First look at amended agreement with UWA to wrap up all possible exons included with all of Sarepta's,now let us look at the amended contract of directors,ceo's ect.
looks like a package to me........ and all agreements and amendments are recent ones.
Genzyme using Sarepta's ppmo
2013 Apr;23(2):109-17. doi: 10.1089/nat.2012.0404. Epub 2013 Jan 11.
Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy.
Leger AJ, Mosquea LM, Clayton NP, Wu IH, Weeden T, Nelson CA, Phillips L, Roberts E, Piepenhagen PA, Cheng SH, Wentworth BM.
Genzyme, a Sanofi Company , Framingham, Massachusetts.
Expansions of CUG trinucleotide sequences in RNA transcripts provide the basis for toxic RNA gain-of-function that leads to detrimental changes in RNA metabolism. A CTG repeat element normally resides in the 3' untranslated region of the dystrophia myotonica-protein kinase (DMPK) gene, but when expanded it is the genetic lesion of myotonic dystrophy type 1 (DM1), a hereditary neuromuscular disease. The pathogenic DMPK transcript containing the CUG expansion is retained in ribonuclear foci as part of a complex with RNA-binding proteins such as muscleblind-like 1 (MBNL1), resulting in aberrant splicing of numerous RNA transcripts and consequent physiological abnormalities including myotonia. Herein, we demonstrate molecular and physiological amelioration of the toxic effects of mutant RNA in the HSA(LR) mouse model of DM1 by systemic administration of peptide-linked morpholino (PPMO) antisense oligonucleotides bearing a CAG repeat sequence. Intravenous administration of PPMO conjugates to HSA(LR) mice led to redistribution of Mbnl1 protein in myonuclei and corrections in abnormal RNA splicing. Additionally, myotonia was completely eliminated in PPMO-treated HSA(LR) mice. These studies provide proof of concept that neutralization of RNA toxicity by systemic delivery of antisense oligonucleotides that target the CUG repeat is an effective therapeutic approach for treating the skeletal muscle aspects of DM1 pathology.
Sarepta May Seek Partnership Advice for Experimental Drug
By Sasha Damouni - 2013-01-11T21:08:43Z
Sarepta Therapeutics Inc. (SRPT), a developer of an experimental drug for Duchenne muscular dystrophy, may seek advisers to find an overseas partner for the therapy, Chief Executive Officer Christopher Garabedian said.
Copp, If CG stated that the PMO-X compound "acts like small molecules" then AVII/SRPT has finally solved the delivery issue because an inherent pharmacological property of small molecules is their ability to cross cell membranes easily and usually without the same toxicity that transport polypeptides exhibit.