Drug Profile

Research programme: C9orf72 translational modulators - Libra Therapeutics

Alternative Names: Research programme:neurodegenerative diseases targeting therapies-Libra Therapeutics

Latest Information Update: 18 Mar 2025

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At a glance

Originator Libra Therapeutics
Class Antidementias; Antiparkinsonians; Neuroprotectants; Small molecules
Mechanism of Action C9orf72 protein modulators; Transcription factor modulators

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

18 Mar 2025 Preclinical development in Frontotemporal dementia and Amyotrophic lateral sclerosis is ongoing USA (Libra Therapeutics pipeline, March 2025)
28 Oct 2024 No recent reports of development identified for research development in Amyotrophic-lateral-sclerosis in USA
28 Oct 2024 No recent reports of development identified for research development in Frontotemporal dementia in USA

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