, thereby effectively destroying gene expression. However, their lack of control over editing results, off-target effects and reliance on DNA double-strand breaks may lead to unpredictable chaos in cells after gene editing.

In this process, many genome mutations occurred in a single base. In order to make gene editing more precise, base gene editing technology came into being. It aims to target these single base errors (point mutations) without causing double-strand breaks in DNA, and can perform precise base repair on DNA. Compared with other existing gene editing technologies, Base gene editing technology is expected to achieve higher editing efficiency and better safety.

The recently learned Positive Sequence (Shanghai) Biotechnology Co., Ltd. (hereinafter referred to as: Positive Sequence Bio) is a company focusing on the use of next-generation gene editing Technology (base editing-based) start-up company for the treatment of human genetic diseases/rare diseases, recently it has completed an angel round of financing of RMB 40 million, led by Wanwu Capital and Lianxin Capital, Sequoia Capital, Taifu Capital participated in the investment and signed a global exclusive perpetual license agreement with ShanghaiTech University for a patent portfolio totaling more than 170 million yuan.

Understood. Currently, more than 75,000 gene mutations are known to cause human diseases, such as thalassemia and Duchenne muscular dystrophy. The vast majority of genetic diseases caused by genetic mutations have no cures. For a small number of these diseases, patients often need to take medications for life and bear the side effects and psychological pressure brought by the drugs. Among them, about half of genetic diseases are caused by single-base mutations, which are difficult to repair with traditional gene editing tools.

In this regard, the base editing technology of positive sequence organisms can not only accurately and permanently change a single base pair in a huge genome, but also according to different diseases. The free combination of mutation sites provides new possibilities for the cure of genetic diseases caused by point mutations and the immunotherapy of cancer.

According toIt is reported that Positive Sequence Biology and Shanghai University of Science and Technology have signed a global exclusive perpetual license agreement for multiple base editing system underlying platform independent patents, including: Enhanced base editor with high product purity(enhanced BE, Cell Res, 2017), Cpf1 base editor that can effectively edit the A/T-enriched region of the genome(dCpf1-BE, Nat Biotechnol, 2018a), Universal base editor for efficient editing in G/C enriched regions and hypermethylated regions(hA3A-BE, Nat Biotechnol, 2018b) and Does not activate DNA damage response pathway Cas12a base editor(BEACON, Cell Rep, 2020) etc., Positive Sequence Biology will use these base editing systems with independent intellectual property rights to create new disease treatment methods.

Looking at the entire world, companies that commercialize base editing technology are currently scarce. Only a few companies, such as Beam Therapeutics and Sprout Gene, are also in Early development. Positive Sequence Biology also wants to rely on the team’s research and development strength to fill the gap in China’s use of new base editing systems for gene therapy in the field of gene editing.

Core team introduction

Positive Sequence Biology is composed of Professor Chen Jia, School of Life Sciences, Shanghai University of Science and Technology, Professor Yang Li, Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences, Professor Yin Hao, Wuhan University Medical Research Institute, and Co-founded by Professor Yang Bei from the Institute of Immunochemistry, Shanghai University of Science and Technology.

• Dr. Chen Jia is a gene editing tool development expert, National Excellent Youth, Shanghai Pujiang Talent, and a young backbone of the Chinese Academy of Sciences Molecular Cell Excellence Center. He has published in Cell, Nat Biotechnol, Nat Struct Mol Biol, Trends Biochem Sci, etc. He has several papers and has been interviewed by Cell and other magazines. He is mainly responsible for the continuous research and development of new gene editing tools. He is also one of the core inventors of the underlying platform-based independent patent of Positive Sequence.

• Dr. Yang Li is a bioinformatics expert, a distinguished professor of Shanghai University of Science and Technology, a national outstanding youth, and a leading talent in the Ten Thousand Talents Program. He is in Cell, Science, Nat Biotechnol, Nat Struct Mol Biol, Mol Cell, Genome Res, etc. The journal has published many papers, mainly responsible for the exploration of new gene therapy targets and the genome-wide safety verification of base editing tools. It also uses big data analysis to provide technical guidance for the positive sequence research and development new pipeline.

• Dr. Yin Hao is a drug delivery expert and a national high-level overseas introduced talent. He has served as a researcher in well-known multinational pharmaceutical companies Vertex Pharmaceuticals and Alnylam Pharmaceuticals, and worked in Nature, Nat Biomed Eng, Nat Rev Drug Disc, etc. The journal has published many papers and is mainly responsible for the delivery of base editing tools and follow-up clinical experimental research.

• Dr. Yang Bei is a protein engineering expert and Shanghai Pujiang talent. He has published papers in Nat Struct Mol Biol, PNAS, Sci Adv and other journals. He is mainly responsible for the structure and function of the key components in the base editing tool. Research and improve editing efficiency through protein engineering methods and provide more possibilities for subsequent delivery.