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AP-2

Development stage: Unknown
Lead developer: Molefy Pharma
Modality: Small Molecules

Overview

AP-2 is a small molecule TDP-43 modulator developed by Molefy Pharma. It is designed to restore the balance of the TDP-43 protein, a key pathological feature in approximately 97% of Amyotrophic Lateral Sclerosis (ALS) cases. The drug is currently undergoing a first-in-human Phase 1 clinical trial to determine its maximum tolerated dose, safety, and therapeutic window for ALS, supported by a grant from The ALS Association.

Targets

TARDBP (TAR DNA-binding protein 43)

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