ProQR Reports First Human Validation of RNA Editing Platform in Liver Disease
ProQR Therapeutics announced first-in-human data for AX-0810, providing dose-dependent evidence that its RNA-editing approach can modulate NTCP, a liver transporter involved in bile acid uptake. The company is advancing NTCP programs for cholestatic liver diseases, initially focusing on pediatric biliary atresia.
Why it matters — This marks the first human validation of ProQR's RNA-editing platform, a key step for its liver disease pipeline that PRQR watchers would be following closely.