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peptide-conjugated phosphorodiamidate morpholino oligonucleotide targeting Umod
- Development stage
- Preclinical
- Lead developer
- Sarepta Therapeutics
- Modality
- 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
- Intravenous
01
Overview
Peptide-conjugated phosphorodiamidate morpholino oligonucleotide targeting Umod is an investigational antisense oligonucleotide therapy being developed by Sarepta Therapeutics for the treatment of autosomal dominant tubulointerstitial kidney disease-uromodulin (ADTKD-UMOD). The drug consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a cell-penetrating peptide (PPMO) designed to bind to complementary sequences of the uromodulin (UMOD) pre-mRNA. By inducing nonsense-mediated decay or exon skipping, it knocks down the expression of mutant, misfolded uromodulin protein, thereby reducing its toxic intracellular aggregation and accumulation in the loop of Henle and distal convoluted tubules, which potentially preserves renal function.
- Other names
- UMOD-targeting PPMOpeptide-conjugated phosphorodiamidate morpholino oligomer targeting Umoduromodulin-targeting PPMO
02
Targets
UMOD (Uromodulin)
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