ProQR reports positive phase 1 data for ax-0810 rna editing candidate

• ProQR Therapeutics (NASDAQ:PRQR) announced positive Phase 1 target engagement data for its first Axiomer RNA editing candidate AX-0810 in healthy volunteers.
• AX-0810 demonstrated dose-dependent NTCP modulation with up to 8-fold increases in total bile acids and a favorable safety profile.
• The ~8-week half-life supports further development in biliary atresia and advancement of next-generation candidate AX-0811.

ProQR Therapeutics (NASDAQ:PRQR) reported positive Phase 1 target engagement data for AX-0810, its first Axiomer RNA editing candidate, in healthy volunteers.

AX-0810 showed dose-dependent modulation of NTCP with up to 8-fold increases in total bile acids.

The candidate demonstrated a favorable safety profile and an approximately 8-week half-life.

These results support the advancement of next-generation NTCP candidate AX-0811 and further development of the program in biliary atresia.

Following the announcement, ProQR's share price was down at $1.81.

ProQR is developing AX-0810 as part of its Axiomer RNA editing platform designed to make precise single-base changes to RNA.

The company continues to advance its pipeline of RNA editing therapies targeting rare genetic disorders with high unmet medical need.