Today is the 73rd’s Children’s Day. On May 31, 2023, the Wuzhishan Branch of Jiusan Society, the branch of Hainan Women and Children's Medical Center and the Wuzhishan Disabled Persons' Federation co-sponsored the "Care for the Children, Walk with the Children" donation activity for exceptional children and thalassemia children to celebrate the Children’s Day, which was held in Wuzhishan Special Education School. The leaders of Hainan Provincial CPPCC, CPPCC, United Front Work Department, Women's Federation, Disabled Persons' Federation, Education Bureau, Health Commission, Family Planning Commission, Jiusan Society, Red Cross of Wuzhishan and other units attended the activity. CorrectSequence Therapeutics (Nasdaq: Correctseq) was invited to participate in the activity and make a donation, wishing the children a happy Children’s Day, and hoping thalassemia children recover soon and enjoy a healthy life.

Figure 1: "Care for the Children, Walk with the Children" donation activity for exceptional children and thalassemia children.

Figure 2: Correctseq made a donation on the activity (left: Katie Li, head of Clinical Operation, Correctseq)

Correctseq attended the donated activity. Correctseq aims to use the innovative base editing technology to help the children with thalassemia. tBE (transformer Base Editor), which was created by the scientific co-founders of Correctseq, has obtained patent authorization from China, U.S.A., Australia. The first pipeline CS-101 was for β-thalassemia. It combined tBE and cell therapy to create ex vivo gene editing hematopoietic stem cell therapy. Compared with traditional normative blood transfusion treatment and general hematopoietic stem cell transplantation, this therapy does not need to wait for matching and allotransplantation, and can truly achieve "once treatment, lifetime cure". Meanwhile, tBE induced efficient on-target editing and undetected OT mutations compared with other BEs and Cas Nuclease gene editors, so it’s more safety to be used to develop innovative gene editing drugs. Compared with Cas Nuclease gene editing therapy, tBE has higher γ-globin induction, lower cytotoxicity, and undetected off-target mutations.   

Figure 3: The ex vivo gene editing therapy of Correctseq

Correctseq is working with various partners to advance CS-101 into the IND application and FIH phase. It is believed that with the loving care and full support of all sectors of society, children with thalassemia will be completely cured and enjoy a normal life!