A collaborative clinical study titled "Clinical application of base editing for treating β-thalassemia", conducted by CorrectSequence Therapeutics Co Ltd, a biopharmaceutical company based in Shanghai's Pudong New Area, with partners including ShanghaiTech University and the Shanghai Clinical Research and Trial Center, was recently published in Nature.

A view of Shanghai's Pudong New Area. [Photo/IC]

A recent study reported on an investigator-initiated trial involving CS-101, a base-editing drug developed using a transformer base editor. Five patients with β-thalassemia experienced rapid recovery of hematopoietic function following treatment and quickly became independent of blood transfusions.

CS-101 is the world's first ex vivo base-editing therapy to enter clinical trials. The first patient treated with this therapy has remained transfusion-free for more than 28 months, making it the only base-editing therapy with over two years of post-treatment follow-up data.

The publication in Nature marks the first time clinical results from a Chinese gene-editing drug pipeline have appeared in the journal's main edition, as well as the first for China's research on β-thalassemia. Reviewers have described the therapy's efficacy and safety as setting a "new high-water mark" for ex vivo editing of hematopoietic stem cells, highlighting the international recognition of China's innovation in this field.

CorrectSequence Therapeutics is accelerating the Investigational New Drug clinical trial and the commercialization of CS-101. So far, nearly 20 patients with β-thalassemia and sickle cell disease have been treated, all of whom have achieved transfusion independence and sustained high hemoglobin levels.