The first in the country! Boya was approved for clinical treatment of thalassemia in the case of crispr/Cas 9 gene β gene editing therapy

On January 18, Boya announced that China's State Drug Administration's Drug Review Center had approved its clinical trial application for CRISPR/Cas9 gene editing therapy product ET-01 for blood transfusion-dependent β thremia, the first gene editing therapy product and hematopoietic stem cell product approved by the State Drug Administration to conduct clinical trials.
, on October 27, 2020, the Drug Administration announced that it had formally accepted its clinical trial application for ET-01.
ET-01, a generic self-contained CD34 plus hematocyte injection called CRISPR/Cas9 gene modification BCL11A red line enhancer, is an in-development, self-contained, in-body gene-edited cell therapy product for the treatment of transfusion-dependent type β threetic 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.
ET-01 clinical trial is a multi-center, open,single-arm clinical study that will evaluate the safety and effectiveness of ET-01 single transplants in patients with blood transfusion-dependent type β thalt-thalassemia.
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."
"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.
" with regard to thalassemia thalassemia refers to a group of hereditary hemolytic anemia diseases that are partially or completely inhibited by the synthesis of the globin peptide chain due to the 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 thalassemia (TDT) and non-transfusion-dependent anemia (non-transfusion-dependentlassemia, NTDT), where people with blood transfusion-dependent anemia need to rely on blood transfusions for life and die early if they do not.
About EdiGene, Inc. is a biopharmaceutical company that is in the clinical stage and 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.
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