Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
What this trial is testing
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 ↗
What a trial result does and does not mean
Early-phase results come from small numbers of participants and short follow-up. They can change with more people and more time, and a trial that is recruiting, active or even completed has not established that a treatment works. Enrolment criteria are strict; eligibility is decided by the trial site, never by a website.