Drug intelligence / Profile preview

SO-003

Development stage
Unknown
Lead developer
SOICUM
Modality
Small Molecules
Administration
Oral
01

Overview

SO-003 is a repositioned small molecule drug candidate developed by **SOCIUM** for the treatment of **TDP-43 proteinopathies**, including **amyotrophic lateral sclerosis (ALS)**, **sporadic inclusion body myositis (sIBM)**, and **frontotemporal lobar degeneration (FTLD)**. Identified through SOCIUM's AI-driven drug discovery platform, SO-003 is an existing approved drug being repurposed to target the aggregation and mislocalization of **TAR DNA-binding protein 43 (TDP-43)**, a pathological hallmark of several neurodegenerative and muscle-wasting diseases. The program is currently in **Phase 2** clinical development for ALS and sIBM, with research conducted in collaboration with **Nanjing Medical University**.

02

Targets

TDP-43 (TAR DNA-binding protein 43)

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