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RC-1001
- Development stage
- Preclinical
- Lead developer
- Sarepta Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptide-Drug Conjugates → Peptide Conjugates → Peptides, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
RC-1001 is a peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) developed by Sarepta Therapeutics as a preclinical research tool and mouse surrogate for Duchenne muscular dystrophy (DMD) therapy. It comprises a morpholino antisense oligonucleotide (PMO) covalently linked to a cell-penetrating peptide (CPP) to enhance cellular uptake and nuclear delivery. RC-1001 specifically targets exon 23 of the murine dystrophin gene in mdx mice, inducing exon skipping to restore the mRNA reading frame and promote the expression of a functional, truncated dystrophin protein. It has been extensively used in pharmacokinetic and pharmacodynamic (PK/PD) modeling to characterize the relationship between plasma/tissue exposure and biological response, supporting the translation of PPMO technology into clinical candidates such as vesirplirsen (SRP-5051).
- Other names
- mouse surrogate PPMOPPMO-1001PPMO1001PPMO 1001Peptide-conjugated phosphorodiamidate morpholino oligomer 1001
02
Targets
DMD (Dystrophin)
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