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FIX-circRNA
- Development stage
- Preclinical
- Lead developer
- Mahidol University
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
FIX-circRNA is a circular messenger RNA (circRNA) therapeutic candidate designed to express human coagulation Factor IX (FIX) for the treatment of hemophilia B. Developed by researchers at Mahidol University, the therapy utilizes a circularized RNA platform to drive sustained and stable expression of biologically active FIX protein. Unlike conventional linear mRNA, the covalently closed loop structure of circRNA provides resistance to exonuclease-mediated degradation, potentially offering a longer half-life and more durable therapeutic protein expression. In preclinical evaluations, transient transfection of FIX-circRNA in Chinese hamster ovary (CHO) cells successfully produced functional FIX zymogens that demonstrated coagulant activity by shortening activated partial thromboplastin time (aPTT) in FIX-deficient plasma.
- Other names
- FIX circular RNAFIX-encoding circular RNAcircular RNA-encoded coagulation factor IX
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