CARLSBAD, Calif., Dec. 3 /PRNewswire-FirstCall/ -- Isis Pharmaceuticals, Inc. announced today that it has added two new drugs to its development pipeline, ISIS-FXIRx and ISIS-SMNRx.
“Using our efficient antisense drug discovery platform, we were able to selectively target and inhibit the production of every clotting factor produced in the liver. This extensive screening effort allowed us to evaluate the relative profiles of inhibitors of each clotting factor and to select ISIS-FXIRx, which we believe will provide the optimal therapeutic activity without causing bleeding, a major side effect of marketed anti-clotting drugs,” said Stanley T. Crooke, M.D., Ph.D., Chairman and Chief Executive Officer of Isis. “With ISIS-FXIRx entering development, we are continuing to broaden our cardiovascular franchise beyond regulation of lipids and inflammatory processes into thrombosis.”
SMA is caused by a genetic deletion of the survival motor neuron 1 (SMN1) gene. ISIS-SMNRx is designed to treat SMA by modulating the splicing of a closely related pre-mRNA (SMN2), which leads to the production of the protein, survival motor neuron (SMN), which is associated with normal motor function. By altering splicing to produce SMN, ISIS-SMNRx may be able to compensate for the underlying genetic defect. ISIS-SMNRx is the first antisense drug to enter Isis’ development pipeline that modulates splicing, a novel antisense mechanism.
ABOUT SPLICING
Splicing is a normal mechanism that the cell uses in order to produce many different, but closely related proteins from a single gene by varying the processing of the RNA. It is estimated that of the approximately 25,000 genes in the human genome, 40% to 60% have alternative splice forms. In some cases, alternative splicing of proteins results in the production of proteins that are involved in disease. These diseases are referred to as splicing diseases and include SMA, cystic fibrosis and Duchenne’s muscular dystrophy.
ABOUT ISIS PHARMACEUTICALS, INC.
Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners. The Company has successfully commercialized the world’s first antisense drug and has 21 drugs in development. Isis’ drug development programs are focused on treating cardiovascular, metabolic and severe neurodegenerative diseases and cancer. Isis’ partners are developing antisense drugs invented by Isis to treat a wide variety of diseases. Isis and Alnylam Pharmaceuticals are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development and commercialization of microRNA therapeutics. Isis also has made significant innovations beyond human therapeutics resulting in products that other companies, including Abbott, are commercializing. As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of over 1,600 issued patents worldwide. Additional information about Isis is available at www.isispharm.com.
In this press release, unless the context requires otherwise, “Isis,” “Company,” “we,” “our,” and “us” refers to Isis Pharmaceuticals and its subsidiaries, including Regulus Therapeutics Inc.
Isis Pharmaceuticals, Inc.
