EdiGene, Inc announced the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has approved the Company’s Investigational New Drug (IND) application for ET-01, an investigational CRISPR/Cas 9 gene-editing therapy for patients with transfusion dependent β-thalassemia. It marks the first gene-editing therapy and the first hematopoietic stem cell therapy IND application approval in China. EdiGene is a biotech company develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases. Previously, the company announced the acceptance of the IND by CDE on 27 October 2020.
“We are delighted that our IND application for ET-01 is approved by the regulatory authority and we’ll roll out phase I clinical trial soon,” said Dong Wei, Ph.D., CEO of EdiGene. “We are embarking on a new journey as a clinical-stage company. We will continue to translate cutting-edge gene-editing technology and advance our pipeline, with a goal of providing better choices for patients in China and around the world.”
ET-01 is an investigational, autologous, ex vivo gene-editing therapy. The approved trial of ET-01 is a multicenter, open-label, single arm study designed to assess its safety and efficacy in transfusion dependent β-thalassemia patients. In China, it is estimated that there are over 300 thousand patients with thalassemia major or thalassemia intermediate. Serious unmet medical needs remain for transfusion dependent β-thalassemia patients today.
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 produced by getting autologous mobilized peripheral blood mononuclear cells, enriching CD34+ cells and editing BCL11A erythroid -specific enhancer using CRISPR/Cas9 system.
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-thalassaemia. From a clinical point of view, the most relevant types are α- and β-thalassemia, resulting from the decrease of one of the two types of polypeptide chains (α or β) that form the normal adult human hemoglobin molecule (HbA, α2β2).
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.