2/1/13 Seeking " SRPT Developing It's Own Antisense " LINK
Isis: FDA Approval Of Kynamro Promises A Bright Future For Company
February 1, 2013 By: Prohost Biotech
In addition to Isis and alnylam, Sarepta (SRPT), which used to be known by the name AVI Pharmaceuticals, is developing its own proprietary far-reaching antisense molecule therapeutics. A few months ago, the firm unveiled results of its lead product eteplirsen for Duchene muscular dystrophy. The drug demonstrated a successful production of dystrophin, a rod-shaped cytoplasmic protein and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane. Dystrophin deficiency has been definitively established as one of the root-causes muscle dystrophy.