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ATL-001
- Development stage
- Unknown
- Lead developer
- Atlas Molecular Pharma
- Modality
- Classical Binding Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
ATL-001 is an orally administered chemical molecule developed by Atlas Molecular Pharma. It functions as a pharmacological chaperone, specifically designed to stabilize the enzyme uroporphyrinogen III synthase (UROIIIS). This stabilization aims to restore the enzyme's proper function, which is deficient in patients suffering from Congenital Erythropoietic Porphyria (CEP) due to inherited mutations. By stabilizing UROIIIS, ATL-001 is intended to reduce the accumulation of toxic porphyrins, thereby addressing the underlying pathology of CEP.
- Other names
- ciclopirox olamineciclopirox
02
Targets
UROS (Uroporphyrinogen III synthase)
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