OBiO Technology Celebrates First Overseas Gene Editing Therapy in China by CorrectSequence Therapeutics

CorrectSequence Therapeutics Co., Ltd. (Correctseq) has recently achieved a significant breakthrough in the treatment of transfusion-dependent β-thalassemia with their innovative base editing therapy, CS-101. Using their cutting-edge transformer Base Editor (tBE), Correctseq successfully treated the first overseas patient suffering from this condition. The clinical trial, conducted in partnership with the First Affiliated Hospital of Guangxi Medical University, resulted in the patient maintaining a transfusion-free status for over two months, with hemoglobin levels consistently above 120 g/L. This health improvement has allowed the patient to return to a normal daily life. This event marks the first time a patient outside of China has been clinically cured of a disease through gene editing therapy, representing a noteworthy milestone for the country's medical advancements.

OBiO Technology (Shanghai) Corp., Ltd. (OBiO Technology), a prominent CDMO company in the field of cell and gene therapy in China, has played a crucial role in this success. OBiO Technology congratulates both the patient and Correctseq on this major achievement. The company has been instrumental in supporting CS-101 through various stages, including process development, analytical development, production, and IND filing services. The collaboration between Correctseq and OBiO Technology exemplifies the potential of innovative partnerships to advance medical treatments and improve patient outcomes.

The Phase I clinical trial of CS-101 for β-thalassemia is progressing smoothly, while preparations for a clinical trial targeting sickle cell disease (SCD) with the same therapy are underway. In addition, a global recruitment program for SCD patients has recently commenced.

CorrectSequence Therapeutics is a clinical-stage biotech company focusing on next-generation gene editing therapies through its proprietary transformer Base Editor (tBE). Their leading candidate, CS-101, targets the HBG gene to treat β-hemoglobinopathies and has received IND approval from the China National Medical Products Administration (NMPA). Clinical data has demonstrated the superior performance of CS-101. Furthermore, proof-of-concept data in mice for in vivo pipelines using tBE-editing therapies delivered via lipid nanoparticles (LNP) are available, targeting metabolic dysfunction and related diseases. Ex vivo multiplex editing of T cells has shown to preserve T cell growth and functionality in vivo, making tBE an ideal tool for developing next-generation cell therapies. Correctseq is actively developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases.

OBiO Technology, established in 2013, specializes in providing comprehensive gene and cell therapy solutions as a Contract Research Organization (CRO) and Contract Development and Manufacturing Organization (CDMO). The company offers a wide range of services across preclinical, IND, clinical, and commercial stages, including plasmids, mRNA, AAV, LVV, Ad viral vectors, various cell therapies such as CAR-T, NK, Treg, iPSCs, and exosomes. They also focus on advanced technologies like inducible viral vector packaging, ultralow endotoxin processes, and AAVneO screening for tissue-specific AAV variants. Committed to their mission of "Enable Gene Therapy for Better Lives," OBiO Technology is dedicated to delivering high-quality services worldwide, from research stages to clinical applications, aiming to benefit global populations.

The collaboration and technological advancements by Correctseq and OBiO Technology are paving the way for new treatments and offering hope to patients with previously untreatable genetic disorders.