What results did AX-0810 show in the Phase 1 study?

AX-0810 demonstrated dose-dependent NTCP modulation with up to 8-fold increases in total bile acids, favorable safety, and an approximately 8-week half-life.

What is the next step for ProQR’s NTCP program?

The positive data support advancement of the next-generation NTCP candidate AX-0811 and continued development in biliary atresia.

What is AX-0810 designed to do?

AX-0810 is an Axiomer RNA editing candidate designed to modulate NTCP through precise single-base RNA editing.

What disease area is ProQR targeting with this program?

ProQR Therapeutics (NASDAQ:PRQR) is developing the program for biliary atresia and other conditions involving disrupted bile acid regulation.

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

Written by Jon CuthbertPublished Jun 25, 2026, 10:30 AM

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.