Correctsequence Therapeutics

CorrectSequence Therapeutics (Correctseq) is a clinical-stage biotech company employing its proprietary transformer base editing (tBE) technology 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 demonstrates its superior performance, indicating significant potential for CS-101 to become a best-in-class gene editing therapy for curing patients with β-hemoglobinopathies. 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. tBE offers significant advantages in controlling off-target effects and enhancing in vivo editing efficiency, making it ideal for both multiplex editing and precise single-target editing. It is compatible with various delivery system, including ex vivo editing in hematopoietic stem cells (HSCs) and T cells, as well as in vivo editing via LNPs or adeno-associated viruses (AAVs). We are developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases. Our focus on biotechnology innovation, research and development underscores our commitment to providing efficient, reliable and safe solution for unmet medical needs.