Drug intelligence / Profile preview

tauroursodeoxycholic acid

Development stage
Phase 4
Lead developer
IRCCS San Matteo Foundation
Modality
Small Molecules
Administration
Oral
01

Overview

Tauroursodeoxycholic acid (TUDCA) is a hydrophilic bile acid that is the taurine conjugate of ursodeoxycholic acid (UDCA). It is naturally occurring in small amounts in humans and is found in high concentrations in bear bile. TUDCA functions as a chemical chaperone that mitigates endoplasmic reticulum (ER) stress and exhibits potent anti-apoptotic effects by inhibiting mitochondrial membrane permeability transition and caspase activation. In the context of transthyretin (ATTR) amyloidosis, TUDCA is being investigated for its ability to disrupt amyloid fibrils and prevent their deposition in tissues, particularly when used in combination with the antibiotic doxycycline. Beyond amyloidosis, it has been extensively studied for its neuroprotective properties in diseases such as amyotrophic lateral sclerosis (ALS) and for its hepatoprotective effects in cholestatic liver diseases.

Brand names
TudcabilTauroliteArcosylane
Other names
Tauroursodeoxycholate3α,7β-dihydroxy-5β-cholan-24-oic acid N-(2-sulfoethyl)amide
02

Targets

NFE2L2 (Nuclear factor (erythroid-derived 2)-like 2)FXR (Farnesoid x-activated receptor)

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