Shanghai Bendao Gene Technology Co., Ltd. (hereinafter referred to as Bendao Gene), a world-leading gene therapy development company with original delivery technology, recently successfully completed the A round of financing of 60 million yuan. This financing was led by Huakong Fund. Men Capital, ETP Fund, Inno Angel Fund and other institutions followed the investment, and Probe Capital acted as the exclusive financial advisor. The funds will be used for preclinical and clinical development of multiple products, laboratory and team building and many other aspects. Bendao Gene previously received strategic investment from Saifu Pharmaceuticals in June 2019, and received tens of millions of yuan in the pre-A round of Triumph Ventures in March 2020.
Gene therapy has become the third most revolutionary therapy, but delivery technology remains the biggest bottleneck
    For hereditary, infectious and degenerative diseases that are incurable or refractory to drugs, gene therapy has the characteristics of "one-shot cure". In recent years, gene therapy has become the third most revolutionary therapy after chemical drugs and antibody drugs.
       After the first appearance of the third-generation gene editing technology CRISPR in 2012, the first in vivo clinical trial of CRISPR in March 2020, and the milestone events such as CRISPR winning the Nobel Prize in Chemistry in October 2020, gene editing technology is becoming more and more mature. , but delivery technology has been the biggest hurdle in the field of gene-editing therapeutics. Nobel laureate Jennifer Doudna wrote in Nature magazine in January 2020 that"Delivery may remain the biggest bottleneck for gene-edited somatic cell therapy"（Delivery remains perhaps the biggest bottleneck to somatic-cell genome editing）。
      In the past, the world's clinically proven gene therapy delivery technologies mainly include three platforms: adeno-associated virus, lentiviral vector, and nanocomposite materials. These platforms have certain drawbacks for gene editing delivery. An ideal gene editing delivery tool needs to be both transient and efficient to ensure the safety and efficacy of the treatment.
       Dr. Cai Yujia, the founder of Bendao Gene, successively studied under the tutelage of Professor Thomas Perlmann, Professor of Karolinska Institutet in Sweden, Secretary General of the Nobel Prize Committee, and Professor Jacob G. Mikkelsen of Aarhus University in Denmark, and obtained a doctorate degree in gene therapy. After returning to China, he served as a researcher at the Institute of Systems Biomedicine, Shanghai Jiao Tong University, mastered the core technology of gene therapy vectors, and has rich experience in the engineering design and transformation of gene therapy viral vectors. In recent years, the company has successfully developed a human clinically validated gene therapy delivery platform——VLP-mRNA（Virosomes mRNA）,deliver CRISPR/Cas9 mRNA to achieve safe and controllable in vivo gene editing therapy.
      Successfully completed in early November 2020 World's second clinical study of in vivo CRISPR gene editing therapy(In March 2020, Editas' Zhang Feng team completed the world's first case),This is the world's first clinical study of CRISPR antiviral therapy.This delivery technology delivers Cas9 mRNA through the lentiviral shell, which combines the advantages of high efficiency of viral vector infection of cells (in vitro infection rate of 99.8%, in vivo infection rate > 50%) and transient mRNA expression (degradation within 72 hours). The therapeutic effect has been achieved in the animal model of CRISPR, and it has the technical advantages of transient expression in vivo, no long-term safety risks associated with AAV expression of CRISPR, no obvious off-target, and low immunogenicity. At present, under the demonstration and permission of the Ethics Committee of the Eye and ENT Hospital Affiliated to Fudan University, the team of Professor Hong Jiaxu is presiding over the initial safe and controllable clinical application research based on this technology.In the future, this technology will be extended to the treatment of other viral infections, ophthalmic diseases and neurological diseases.
   Dr. Cai Yujia has been devoted to the development of gene therapy delivery technology for more than 10 years, and has successfully developed a number of original delivery technology platforms such as virosome CRISPR/Cas9 mRNA gene editing delivery technology and next-generation lentivirus delivery technology. The company's original viral keratitis treatment products have perfect pre-clinical animal experimental data, and are currently conducting non-registered human clinical trials in top eye hospitals in China. At the same time, the company has deployed multiple product pipelines such as gene editing technology for the treatment of hereditary, infectious and degenerative diseases, viral vector treatment for blood diseases, and mRNA vaccines.