AVI-3307

AVI-3307 is an antisense phosphorodiamidate morpholino oligomer (PMO) developed by AVI BioPharma (now Sarepta Therapeutics) for the treatment of Duchenne muscular dystrophy (DMD). The drug is designed to target and bind to exon 50 of the dystrophin pre-mRNA, inducing exon skipping during the splicing process. This mechanism aims to restore the reading frame of the dystrophin gene in patients with specific deletions, allowing for the production of a truncated but partially functional dystrophin protein, similar to that found in the milder Becker muscular dystrophy. AVI-3307 was part of an early suite of exon-skipping candidates developed using AVI BioPharma's proprietary PMO platform, which has since produced several FDA-approved therapies for other DMD exons. While AVI-3307 itself was an early research-stage candidate, the program for exon 50 skipping has continued to evolve within Sarepta's pipeline, including the development of next-generation peptide-conjugated PMOs (PPMOs).

Other names

exon 50 skipping PMOexon50 skipping PMOexon-50 skipping PMO