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On January 18, Boya Gene announced that the Center for Drug Evaluation of the National Medical Products Administration of China had approved its clinical trial application (IND) for the CRISPR/Cas9 gene editing therapy product ET-01 for blood transfusion-dependent β-thalassemia. This is also The first gene editing therapy product and hematopoietic stem cell product approved by the State Food and Drug Administration for clinical trials in China.
Prior to this, Boya Jiyin announced on October 27, 2020 that the Food and Drug Administration officially accepted its clinical trial application for ET-01. It is worth noting that, as an innovative treatment with no precedent in China, the IND application of Boya Jiyin will be approved at one time within 60 working days specified by the regulations.
“We are very pleased that the ET-01 clinical trial application has been approved by the State Food and Drug Administration, and the phase I clinical trial will be launched as soon as possible.” said Dr. Wei Dong, Chief Executive Officer of Boya Gene. “This also marks the company’s formal development In the clinical stage. We will continue to work on high-quality transformational gene editing technology, continue to promote the company's other product pipeline research and development process, to bring more new treatment options for patients in China and the world."
According to reports, ET-01 is an autologous, in vitro gene editing therapy product in the research and development stage. The clinical trial approved this time is a multi-center, open, single-arm clinical study. To evaluate the safety and effectiveness of ET-01 single transplantation in patients with transfusion-dependent β-thalassemia. In China, there are 300,000 patients with thalassemia medium and severe, and patients with transfusion-dependent beta thalassemia still have huge unmet medical needs.
The approval of the IND application for β-thalassemia gene therapy products means that Boyak has successfully moved closer to the international first-line echelon. Currently, the CTX001 pipeline under CRISPR Therapeutics is in phase I/II clinical trials, and similar products under Intellia Therapeutics are also in phase I clinical trials in the United States, and the pipeline of Editas Medicine is expected to enter phase I clinical trials this year.