Base-Editing Therapy Achieves Transfusion Independence Across Ethnicities
Shanghai – September 12, 2026 -- CorrectSequence Therapeutics (Correctseq) reported that its transformer Base Editor (tBE) therapy CS-101/CS-206 delivered durable clinical remission in patients with sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) across four distinct genetic backgrounds, according to a peer-reviewed study published in Cell Stem Cell on September 7, 2026.
The study, conducted with multiple research institutions, expands on earlier data showing five Chinese TDT patients treated with CS-101 all achieved transfusion independence. The new cohort includes four patients from Nigeria, Laos, Malaysia and Pakistan -- one with SCD and three with TDT -- covering genotypes βS/βS, β0/βE, and two β0/β0 variants with different mutation types.