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casimersen
- Development stage
- Approved
- Lead developer
- Sarepta Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → 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
- Intravenous
01
Overview
Casimersen is an antisense phosphorodiamidate morpholino oligonucleotide (PMO) indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 45 skipping. It works by binding to exon 45 of the DMD pre-mRNA, causing this exon to be skipped during mRNA processing. This restores the reading frame and enables production of a truncated but partially functional dystrophin protein, similar to that seen in Becker muscular dystrophy, thereby improving muscle stability and potentially slowing disease progression. Casimersen is administered as a once-weekly intravenous infusion and was developed by Sarepta Therapeutics. Its FDA approval was granted under accelerated approval based on increased dystrophin levels as a surrogate endpoint.
- Brand names
- Amondys 45Amondys45Amondys-45
- Other names
- casimersenSRP-4045SRP4045SRP 4045Amondys 45Amondys45Amondys-45
02
Targets
Neuraminidase
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