The Chinese biotechnology company EdiGene announced yesterday that the first patient was enrolled in the multicentre, Phase I clinical study of its investigational gene editing hematopoietic stem cell therapy ET-01 for patients with transfusion-dependent β-thalassaemia (TDT).
Clinical sites of the approved trial of ET-01 are currently the Nanfang Hospital of Southern Medical University, the Shenzhen Children’s Hospital, and the Guangzhou Women and Children’s Medical Center, China.
“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.
Dong Wei, Ph.D., CEO of EdiGene, added that as the company is scaling up and expanding its global presence, it will continue to advance its gene-editing-focused pipeline for severe genetic diseases, aiming at bringing 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 Investigational New Drug (IND) application approval by the National Medical Products Administration (NMPA) in China.