A study published in Nature on April 15, 2026 announced that China’s self-developed gene-editing therapy CS-101 has cured β-thalassemia patients, freeing them from lifelong transfusions.

All five trial patients achieved rapid hematopoietic reconstruction and transfusion independence.

Developed by Chinese institutions and Shanghai’s CorrectSequence Therapeutics, CS-101 uses a precise "transformer Base Editor" that corrects genes without cutting DNA, lowering risks of mutations and structural damage.

The first patient was treated in October 2023 and has been transfusion-free for over 28 months. Nearly 20 patients with β-thalassemia and sickle cell anemia have been treated with a 100% success rate.

The therapy is being commercialized globally, with foreign patients already joining trials, aiming to help families worldwide.