Domestic first: Boya series because of gene editing therapy products ET-01 was approved into the clinic

"We are pleased that the ET-01 clinical trial application has been approved by the National Drug Administration and that Phase I clinical trials will be conducted as soon as possible.
. Wei Dong, chief executive officer of Boa's company, said, "This also marks the official clinical phase of the company's development."
we will continue to work on high-quality transgene editing technologies, continue to advance the development of the company's pipeline of other products, and bring more new treatment options to patients in China and around the world.
" ET-01 is a research and development phase, autophysic, in-body gene editing therapy products, the approved clinical trial is a multi-center, open, one-arm clinical study, the study will be blood transfusion-dependent β thalassemia patients to evaluate the safety and effectiveness of ET-01 single transplant.
in China, there are 300,000 people with medium- and severe thalassemia, and there are still huge unsealed medical needs for people with blood transfusion-dependent β three-thalassemia.
About ET-01 ET-01, a generic gene modification BCL11A red line enhancer of the in-body CD34 plus hematocyte injection, is in the research and development stage, self-contained, in-body gene-editing cell therapy products for the treatment of transfusion-dependent type β throme anemia.
ET-01 primary fluid is made from red line enhancers of the CRISPR/Cas9 system editing the BCL11A gene by collecting a single nucleocyte of the patient's self-mobilizing exosome blood.
about thalassemia Thalassemia refers to a group of hereditary hemolytic anemias that are partially or completely inhibited by the synthesis of the globin peptide chain due to a loss or point mutation of the globin gene.
Depending on the type of globin peptide chain synthesis disorder, thalassemia is generally classified as α thalassemia, β thalassemia, γ thalassemia, δ thalassemia, beta thalassemia and gamma beta thalassemia.
clinically, the most common cases are α thylamic anemia and β thalassemia, caused by a decrease in one of the two peptide chains (α or β) that make up hemoglobin (HbA, alpha2 beta2) in normal adults.
At the same time, according to the classification and clinical characteristics of the International Association of Anaemia, thalassemia can be classified as transfusion-dependent anemia (transfusion-dependentlassemia, TDT) and non-transfusion-dependent endemic anemia (non-transfusion-dependent thalassemia, NTDT), where people with blood transfusion-dependent anaemia need to rely on blood transfusions for life for life, if no blood transfusion is allowed, and early complications.
About EdiGene, Inc. is a clinically advanced biopharmaceutical company dedicated to accelerating drug research and developing innovative therapies for a wide range of genetic diseases and cancers through international cutting-edge genomic editing technologies.
Boya has established an in vitro cell gene editing treatment platform with independent intellectual property rights for hematopoietic stem cells and T-cells, an in vivo gene therapy platform based on RNA single base editing technology, and a high-volume genome editing and screening platform dedicated to targeted drug development.
founded in 2015, the company is headquartered in Beijing and has offices in Guangzhou and Cambridge, USA.
: 1.Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT) 3rd Edition: Thalassaemia International Federation (TIF); 2014.