Clinical trials of gene editing therapy have been approved β of thalassemia
Last Update: 2021-01-22
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News () Reporters from the domestic gene editing pioneer EdiGene, Inc. was informed that on January 18, the company announced that China's State Drug Administration Drug Review Center has approved its application for a clinical trial of THE CRISPR/Cas9 gene editing therapy product ET-01 for blood transfusion-dependent β thalassemia.
is the first gene editing therapy product and hematopoietic stem cell product approved by the State Drug Administration to conduct clinical trials.
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.
, On October 27, 2020, Boya announced that the Drug Review Center of the State Drug Administration of China had officially accepted its clinical trial application for ET-01.
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. The ET-01 original solution 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 after the CD34 plus cell population is collected.
"We are pleased that the ET-01 clinical trial application has been approved by the National Drug Administration and that Phase I clinical trials will be conducted as soon as possible." "This also marks the official clinical phase of the company's development, " said Dr. Wei Dong, CHIEF Executive Officer of Boya. We will continue to work on high-quality transgene editing technologies, continue to advance the development of other company product pipelines, and bring more new treatment options to patients in China and around the world. prior to
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-thalassemia 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.
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 the 2020 Nobel Prize in Chemistry. In the treatment of thalassemia, biopharmaceutical company CRISPR Therapeutics and U.S. pharmaceutical company Vertex Pharmaceuticals, holders of gene editing technology ZFN Sangamo Therapeutics and Sanofi subsidiary Bioverativ are both in clinical stages.
founder of The University of Beijing, is Wei Wensheng, a professor at Peking University's School of Life Sciences. Wei received his bachelor's degree in biochemistry from Peking University in 1991 and his ph.D. in genetics from Michigan State University in 1999 before going on to do postdoctoral research at Stanford University School of Medicine, where he was taught by professor Stanley Cohen of
America. Wei wensheng also serves as a researcher at Peking University's Biomedical Frontier Innovation Center (BIOPIC), Beijing Future Gene Diagnostics High-Sophisticated 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.
addition, on October 13, 2020, 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.
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