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C9orf72 Transcriptional Modification Program
- Development stage
- Preclinical
- Lead developer
- Libra Therapeutics
- Modality
- Small Molecules
- Administration
- Oral, Intrathecal, Intranasal, Subpial Injection (cns), Intra-striatal Injection
01
Overview
The C9orf72 Transcriptional Modification Program is a preclinical small molecule development effort by Libra Therapeutics aimed at treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The program focuses on modulating the transcriptional regulation of the *C9orf72* gene locus, which is frequently affected by a hexanucleotide repeat expansion (HRE). This genetic mutation leads to a loss of normal C9orf72 protein function and the gain of toxic dipeptide repeat proteins through RAN translation. By identifying small molecules that can restore or boost normal transcription levels at the *C9orf72* locus, Libra Therapeutics aims to address both the loss-of-function and gain-of-toxicity mechanisms underlying these neurodegenerative disorders.
- Other names
- C9orf72 Transcriptional Regulation ProgramC-9orf72 Transcriptional Regulation ProgramC 9orf72 Transcriptional Regulation ProgramLibra Therapeutics C9orf72 Transcriptional Modification Program
02
Targets
C9orf72 (C9orf72 hexanucleotide repeat expansion and transcripts)
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