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C9orf72 Transcriptional Modification

C9orf72-associated ALS/FTD

DiscoveryActive

Key Facts

Indication

C9orf72-associated ALS/FTD

Phase

Discovery

Status

Active

Company

Libra Therapeutics

About Libra Therapeutics

Libra Therapeutics is a private, preclinical-stage biotech targeting neurodegenerative diseases through the modulation of lysosomal function and autophagy. Its core strategy involves a proprietary Lysofunction screening platform to identify compounds that enhance these cellular clearance pathways, with programs focused on TRPML1 activation and targeting the C9orf72 genetic mutation. The company is led by a team with deep neuroscience and drug development experience and is backed by a syndicate of life science venture capital firms.

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Therapeutic Areas

Neurodegenerative Diseases Amyotrophic Lateral Sclerosis Frontotemporal Dementia Alzheimer's Disease Parkinson's Disease Huntington's Disease

Other C9orf72-associated ALS/FTD Drugs

Drug	Company	Phase	Status
PAV-615 (Neuroscience)	Prosetta Biosciences	Preclinical	Active
C9orf72 RAN Translation Inhibition	Libra Therapeutics	Discovery	Active