Boyage completes first patient enrollment due to CRISPR/Cas9 gene editing therapy

Text|Pharmaceutical Mission Hills

On September 8, Boya Gene announced that its subsidiary Guangzhou Gene has a multi-center phase 1 registered clinical trial of the hematopoietic stem cell gene editing therapy product ET-01 for blood transfusion-dependent β-thalassemia.

Thalassemia refers to a group of inherited hemolytic anemia diseases in which the synthesis of globin peptide chain is partially or completely inhibited by the deletion or point mutation of the globin gene

According to the press release, a number of gene-edited hematopoietic stem cell clinical trial applications (IND) have been approved by regulatory agencies around the world, and they are currently in the clinical stage.

ET-01's generic name is CRISPR/Cas9 gene modified BCL11A erythroid enhancer autologous CD34+ hematopoietic stem progenitor cell injection.

The clinical trial conducted this time is a multi-center, open clinical study evaluating the safety and effectiveness of ET-01 in the treatment of transfusion-dependent β-thalassemia.