Τhe Chinese biotechnology company EdiGene announced yesterday that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has accepted for review 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-thalassaemia (TDT).
“We are happy to achieve this important milestone and bring ET-01 closer to clinical-stage,” said Dong Wei, Ph.D.CEO of EdiGene. “We are committed to translating cutting-edge gene-editing technologies into transformative therapies so as to bring patients better choices, and for some, a potential one-time cure. We look forward to receiving approval from NMPA and initiating ET-01 clinical studies in the near future.”
The trial of ET-01 is designed to assess its safety and efficacy in transfusion dependent β-thalassemia patients. In China, it is estimated that there are 30 million thalassemia gene carriers, and over 300 thousand patients with thalassemia major or thalassemia intermediate. Serious unmet medical needs remain for transfusion-dependent β-thalassemia patients today.
ET-01 is EdiGene’s most advanced candidate. EdiGene makes ET-01 by editing autologous CD34+ cells using CRISPR/Cas9 to disrupt the BCL11A erythroid enhancer. In doing so, EdiGene aims to elevate fetal haemoglobin to levels needed to ease the clinical symptoms of β-thalassaemia.