CorrectSequence Therapeutics Co., Ltd
- Biotech or pharma, therapeutic R&D
CorrectSequence Therapeutics (Correctseq) is a clinical-stage biotechnology company employing its proprietary transformer Base Editor (tBE) to pioneer next-generation gene editing therapies. tBE offers significant advantages in controlling off-target effects and enhancing in vivo editing efficiency.
Our leading pipeline program, CS-101, targeting β-hemoglobinopathies, is in Phase I clinical stage currently. Clinical data demonstrates its superior performance, indicating its great potential to become a best-in-class gene editing therapy β-hemoglobinopathies.
Proof-of-concept (POC) efficacy and safety data in mice and NHP using tBE-editing therapies via LNP delivery are available, including targets for metabolic dysfunction and associated diseases.
Multiplex editing of primary T cells and production cell lines like CHO and HEK293 preserved cell growth and function compared with non-edited cells, establishing tBE as the ideal gene editing tool for cell line engineering.
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