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ORT-247

Development stage: Phase 1
Lead developer: Orthogonal Neuroscience
Modality: Monoclonal Antibodies → Antibody-Based Therapeutics
Administration: Intravenous

Overview

ORT-247 is a monoclonal antibody developed by Orthogonal Neuroscience that targets the Ephrin type-A receptor 4 (EphA4). It acts as an antagonist of EphA4, a mechanism considered novel in the context of neurodegenerative diseases. Preclinical studies have shown promising results for ORT-247 in models of Charcot–Marie–Tooth disease (CMT), including both demyelinating (CMT1A) and axonopathy (CMT2E) forms, with improvements observed in several disease endpoints. The drug has also entered clinical development for amyotrophic lateral sclerosis (ALS), where it has completed a Phase 1 trial evaluating safety, tolerability, and pharmacokinetics in healthy volunteers[1][2][3][5].

Other names: ORT247ORT-247ORT 247

Targets

EPHA4

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