ProQR Therapeutics
the Axiomer platform uses ADAR-recruiting editing oligonucleotides (EONs); AX-0810, a GalNAc-conjugated EON modulating NTCP for cholestatic liver disease, produced the first clinical validation of the platform in 2026 — a dose-dependent up to 8-fold rise in total bile acids at 6 mg/kg (above the 2-fold target-engagemen…
- Country
- Netherlands
- Ticker
- NASDAQ:PRQR
NTCP editor (AX-0810)
Coverage
| CN | EU | US | |
|---|---|---|---|
| therapeutics-platforms | ✓ | ✓ | ✓ |
Regulatorsfdaemanmpa
Technologies
| Name | Cluster | Risk | |
|---|---|---|---|
| RNA editing (ADAR) therapeutics | therapeutics-platforms | Medium | → |
Compiled by BIOECON via the bioecon-brief pipeline.
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