China's first clinical trial application for gene editing therapy was accepted

October 27th, a reporter from Edi Gene, a pioneer in the field of gene editing in China. Inc. was informed that the company announced the same day that China's State Drug Administration Drug Review Center has accepted its blood transfusion-dependent type β thalassemia gene editing therapy product ET-01 (receiving number: CXSL2000299), that is, CRISPR/Cas9 gene modification BCL11A red line enhancer auto-CD34 plus hematocyte injection clinical trial applications.
is the first clinical trial application for gene editing therapy accepted by a drug review center in China. According to reports, this clinical trial plans to evaluate the β and effectiveness of ET-01 single transplants in patients with blood transfusion-dependent patients with thalassemia.
ET-01, an autosomal CD34 plus hematocyte injection modified by the CRISPR/Cas9 gene modified BCL11A red line enhancer, is a product at the research stage for the treatment of transfusion-dependent type β thremia. Et-01 primary fluid is made by collecting a single nucleocyte of the patient's self-mobilized exosome blood, which is made from red line enhancers of the CRISPR/Cas9 system editing the BCL11A gene.
Previously, in 2018, Boya has established a cGMP standard clinical conversion application base for gene editing in Nansha District, Guangzhou, and released ET-01 large-scale production and preclinical safety and ability trial data at the 61st American Hematological Society (ASH) in 2019.
refers to a group of hereditary hemolytic anemias that are partially or completely inhibited by the synthesis of the globin peptide chain due to the loss or point mutation of the globin gene. Clinically, the most common cases are α thalt anemia and β thalassemia, caused by a reduction in one of the two peptide chains (α or β) that make up hemoglobin (HbA, alpha2 beta2) in normal adults.
according to the 2015 China Blue Book on Thalassemia, there are 30 million people living with the gene for thalassemia in China and 300,000 people with medium to severe thalassemia. β After birth, the disease of poor children in the land increased sexually, in addition to anemia symptoms, prone to spleen swelling, backward development and low immunity caused by the loss of multi-organ function 50% of heavy-duty poor patients died before the age of 5 years, if not effective treatment, rarely live beyond 20 years of age.
"We are pleased to see the company reach this important milestone and continue to move ET-01 forward into clinical trials." "We are committed to transforming cutting-edge gene editing techniques into transformative therapies that bring better treatment options to patients and the possibility of one-off cures for patients with some diseases." We look forward to the time when ET-01 clinical trials are licensed, and we expect our products to truly change the lives of patients and help them live healthier and longer lives. "
note that the field of gene editing has continued to be hot since 2013. Emmanuele Carpenter and Jennifer A. Doudna, two developers of CRISPR gene editing systems, also won this year's Nobel Prize in Chemistry.
in the past year and a half alone, at least 11 gene editing research and development projects have entered clinical development in the United States and the European Union, six of which are based on CRISPR gene editing systems. In the case of thalassemia treatment, in 2018, the biopharmaceutical company CRISPR Therapeutics and the US pharmaceutical company Vertex Pharmaceuticals' CTX001 received approval from U.S. and European regulators for new drug research applications, the world's first in-body CRISPR therapy clinical trial initiated by pharmaceutical companies, and is currently in phase I/II clinical trials.
In addition, Sangamo Therapeutics, the holder of gene editing technology ZFN, is using ZFN technology for thalassemia to repair hematopoietic stem cells, a project developed in collaboration with Sanofi subsidiary Bioverativ and now in the phase I/II clinical research phase.
was founded in 2015 and is headquartered in Beijing, with branches in Guangzhou and Cambridge, USA. According to its official website, Boya is a biopharmaceutical company dedicated to accelerating drug research for a wide range of genetic diseases and cancers and developing innovative therapies through international cutting-edge genomic editing technologies.
founder of The University of Beijing, is Wei Wensheng, a professor at Peking University's School of Life Sciences. Wei Wensheng, 51, was born in Jiangsu Province, received a bachelor's degree in biochemistry from Peking University in 1991 and a doctorate in genetics from Michigan State University in 1999 before going on to study postdoctoral studies at Stanford University School of Medicine, where he was taught by professor Stanley Cohen of
USA. Wei wensheng also serves as a researcher at Peking University's Biomedical Frontier Innovation Center (BIOPIC), Beijing Future Genetic Diagnostics High-Sharp Innovation Center (ICG) and Peking University-Tsinghua Joint Center for Life Sciences (CLS), as well as Director of the Genome Editing Research Center at Peking University.
it is worth mentioning that on October 13th Boya announced the completion of a Rmb450m round of B financing. This is the largest amount of financing in the domestic gene editing therapy research and development enterprises so far, and is also the first B round of financing. From August 2018 to the present, Boya has totaled RMB700 million in financing over the past two years.