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PRO-045
- Development stage
- Discontinued
- Lead developer
- BioMarin Pharmaceutical
- Modality
- 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
- Administration
- Subcutaneous, Intravenous
01
Overview
**PRO-045** (also known as varodarsen or BMN 045) is an **antisense oligonucleotide (ASO)** developed by BioMarin Pharmaceutical (originally by Prosensa) designed to induce **exon 45 skipping** in the **DMD gene** for the treatment of **Duchenne muscular dystrophy (DMD)** in patients with amenable mutations. By modulating pre-mRNA splicing, it aims to restore the reading frame and produce a truncated, partially functional dystrophin protein, potentially ameliorating disease progression. The drug was evaluated in a Phase IIb open-label study assessing safety, efficacy, pharmacodynamics, and pharmacokinetics via subcutaneous administration but has since been **discontinued**, with similar exon-skipping ASOs like SRP-4045 (casimersen) advancing to approval for the same indication.[1][5][9][13]
- Other names
- varodarsen
02
Targets
DMD (Dystrophin)
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