August 26, 2025 — Leads & Copy — CorrectSequence Therapeutics Co., Ltd. (Correctseq) has announced the successful treatment of a sickle cell disease (SCD) patient using its high-precision base-editing therapy CS-101. The treatment was part of an Investigator-Initiated Trial (IIT) with the First Affiliated Hospital of Guangxi Medical University.
The patient experienced an increase in fetal hemoglobin (HbF) levels and a reduction in sickle hemoglobin (HbS). Six months post-treatment, the HbF-to-HbS ratio stabilized at 6.5:3.5, with total hemoglobin exceeding 120 g/L. The patient, a 21-year-old woman from Nigeria, had previously suffered from recurrent VOCs. For six months after receiving CS-101, she remains crisis-free and in good health.
CS-101 is a gene-editing therapy based on the transformer Base Editor (tBE) platform. It modifies a regulatory element in the promoter of the γ-globin genes (HBG1/2), mimicking single-nucleotide variants found in individuals with hereditary persistence of fetal hemoglobin.
To date, CS-101 has been used to treat nearly 20 patients with β-thalassemia or SCD in clinical trials. The first β-thalassemia patient treated has remained transfusion-free for over 22 months.
Pivotal Phase II/III trials are set to begin soon, with global recruitment underway for both SCD and β-thalassemia trials.
Media Contact:
Business Cooperate: BD@correctsequence.com
Clinical Trial Recruitment: CT@correctsequence.com
Source: CorrectSequence Therapeutics
