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ORT 247

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Drug Profile

ORT 247

Alternative Names: ORT-247

Latest Information Update: 26 May 2025

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At a glance

  • Originator Orthogonal Neuroscience
  • Developer Charcot-Marie-Tooth Association; Orthogonal Neuroscience
  • Class Monoclonal antibodies; Neuroprotectants
  • Mechanism of Action EphA4 receptor inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Amyotrophic lateral sclerosis
  • Preclinical Charcot-Marie-Tooth disease

Most Recent Events

  • 17 Jan 2025 Pharmacodynamics and adverse event data from preclinical trials in Amyotrophic lateral sclerosis and Charcot Marie Tooth disease released by Orthogonal Neuroscience
  • 24 Nov 2023 Orthogonal Neuroscience completes a phase-I trial in Amyotrophic lateral sclerosis (In volunteers) in USA (IV) (NCT06769620)
  • 30 Jun 2023 Orthogonal Neuroscience plans to initiate clinical trials in Charcot-Marie-Tooth disease in mid-2023

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