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SOM1311
- Development stage
- Unknown
- Lead developer
- SOM Biotech
- Modality
- Classical Binding Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
SOM1311 is a repurposed small molecule pharmacological chaperone of phenylalanine hydroxylase (PAH) under development by SOM Biotech for the treatment of phenylketonuria (PKU), a rare congenital metabolic disorder characterized by the accumulation of phenylalanine due to deficient PAH activity. As a PAH agonist and modulator, SOM1311 aims to stabilize and enhance the function of mutant or misfolded PAH enzyme, thereby reducing toxic levels of phenylalanine in patients with PKU. The drug was identified using artificial intelligence-based screening technology (SOMAIPRO) and has shown promising preclinical data supporting advancement directly into Phase 2a proof-of-concept clinical trials[1][2][3][4][6].
- Other names
- small-molecule pharmacological chaperone of phenylalanine hydroxylase
02
Targets
PAH (Phenylalanine hydroxylase)
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