PRO044

PRO044 is an antisense oligonucleotide drug developed for the treatment of Duchenne Muscular Dystrophy (DMD) in patients with mutations amenable to exon 44 skipping. It is designed to induce specific skipping of exon 44 during pre-mRNA splicing of the dystrophin gene, thereby restoring the reading frame and enabling production of a functional, albeit truncated, dystrophin protein. The drug was evaluated in Phase I/II and Phase II clinical trials using both subcutaneous and intravenous administration routes. Clinical studies demonstrated that PRO044 could increase dystrophin expression in muscle tissue and was generally well tolerated by patients[1][3][4][5]. The development program included dose-escalation studies as well as an open-label extension study[6][8].

Other names

Exon 44 specific phosphorothioate oligonucleotideExon44 specific phosphorothioate oligonucleotideExon-44 specific phosphorothioate oligonucleotide