SHANGHAI, Aug. 6, 2024 /PRNewswire/ -- Recently, CorrectSequence Therapeutics Co., Ltd. (Correctseq) announced a significant milestone in their base editing therapy CS-101 for transfusion-dependent β-thalassemia. Utilizing their pioneering transformer Base Editor (tBE), Correctseq has successfully cured the first overseas patient with transfusion-dependent β-thalassemia in a clinical trial in collaboration with the First Affiliated Hospital of Guangxi Medical University. The patient has achieved a sustained transfusion-free status for over two months, with the hemoglobin level stabilized at above 120 g/L. As a result, the patient has been able to resume a normal life. This milestone marks China's first documented report of an overseas patient being clinically cured through gene editing therapy. OBiO Technology (Shanghai) Corp., Ltd. (OBiO Technology) extends its heartfelt congratulations and best wishes to the patient who has been cured, and warmly congratulates the partner Correctseq on this significant progress.

The Phase I clinical trial for the treatment of β-thalassemia with CS-101 is proceeding steadily. In tandem, a clinical trial targeting sickle cell disease (SCD) with CS-101 are in active preparation. A global recruitment program for SCD patients is recently launched.

OBiO Technology is a leading CDMO company specializing in cell and gene therapy in China, equipped with 15 GMP vector production lines and 20 GMP cell therapy production lines. As a reliable partner with Correctseq, OBiO Technology has provided solid support for CS-101, including process development, analytical development, production and IND filling services. OBiO Technology celebrates the significant achievements made in treating patients with CS-101, affirming the successful collaboration between the two companies. Driven by innovation and united in purpose, we believe our close partnership will bring hope and a brighter future to patients worldwide!

About CorrectSequence Therapeutics

CorrectSequence Therapeutics^TM (Correctseq^TM), is a clinical-stage biotech company employing its proprietary transformer Base Editor (tBE) to pioneer next-generation gene editing therapies. Our leading pipeline candidate, CS-101, utilizing innovative base editor targeting HBG, curing β-hemoglobinopathies, has obtained IND approval from the China NMPA. Clinical data demonstrate its superior performance. Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for metabolic dysfunction and associated diseases. Ex vivo multiplex editing of T cells on multiple targets simultaneously preserved T cell growth and function in vivo compared to non-edited cells, establishing tBE as the ideal gene editing tool for the next-generation cell therapy development. We are developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases.

Please refer to our homepage for more information about the tBE and its therapeutics applications at www.correctsequence.com

About OBiO Technology

Established in 2013, OBiO Technology is a pioneering Contract Research Organization (CRO) and Contract Development and Manufacturing Organization (CDMO) in gene and cell therapy, offering comprehensive solutions. Our unwavering dedication ensures high-quality CRO and CDMO services for consumers worldwide across preclinical, IND, clinical, and commercial stages, including plasmids, mRNA, AAV, LVV, Ad viral vectors, cell therapy (CAR-T, NK, Treg, iPSCs), exosomes, and cutting-edge technologies like inducible viral vector packaging, ultralow endotoxin processes, and AAVneO screening for tissue-specific AAV variants. Guided by our mission "Enable Gene Therapy for Better Lives", we are dedicated to delivering top-tier services globally. From bench to clinic, we are committed to advancing your product and benefiting populations worldwide.