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Drug intelligence / Profile preview
EX-044
- Development stage
- Preclinical
- Lead developer
- Excelsior
- Modality
- Small Molecules
01
Overview
EX-044 is a small molecule inhibitor of the Transforming Growth Factor-beta (TGF-β) pathway currently under preclinical development by Excelsior Group. It is specifically being investigated for the treatment of Wilson's disease, a rare genetic disorder that leads to copper poisoning in the body, particularly affecting the liver and brain. Within Excelsior's TGF-β Research Center, EX-044 is described as a candidate for rare diseases utilizing a mechanism of action distinct from current therapeutic options, likely aimed at mitigating the tissue fibrosis and organ damage associated with the progression of Wilson's disease.
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