Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
この試験で検証していること
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 ↗
試験結果が意味すること・意味しないこと
初期段階の結果は少人数の参加者と短い追跡期間から得られたものです。参加者が増え時間が経つにつれて変わる可能性があり、募集中・実施中・完了済みの試験であっても、治療効果が確立されたわけではありません。登録基準は厳格で、適格性の判断は試験実施施設が行うものであり、ウェブサイトが行うものではありません。