Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Qué está evaluando este ensayo
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 ↗
Qué significa y qué no significa el resultado de un ensayo
Los resultados de fases tempranas provienen de un número reducido de participantes y un seguimiento breve. Pueden cambiar con más personas y más tiempo, y un ensayo en reclutamiento, activo o incluso completado no ha demostrado que un tratamiento funcione. Los criterios de inscripción son estrictos; la elegibilidad la decide el centro del ensayo, nunca un sitio web.