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EdiGene Announces IND Application for its CRISPR/Cas 9 Gene-editing Hematopoietic Stem Cell Therapy ET-01 in ?-thalassemia Accepted for Review by China National Medical Products Administration

EdiGene, Inc. ( "Company", or "EdiGene"), today announced the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has accepted for review the Company's Investigational New Drug (IND) application for ET-01, autologous CD34+ hematopoietic stem/progenitor cells with the erythroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9, an investigational gene-editing therapy for patients with transfusion dependent ?-thalassemia. EdiGene is a biotech company develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases.

"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 ?-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 ?-thalassemia patients today.

EdiGene launched a cGMP manufacturing facility in 2018 in Guangdong Province, and presented data on manufacturing scale-up and preclinical development of ET-01 in 2019 during the 62st American Society of Hematology (ASH) Annual Meeting.

About ET-01

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 gene-edited hematopoietic stem cell therapy for transfusion dependent ?-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.

About Thalassemia

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 ?-, ?-, ?-, ? -, ??-, or ????-thalassaemia. From a clinical point of view, the most relevant types are ?- and ?-thalassemia, resulting from the decrease of one of the two types of polypeptide chains (? or ?) that form the normal adult human hemoglobin molecule (HbA, ?2?2)1.

About EdiGene, Inc

EdiGene is a biotechnology company focused on leveraging the cutting-edge genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with subsidiaries in Guangzhou, China and Cambridge, Massachusetts, USA. More information can be found at www.edigene.com.


1Guidelines for the Management of Transfusion Dependent Thalassaemia(TDT) 3rd Edition: Thalassaemia International Federation(TIF); 2014.

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