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AVI-5038
- Development stage
- Discontinued
- 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
AVI-5038 is a peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) antisense oligonucleotide developed to induce skipping of exon 50 of the dystrophin gene in patients with Duchenne muscular dystrophy (DMD). By targeting exon 50, AVI-5038 aims to restore the reading frame of the dystrophin mRNA, potentially enabling production of a truncated but functional dystrophin protein, thus ameliorating the disease process. AVI-5038 represents a second-generation advancement in phosphorodiamidate morpholino oligomer chemistry that includes enhanced cellular uptake via peptide conjugation. Clinical studies and preclinical animal models have shown that AVI-5038 can efficiently induce exon skipping and dystrophin expression, and it has received orphan drug designation in the EU for DMD[1][3][4][5][11].
- Other names
- B-PMO
02
Targets
DMD (Dystrophin)
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