CorrectSequence Therapeutics

Shanghai – September 12, 2026 -- CorrectSequence Therapeutics (Correctseq) reported that its transformer Base Editor (tBE) platform achieved sustained transfusion independence or elimination of vaso-occlusive crises (VOCs) in four new patients from Nigeria, Laos, Malaysia and Pakistan, extending clinical validation beyond the Chinese cohort where five patients previously achieved full transfusion independence.

Study published in Cell Stem Cell confirms cross-genetic efficacy of CS-101/CS-206

The peer-reviewed paper, published online September 7, 2026, details results from one sickle cell disease (SCD) patient and three transfusion-dependent β-thalassemia (TDT) patients treated with CS-101/CS-206. All four achieved rapid hematopoietic reconstitution, sustained high-level pancellular fetal hemoglobin (HbF) expression, and no detectable off-target edits or product-related adverse events.

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.

Shanghai – September 11, 2026 -- CorrectSequence Therapeutics (Correctseq) reported that its transformer Base Editor (tBE) therapy CS-101/CS-206 achieved full transfusion independence or elimination of vaso-occlusive crises (VOC) in four new patients from Nigeria, Laos, Malaysia and Pakistan, expanding on earlier results in five Chinese patients who all reached transfusion independence. The findings, published September 7, 2026 in Cell Stem Cell, cover patients with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) carrying distinct genotypes, including βS/βS, β0/βE, and two β0/β0 variants.