Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
本试验正在测试什么
A trial of a base-editing treatment for sickle cell disease, which installs a change that keeps fetal haemoglobin switched on. Dosing has finished in all the adults and adolescents enrolled, and the company has said it may apply for approval as early as the end of 2026.
A Phase 1/2 study of ristoglogene autogetemcel (risto-cel, formerly BEAM-101), an autologous base-edited CD34+ cell therapy inducing fetal haemoglobin, in patients with sickle cell disease and severe vaso-occlusive crises. Dosing is complete in all adult and adolescent participants, with updated data expected by the end of 2026.
Sources
- Beam Therapeutics · 2026
Beam Therapeutics second quarter 2026 results ↗
试验结果意味着什么，又不意味着什么
早期试验结果来自少量参与者和较短的随访周期，随着人数增加和时间延长，结论可能发生变化。正在招募、进行中甚至已完成的试验，并不代表某种疗法已被证实有效。入组标准十分严格，是否符合条件由试验机构决定，而非由网站判断。