China-based CorrectSequence Therapeutics has reported positive, long-term data from an investigator-initiated trial (IIT) of its ex vivo base-edited cell therapy, CS-101. The study detailed results in five patients with transfusion-dependent beta-thalassaemia who were treated with the therapy, which utilises the company's transformer Base Editor (tBE) to edit haematopoietic stem cells. All five patients achieved rapid and sustained increases in foetal (HbF) and total haemoglobin, becoming transfusion-independent within one month of treatment, with the longest follow-up exceeding 28 months. The therapy demonstrated a favourable safety profile with no product-related adverse events detected.

CS-101 is designed to reactivate HbF production without creating DNA double-strand breaks, a potential advantage over CRISPR-Cas9 nuclease approaches. CorrectSequence is now advancing CS-101 towards formal investigational new drug (IND) trials and potential regulatory approval, with the aim of developing a best-in-class therapy for beta-haemoglobinopathies.

PharmCube's NextBiopharm® database shows that CS-101 is the third-most advanced BCL11A genome editing therapy globally. Click here to request a free trial for NextBiopharm®.