The Center for Drug Evaluation (CDE) of China‘s National Medical Products Administration (NMPA) has recently approved EdiGene’s application for ET-01, an investigational CRISPR-Cas9 gene editing therapy for patients with transfusion dependent β-thalassaemia.
This marks the first gene editing therapy and the first hematopoietic stem cell therapy IND application approval in China, where it is estimated that there are over 300 thousand patients with thalassaemia major or thalassaemia intermedia.
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-thalassaemia patients.
“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. 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,” said Dong Wei, Ph.D., CEO of EdiGene.
Read Edigene’s Full Press Release HERE