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EdiGene to Present Data on Manufacturing Scale-up and Preclinical Development of its Leading Gene Editing Therapeutic Program to Treat Patients with β-Thalassemia Major

Time:19-11-07 09:07 Form: Null Views:2979

BEIJING, CHINA and CAMBRIDGE, MASSACHUSETTS, US – (Nov 19, 2019) – EdiGene, Inc., which develops genome editing technologies into novel therapeutics for a broad range of diseases and into creative solutions to advance drug discovery, today announced presentation of preclinical data on ET-01, its leading therapeutic program for patients with β-Thalassemia Major, during the 61st American Society of Hematology (ASH) Annual Meeting taking place in Orlando, FL on December 7-10th, 2019.


The presentation will highlight selected data from scaled-up manufacturing campaigns at clinical scale and IND-enabling preclinical studies of ET-01, in which autologous CD34+ cells are edited with high precision and efficiency, resulting in significantly higher fetal hemoglobin levels when differentiating into red blood cells. 

“In China, patients with β-Thalassemia Major have much higher morbidity burden and shorter life expectancy compared to those in Western countries,” said Dong Wei, Ph.D., MBA, CEO of EdiGene, “we are very excited about the progress of our ET-01 program, and plan to work with Chinese regulatory authorities in the near future to advance the program to clinics”.

Details of the presentation are listed below:


Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34+ Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major


Saturday, December 7, 2019, 5:30 PM - 7:30 PM, Hall B (Orange County Convention Center)

Session Name:

112. Thalassemia and Globin Gene Regulation: Poster I 

About EdiGene, Inc

EdiGene Inc is founded in 2015, and now headquartered in Beijing, with operational subsidiaries in Guangzhou, China and Cambridge, USA. 

Our mission is to translate the cutting-edge genome editing technologies into novel therapeutics for genetic diseases and cancer, and into creative solutions to advance drug discovery. We leverage our proprietary platforms to develop gene-editing therapies for a wide range of diseases, and to conduct high-throughput genome screening to enable dissection of functional big data in biological contexts.


Meng Wang

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