ORT 247
Alternative Names: ORT-247Latest Information Update: 26 May 2025
At a glance
- Originator Orthogonal Neuroscience
- Developer Charcot-Marie-Tooth Association; Orthogonal Neuroscience
- Class Monoclonal antibodies; Neuroprotectants
- Mechanism of Action EphA4 receptor inhibitors
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- 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