ORIGINAL RESEARCH article
Front. Cell Dev. Biol.
Sec. Stem Cell Research
Volume 13 - 2025 | doi: 10.3389/fcell.2025.1607707
Preclinical Efficacy and Safety Evaluation of BD211 Autologous CD34 + Hematopoietic Stem Cell Injection for Transfusion-Dependent β-Thalassemia in NCG-X Mice
Provisionally accepted
- 1Department of Toxicology, School of Public Health, Peking University,China, Beijing, China
- 2SAFE Pharmaceutical Technology Co. Ltd, Beijing, China
- 3BDGENE Therapeutics, Shanghai, China
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Introduction: Autologous CD34 + hematopoietic stem cell-based therapies have shown promise in addressing therapeutic needs. However, a comprehensive evaluation of their efficacy and safety is crucial before clinical application. This study aimed to assess the efficacy and safety profile of BD211 autologous CD34 + hematopoietic stem cell injection in NCG-X mice. Methods: NCG-X mice were administered BD211 intravenously at doses of 4.0×10 5 and 1.2×10 6 cells per mouse, followed by withdrawal and observation for 13 weeks. Efficacy was evaluated by monitoring the engraftment and differentiation of BD211 into human erythroid cells within the mouse bone marrow and blood. Safety was assessed through clinical observation, pathology, organ weight measurements, and histopathology. Toxicokinetic studies and distribution of BD211 were determined via validated quantitative PCR. Results: Mortality was observed in all groups of mice with no correlation to dose or BD211. No abnormal effects related to BD211 administration on clinical responses, body weight, or food intake were observed. BD211 successfully engrafted and differentiated into human erythroid cells within the mouse bone marrow and blood. Conclusions: The no observed adverse effect level of BD211 was established at 1.2×10 6 cells per mouse. BD211 shows potential as a safe therapeutic approach for treating transfusion-dependent thalassemia.
Keywords: Transfusion-dependent β-thalassemia, BD211, NCG-X Mice, No-observed-adverseeffect level, Toxicokinetic, distribution
Received: 08 Apr 2025; Accepted: 28 May 2025.
Copyright: © 2025 Dai, Li, Chen, Huang, Ling, Wang and Wang. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Sikai Ling, BDGENE Therapeutics, Shanghai, China
Quanjun Wang, SAFE Pharmaceutical Technology Co. Ltd, Beijing, China
QI Wang, Department of Toxicology, School of Public Health, Peking University,China, Beijing, China
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.