An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1 - PubMed
4 days ago
- #Clinical Trial
- #Oligonucleotide Therapy
- #Myotonic Dystrophy
- Myotonic dystrophy type 1 is a rare, progressive neuromuscular disease with no approved therapies.
- The disease is caused by a trinucleotide repeat expansion in DMPK, leading to dysregulated alternative splicing.
- Delpacibart etedesiran (AOC 1001) is a monoclonal antibody-oligonucleotide conjugate targeting transferrin receptor 1 and DMPK mRNA.
- A phase 1-2 trial assigned participants to receive del-desiran in single or multiple doses or placebo.
- Primary endpoint was safety; secondary endpoints included pharmacokinetic and pharmacodynamic profiles and changes in splicing patterns.
- Adverse events were mostly mild or moderate, with two severe events in higher dose groups.
- DMPK mRNA levels in muscle decreased significantly in treatment groups compared to placebo.
- Reductions in missplicing scores were observed, indicating potential therapeutic benefit.
- Results support further clinical investigation of del-desiran for myotonic dystrophy type 1.