Disclaimer: Due to limited expertise, errors are inevitable, and some information may not be the most up-to-date. Feel free to point out in the comments. This article is only an introduction to medical and health-related drugs, not a recommendation of treatment plans (if involved); it does not constitute any investment advice.
On April 22 (Eastern Time), the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) released online the abstracts of several latest research achievements selected for this year's conference. Focusing on gene and cell therapy, BRL Medicine’s BRL-101 research results have been successfully selected for the ASGCT Annual Meeting for the second consecutive year and will be presented in an oral report to disclose the latest clinical progress data."BRL-101 Autologous Hematopoietic Stem and Progenitor Cell Injection" is a gene therapy product targeting transfusion-dependent β-thalassemia.As early as August 16, 2022, the Investigational New Drug (IND) application for BRL-101 was officially approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA), advancing to the registration clinical trial stage.
The results announced at this meeting are from the Phase I IIT and IND clinical trials of BRL-101, with a total ofEnrolled 15 casesPatients aged 6-26 years: Clinical treatment results show that, compared with other gene therapies,BRL-101 gene therapy is more efficient, convenient, and safer, with significant advantages such as good targeting, high safety, broad scope of action, and remarkable therapeutic effects.。
BRL Medicine Inc. is committed to becoming a global leading cell and gene pharmaceutical company in the era of new commercial civilization. With the mission of "leading innovation with gene editing technology, developing breakthrough therapies, and benefiting all humanity," BRL Medicine relies on its self-developed center and the "Shanghai Gene Editing and Cell Therapy Research Center" co-built with universities. To date, it has generated more than 100 patent achievements. Five projects are conducting investigator-initiated clinical trials at eight renowned hospitals, three projects have been approved for IND, officially entering the registered clinical trial phase, and several other projects are in the IND application stage. BRL Medicine has established five proprietary technology platforms: a gene editing technology innovation platform, a hematopoietic stem cell platform, a non-viral targeted integration CAR-T platform, a universal cell platform, and an enhanced T-cell platform. It also operates a 7,000-square-meter GMP pilot production base and a nearly 200-person operations team, strongly ensuring that innovative research results can be rapidly transformed and applied.Source: BRL Medicine Announcement
