EdiGene Announces Completion of Last Patient Infusion in Phase I Clinical Trial of ET-01 for Transfusion-Dependent Beta Thalassemia

Beijing, China and Waltham, Massachusetts, USA, November 18, 2022,Boya Jiyin Announces Its Gene-Edited Hematopoietic Stem Cell Investigational Product for Transfusion-Dependent Beta-ThalassemiaET-01MulticenterThe Phase I clinical trial has completed the infusion of the last patient. Boya jiyin(Beijing)Biotechnology Co., Ltd. is a global biopharmaceutical company at the clinical stage, focusing on the transformation of gene editing technology, and is committed to developing transformative therapies for unmet clinical needs.

“ET-01"The infusion of the 8th and final patient has been completed in the multicenter Phase I clinical trial, marking another significant milestone in our development of a potential one-time curative therapy for transfusion-dependent β-thalassemia patients," said Dr. Wei Dong, CEO of Boya jiyin (Beijing) Biotechnology Co., Ltd. "We are deeply grateful to all the patients, their families, and the researchers involved in this clinical trial; their contributions have played a crucial role in advancing this Phase I clinical trial. The results of this multicenter Phase I clinical trial will demonstrate E."T-01Regarding the safety and efficacy in specific patient populations, we plan to engage with regulatory authorities on I in 2023.IDiscussion on the Strategy and Trial Protocol of Clinical Trials, Aiming for ET-01"to benefit Chinese patients with transfusion-dependent β-thalassemia as early as possible."

β-Thalassemia is a hereditary hemolytic anemia disease. The standard therapy for transfusion-dependent β-thalassemia is lifelong blood transfusion.And iron chelation therapy. ET-01 aims to achieve a one-time functional cure through a single infusion of CRISPR/Cas9 gene-edited autologous hematopoietic stem and progenitor cells.

This Phase I clinical trial is a multi-center, open-label clinical study designed to evaluate a single dose of ET-01In patients with transfusion-dependent β-thalassemiaSafety and Effectiveness, with a total of 8 subjects enrolled. Previously, in September 2021, the company announced that the institution of the principal investigator had completed the clinical trial initiation and the enrollment of the first patient.

ET-01, namely CRISPR/Cas9 gene editingBCL11AAutologous Erythroid EnhancerCD34+ Hematopoietic Stem and Progenitor Cell Injection for the treatment of transfusion-dependent β-thalassemia. ET-01 is the first gene-editing therapy and hematopoietic stem cell therapy product in China to be approved by the National Medical Products Administration (NMPA) for clinical trials.

Boya jiyin(Beijing)Biotechnology Co., Ltd. is a global biopharmaceutical company at the clinical stage, focusing on the transformation of gene editing technology, and is committed to developing transformative therapies for unmet clinical needs.

Based on its scientific research and technological strength in gene editing technology, high-throughput genomic editing screening, and bioinformatics, the company is advancing the development of internationally leading in vitro and in vivo gene editing therapies. The company has established multiple therapeutic platforms, including an in vitro hematopoietic stem cell therapy platform, an in vivo RNA base editing platform, and an in vitro universal CAR-T therapy platform.