- **ProQR presented clinical data for its Alpha-1 Antitrypsin Deficiency (AATD) program, showing patients achieved MZ-like protein levels with 12 micromolar of total protein, increasing to 13 micromolar, and a shift from 0% to 44% M protein after a single dose in the lowest 200 mg cohort**.  **[0001612940_2193860_1]** 
- **The AATD program also demonstrated nearly 11 micromolar of M protein and over 20 micromolar of endogenous protein production during an acute phase event, with sustained editing for over two months from a single dose**.  **[0001612940_2193860_2]** **[0001612940_2193860_3]** 
- **ProQR announced clearance to initiate a Phase One trial for AX-0810, its lead program for cholestatic diseases, which is designed to edit NTCP and block bile acid transport into the liver**.  **[0001612940_2193860_5]** 
- **The company is also advancing its pipeline in CNS, having shown up to 60% editing efficiency in non-human primates, and is targeting PNPLA3 for hepatic diseases, with a research day scheduled for October 29 to discuss these developments**.  **[0001612940_2193860_6]** **[0001612940_2193860_7]**

ProQR Provides Updates on RNA Editing Programs, Including AATD Clinical Data and AX-0810 Phase 1 Initiation

- **WaveLife** reported clinical data for its alpha-one antitrypsin deficiency (AATD) program, demonstrating **12-13 micromolar** total protein and **44-65% M protein** after single/multi-dose, with editing sustained for over **two months**.  **[0001612940_2193885_1]** **[0001612940_2193885_2]** 
- **ProQR** received **clearance to initiate a Phase 1 trial** for AX-810, targeting cholestatic diseases, with an event scheduled for **November 3** to detail the trial design.  **[0001612940_2193885_5]** **[0001612940_2193885_6]**  Preclinical data indicated **15% editing** led to a **two-fold increase in serum bile acids**.  **[0001612940_2193885_5]** 
- **Airena** presented preclinical data for its AATD program, demonstrating high mAAT (**39 micromolar**) and total AAT (**63 micromolar**) in mouse models, and durability (half-life over **30 days**) in non-human primates, with a **CTA filing expected by year-end**.  **[0001612940_2193885_4]** 
- The companies are expanding their ADAR pipelines; **WaveLife** will discuss its PMVLA-three program for liver diseases on **October 29**, and **ProQR** highlighted its CNS pipeline, achieving up to **60% editing efficiency** in non-human primates.  **[0001612940_2193885_6]** **[0001612940_2193885_7]**

Company not found (0PQ)