Cell Stem Cell: tBE-mediated Base Editing Therapy Achieves Durable Clinical Remission in Sickle Cell Disease and β-Thalassemia Across Different Genetic Backgrounds

Following 100% transfusion independence in Chinese TDT patients, new study confirms tBE is equally safe and effective for African SCD patients and TDT patients from South/Southeast Asia.

SHANGHAI, Sept. 8, 2026 /PRNewswire/ -- On September 7, 2026, Shanghai, Cell Stem Cell published online clinical research from CorrectSequence Therapeutics (Correctseq) in collaboration with multiple institutions in a paper titled "Clinical base editing for β-hemoglobinopathies across different genetic backgrounds", demonstrating that CS-101/CS-206—base-editing therapy developed with the transformer Base Editor (tBE)—achieved consistent efficacy and safety in β-hemoglobinopathy patients of diverse genetic origins. This follows the team's prior clinical report on five Chinese transfusion-dependent β-thalassemia (TDT) patients treated with CS-101, all achieving transfusion independence (Lai et al., Nature, 2026). The new study extends treatment to four additional patients from Nigeria, Laos, Malaysia, and Pakistan—one with sickle cell disease (SCD) and three with TDT. All achieved rapid hematopoietic reconstitution, sustained high-level pan-cellular HbF expression, complete transfusion independence or freedom from vaso-occlusive crises (VOCs), with no detectable off-target edits, or product-related adverse events.