BEIJING & CAMBRIDGE, Mass. – EdiGene, Inc. (“Company”, or “EdiGene”), today announced the first patient enrolled in multicenter Phase I clinical study of its investigational gene-editing hematopoietic stem cell therapy ET-01 for patients with transfusion dependent β-thalassemia at the Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, and activation of the investigational site at Nanfang Hospital of Southern Medical University in Guangzhou, Guangdong Province. EdiGene is a global, clinical-stage biotechnology company focused on translating gene editing technologies into transformative therapies for patients with serious genetic diseases and cancer.
The NMPA approved clinical trial of ET-01 is a multicenter, open-label study designed to assess its safety and efficacy in transfusion dependent β-thalassemia patients. The co-lead Principal Investigators of the trial are Jun Shi, M.D., Ph.D., Professor, Director of Regenerative Medicine Clinic Center, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC and Xuedong Wu, M.D., Professor of Pediatrics, Director of Departments of Pediatrics, Nanfang Hospital of Southern Medical University. The other participating clinical sites are Shenzhen Children’s Hospital and Guangzhou Women and Children’s Medical Center. Additional details are available on www.clinicaltrials.gov (NCT04925206).
“We are delighted about activating these investigational sites and enrolling the first patient, and are grateful for the investigators, patients and their families who are involved in or supportive to the clinical study,” said Yun Li, M.D., Senior Vice President of Clinical Development, EdiGene. “We will continue our collaboration with the investigators to advance the clinical study, and we look forward to bringing cutting-edge gene-editing therapy to patients with transfusion dependent β thalassemia soon.”
“The site activation and first patient enrollment mark a significant step towards bringing the potential one-time cure for patients with transfusion dependent β thalassemia.” said Dong Wei, Ph.D.，CEO of EdiGene, “As the company is scaling up and expanding its global presence, we will continue to advance our gene-editing focused pipeline for patients with severe genetic diseases and cancer, and bring better therapeutic solutions to patients in China and worldwide.”
ET-01 refers to autologous CD34+ hematopoietic stem/progenitor cells with the elytroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9. It is an investigational, autologous, ex vivo gene-edited hematopoietic stem cell therapy for transfusion dependent β-thalassemia patients. ET-01 is the first gene-editing experimental therapy and the first hematopoietic stem cell experimental therapy with IND application approval by NMPA in China.
Thalassemia refers to a group of blood diseases characterized by decreased or absent synthesis of normal globin chains. According to the chain whose synthesis is impaired, the thalassemia is classified as α-, β-, γ-, δ -, δβ-, or εγδβ-thalassemia. From a clinical point of view, the most relevant types are α- and β-thalassemia. Based on the thalassemia syndromes ’clinical severity and transfusion requirement, they can be classified into two main groups: Transfusion Dependent Thalassemia (TDT) and Non-Transfusion Dependent Thalassemia (NTDT). The TDT requires regular blood transfusion to survive and without adequate transfusion support, they would suffer several complications and a short life span.
About EdiGene, Inc
EdiGene is a global, clinical-stage biotechnology company focused on translating gene editing technologies into transformative therapies for patients with serious genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China and Cambridge, Massachusetts, USA. More information can be found at www.EdiGene.com.