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Exondys 51

Sarepta Therapeutics, Inc.

Drug Names(s): AVI-4658, Eteplirsen, Exondys51

Description: AVI-4658 is a novel phosphorodiamidate morpholino oligomer (PMO) designed to induce the skipping of exon 51 in the dystrophin gene. Morpholino oligomers (oligos) are an antisense technology used to block access of other molecules to specific sequences within nucleic acid. Morpholinos block small (~25 base) regions of the base-pairing surfaces of ribonucleic acid (RNA).

Duchenne's muscular dystrophy is caused by the absence of the protein dystrophin. 65% of cases are caused by deletions of DNA that can disrupt the open reading frame (the part of the DNA that gets made into RNA), leading to premature termination of the translation from DNA to RNA.

AVI-4658 is designed to benefit patients with certain deletion mutations in the dystrophin gene by inducing the skipping of exon 51 of the dystrophin gene, which restores the open reading frame for these specific mutations to make a shorter but working form of the protein.

Deal Structure: Sarepta and University of Western Australia
In November 2008, AVI (Sarepta) announced the signing of an exclusive worldwide license agreement with the University of Western Australia (UWA) to a patent application related to the treatment of Duchenne Muscular Dystrophy (DMD). The patent application claims compositions and methods for treating DMD in humans by skipping exons in the dystrophin gene using antisense oligomers. In addition to the UWA patent application, AVIs patent position in exon skipping includes exclusive rights to general RNA splice altering patents gained though AVIs acquisition of Ercole as well as other AVIfiled patents and inlicensed intellectual property specific to exon skipping of the dystrophin gene as a therapeutic target.

In April 2013, Sarepta and the University of Western Australia (UWA) announced that they have entered into an exclusive, worldwide licensing agreement for intellectual property rights to support the development of...See full deal structure in Biomedtracker

Partners: University of Western Australia Flagship Biosciences LLC


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