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ATL-001b

Development stage: Preclinical
Lead developer: Atlas Molecular Pharma
Modality: Classical Binding Small Molecules → Small Molecules
Administration: Oral

Overview

ATL-001b is a preclinical drug candidate developed by Atlas Molecular Pharma SL, primarily indicated for Porphyria Cutanea Tarda (PCT). It is being investigated as a pharmacological chaperone designed to improve heme homeostasis by addressing deficiencies in uroporphyrinogen decarboxylase (UROD) activity. This approach aims to stabilize the UROD enzyme and restore its function, thereby preventing the accumulation of damaging porphyrins associated with PCT. The development is currently in the lead validation phase for therapeutic chaperones.

Targets

UROD (Uroporphyrinogen decarboxylase)

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