Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
UX123
- Development stage
- Preclinical
- Lead developer
- Cincinnati Children's Hospital Medical Center
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
UX123 is a codon-optimized, pseudo-uridine-modified human ABCB11 mRNA encapsulated in lipid nanoparticles (LNPs) [4580]. It is designed to restore the expression and function of the Bile Salt Export Pump (BSEP) protein in hepatocytes [4580]. BSEP is a critical transporter of bile acids encoded by the ABCB11 gene, and its deficiency leads to Progressive Familial Intrahepatic Cholestasis (PFIC), a severe cholestatic liver disease [4580]. Developed by Ultragenyx in collaboration with Cincinnati Children's Hospital Medical Center, UX123 is being investigated as a disease-modifying mRNA-LNP therapy to rescue bile acid transport and prevent bile acid-induced cell injury in BSEP-deficient patients [4580].
- Other names
- ABCB11 mRNAABCB-11 mRNAABCB 11 mRNAcodon-optimized human ABCB11 mRNA
Beyond the preview
Go deeper on UX123.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on UX123.