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RTX-101
- Development stage
- Phase 1
- Lead developer
- Repeat Therapeutics
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
RTX-101 is an antisense oligonucleotide (ASO) therapeutic candidate developed by Repeat Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with hexanucleotide repeat expansions in the C9orf72 gene. The drug is designed to selectively target and degrade the sense and/or antisense transcripts containing the G4C2 repeat expansion, thereby preventing the formation of toxic RNA foci and the translation of neurotoxic dipeptide repeat (DPR) proteins. These DPR proteins and RNA foci are central to the pathogenesis of C9orf72-mediated neurodegeneration. RTX-101 is currently being evaluated in early-phase clinical trials to assess its safety, tolerability, and pharmacokinetic profile following intrathecal administration in patients with ALS.
02
Targets
C9orf72 HRE (C9orf72 hexanucleotide repeat expansion)
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