DS-5141b is an antisense oligonucleotide (ASO) consisting of 2′-O,4′-C-ethylene-bridged nucleic acids (ENA) and 2′-O-methyl RNA that induces dystrophin mRNA exon 45 skipping for treatment of Duchenne muscular dystrophy (DMD). As sufficient efficacy and safety had been observed in previous non-clinical studies of DS-5141b, DS-5141b currently has been in Ph1/2 clinical trial in Japan. In a repeated dosing study once a week for 4 weeks, DS-5141b showed clear exon 45 skipping at doses over 3 mg/kg in all examined tissues, anterior tibial muscles, diaphragm, and heart in mdx mice.

Read More...

Leave a comment.

Your email address will not be published. Required fields are marked*

Andoird App
Loading...