Boya Ji completes the initiation of Phase I clinical trial for the treatment of β-thalassemia gene editing therapy and the first patient enrolled in the group, Dr. Mai Meng broke the news

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, September 8, 2021, Boya Gene announced its subsidiary, Guangzhou Gene, a hematopoietic stem cell gene editing therapy product ET for transfusion-dependent β-thalassemia -01's multi-center phase I registered clinical trial (registration number: CTR20211297) has completed the first patient enrollment at the Hematology Hospital of the Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences), another major study of this clinical trial The Nanfang Hospital of Southern Medical University in Guangzhou, the institution where the author is located, has also completed the start-up of the research center
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Boya Gene is a global biomedical company in the clinical stage focusing on the transformation of gene editing technology, dedicated to the research and development of innovative therapies for genetic diseases and cancers that are difficult to cure
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This clinical trial is a multi-center, open clinical study evaluating the safety and effectiveness of ET-01 in the treatment of transfusion-dependent β-thalassemia.
The main investigator is the Hematology Hospital of the Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences).
Dr.
Shi Jun, the director and professor of the Medical Clinic, and Wu Xuedong, the director of the Pediatrics Teaching and Research Section of Nanfang Hospital of Southern Medical University, and the professor of Pediatrics Wu Xuedong.
The participating institutions also include Shenzhen Children's Hospital and Guangzhou Women and Children's Medical Center
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For more information about this clinical trial, please visit the Drug Clinical Trial Registration and Information Disclosure Platform of the Drug Evaluation Center of the State Drug Administration (
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"We are very pleased to see the initiation of the ET-01 multi-center phase I registered clinical trial and the enrollment of the first patient.
We are very grateful to the clinical experts, patients and patient families who participated in and supported this study
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" Senior Vice President Li Yun, MD, said, “We will work with clinical experts to fully advance this clinical research and look forward to cutting-edge innovative gene editing therapies that can benefit patients with transfusion-dependent β-thalassemia as soon as possible
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” “With the development of ET-01 Participating institutions for phase I clinical trials have successively started and the first patients have been enrolled.
We are furthering our goal of bringing a one-time cure for patients with transfusion-dependent β-thalassemia
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" said Dr.
Wei Dong, Chief Executive Officer of Boya Jiyin, "We Based on the company's increasingly complete industrial layout and global R&D layout, it will continue to advance multiple R&D pipelines based on gene editing technology for serious genetic diseases and cancer, and bring better treatment options for patients in China and even the world
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"About ET -01ET-01, the autologous CD34+ hematopoietic stem progenitor cell injection of CRISPR/Cas9 gene-modified BCL11A erythroid enhancer, is an autologous, in vitro gene-edited cell therapy research product in the clinical development stage, used to treat blood transfusion dependent type Beta thalassemia
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ET-01 is China's first gene editing therapy and hematopoietic stem cell therapy research product approved by the State Food and Drug Administration for clinical trials
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About Thalassemia Thalassemia refers to a group of hereditary 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
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According to the different types of globin peptide chain synthesis disorders, thalassemias are generally divided into alpha thalassemia, beta thalassemia, gamma thalassemia, delta thalassemia, delta beta thalassemia and epsilon delta beta thalassemia
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Clinically, the most common are alpha thalassemia and beta thalassemia
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According to the classification and clinical characteristics of the International Thalassaemia Association, thalassemia can be divided into transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT), and transfusion-dependent thalassemia (NTDT).
Patients with thalassaemia need to rely on blood transfusion for life.
If blood transfusion is not given, serious complications and early death will occur
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About EdiGene (EdiGene, Inc.
) is a clinical-stage global biomedical company focusing on the transformation of gene editing technology, dedicated to the research and development of innovative therapies for genetic diseases and cancers that are difficult to cure
.

Boya Gene has established an in vitro cell gene editing therapy platform for hematopoietic stem cells and T cells with independent intellectual property rights, an in vivo gene therapy platform based on RNA single-base editing technology, and a high-throughput genome editing dedicated to the development of targeted drugs Screening platform
.

Boya Jiyin was founded in 2015 and is headquartered in Beijing, with offices in Guangzhou, Shanghai and Cambridge, USA
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Article source: Boya Jiyin "Boya Jiyin treatment of β-thalassemia gene editing therapy research product ET-01 multi-center phase I registered clinical trial completed the clinical initiation of the main investigator’s institution and the first patient enrollment" statement: This article involves content It is only used to explore the cutting-edge progress of biomedicine and does not constitute any medical guidance.
If necessary, please go to a regular hospital for treatment
.