EdiGene Announces First Patient Dosed in Phase I Clinical Trial of ET-01, China’s First CRISPR-Based Gene-Edited Cell Therapy for Transfusion-Dependent Beta Thalassemia

BEIJING, China and CAMBRIDGE, Mass., Sept. 8, 2021 -- EdiGene announced today that its subsidiary, Guangzhou EdiGene, has completed the enrollment of the first patient in the multicenter Phase I registration clinical trial (Registration No.: CTR20211297) for ET-01, a hematopoietic stem cell gene-editing therapy for transfusion-dependent β-thalassemia, at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences. Additionally, site initiation has been completed at Nanfang Hospital, Southern Medical University in Guangzhou, which serves as the other principal investigator site for this clinical trial.

This clinical trial is a multi-center, open-label clinical study evaluating the safety and efficacy of ET-01 in the treatment of transfusion-dependent β-thalassemia. The principal investigators are Dr. Shi Jun, Professor and Director of the Regenerative Medicine Diagnosis and Treatment Center at the Institute of Hematology & Blood Diseases Hospital (Institute of Hematology, Chinese Academy of Medical Sciences), and Dr. Wu Xuedong, Professor of Pediatrics and Director of the Department of Pediatrics at Nanfang Hospital, Southern Medical University. Participating institutions also include Shenzhen Children's Hospital and Guangzhou Women and Children's Medical Center. For more information regarding this clinical trial, please visit the Drug Clinical Trial Registration and Information Publicity Platform of the Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA) at www.chinadrugtrials.org.cn.

“We are delighted to witness the initiation of the ET-01 multi-center Phase I registrational clinical trial and the enrollment of the first patient. We extend our sincere gratitude to the clinical experts, patients, and their families for their participation in and support of this study,” said Dr. Li Yun, Senior Vice President of Clinical R&D at Boya Jiyin (Beijing) Biotechnology Co., Ltd. “We will work closely with clinical experts to fully advance this clinical research, and we look forward to this pioneering gene-editing therapy soon benefiting patients with transfusion-dependent β-thalassemia.”

“With the successive activation of participating clinical sites and the enrollment of the first patient in the Phase I clinical trial of ET-01, we have moved one step closer to our goal of delivering a one-time curative therapy for patients with transfusion-dependent β-thalassemia,” said Dr. Wei Dong, Chief Executive Officer of Boya Ji Yin. “Building on our increasingly comprehensive industrial layout and global R&D strategy, we will continue to advance multiple gene-editing-based R&D pipelines targeting severe genetic diseases and cancer, bringing better treatment options to patients in China and globally.”

About ET-01

ET-01, an autologous CD34+ hematopoietic stem and progenitor cell injectable product featuring CRISPR/Cas9-mediated editing of the BCL11A erythroid enhancer, is an investigational ex vivo gene-edited cell therapy currently in the clinical development stage for the treatment of transfusion-dependent β-thalassemia. ET-01 is the first investigational gene-editing therapy and hematopoietic stem cell therapy product in China approved by the National Medical Products Administration (NMPA) for clinical trials.

Responsible Editor: Liuli

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