ProQR Therapeutics NV has received authorization from the Central Committee on Research Involving Human Subjects (CCMO) for its Clinical Trial Application $(CTA)$ to begin a Phase 1 study of AX-0810 in healthy volunteers. This approval follows review under the European Medicines Agency's centralized process. AX-0810 is ProQR's lead investigational editing oligonucleotide targeting NTCP, being developed for the treatment of cholestatic diseases such as primary sclerosing cholangitis and biliary atresia. The Phase 1 trial, to be conducted in the Netherlands, will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of AX-0810.
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