It is reported to be approximately 15,000 to 30,000 patients with β-Thalassemia Major (β-TM) in China. A recent cross-sectional survey initiated by the Institute of Hematology & Blood Diseases Hospital found that only 7% of the samplings are expected to live beyond age 18.

The presentation highlights data from an investigator initiated trial of ET-01 performed at the Institute of Hematology and Blood Diseases Hospital of the Chinese Academy of Medical Sciences and Peking Union Medical College involving one subject with TDT.

The study demonstrated that a single dose of ET-01 consisting of gene-edited autologous CRISPR/Cas9 mediated CD34+ cells resulted in timely engraftment, a significant and durable increase in the level of fetal hemoglobin production, and achieved transfusion independence for 15 months since three months after ET-01 infusion at the time of data cut-off by May 20, 2022. The safety profile of ET-01 was consistent with that of autologous hematopoietic stem cell transplantation and myeloablative conditioning. These preliminary data support further testing of ET-01 treatment for patients with TDT.

“I am glad to see that the patient, who needed a transfusion of red blood cells every two weeks, has been transfusion free since February 2021. We are conducting additional research to further elucidate how gene-edited hematopoietic stem cells engraft and differentiate, hoping to shed light on the implication of gene editing in the short and long term,” said Dr. Jun Shi, Director of the Regenerative Medicine Clinic at the Institute of Hematology and Blood Diseases Hospital of the Chinese Academy of Medical Sciences and Peking Union Medical College and the first presenter of the study.

“These data preliminarily validate ET-01’s safety profile and its robust and potentially lasting clinical benefits to patients,” said Dong Wei, Ph.D., CEO of EdiGene. “Leveraging our expertise in gene editing and in-depth knowledge in the field of hematopoietic stem cells, we’re working to advance the development of ET-01 with the hope of bringing the potential one-time cure for patients with transfusion dependent β-thalassemia.”

ET-01 is currently in a multi-center Phase I clinical trial in China, with subjects’ enrollment completed earlier this year.

Details of the presentation

Title: Preliminary Safety and Efficacy Results of EDI001: An Investigator Initiated Trial on CRISPR/Cas9-Modified Autologous CD34+ Hematopoietic Stem and Progenitor Cells for Patients with Transfusion Dependent β-Thalassemia
Publication Number: 4778
Session Name: 801. Gene Therapies: Poster III
Session Date and Time: Monday, December 12, 2022, 6:00 PM – 8:00 PM (ET)
Location: Ernest N. Morial Convention Center, Hall D
Presenter: Dr. Jun Shi, Director of the Regenerative Medicine Clinic at the Institute of Hematology & Blood Diseases Hospital of the Chinese Academy of Medical Sciences & Peking Union Medical College

About ET-01

ET-01 is an autologous CD34+ hematopoietic stem/progenitor cells with the erythroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9. ET-01 is the first gene-editing experimental therapy and the first hematopoietic stem cell experimental therapy with IND application approval by China National Medical Products Administration (NMPA).

About EdiGene, Inc.

EdiGene Inc. is a global, clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPER™-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T. 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