Antisense Drug Discovery Platform

Antisense is a new platform for drug discovery. Platform technologies combine all the elements necessary to create rapidly and efficiently a stream of new products.

Our antisense technology offers all of the qualities to build a successful drug discovery platform and to create better drugs to benefit patients.

• Specificity. We design our antisense drugs to selectively target one and only one gene product. All other things being equal, the more selectivity a drug has for its target the better the drug.
• Broad Applicability. Antisense drugs can be created for any target including targets that are considered "un-druggable" by traditional small molecule technology. Antisense drugs have been shown to accumulate in specific organs and tissues, including the liver, kidney, spleen, bone marrow and fat cells. Our drug research is focused on diseases that are associated with RNA targets found in these tissues. Because antisense drugs distribute to many tissues, antisense drugs can be used to treat a wide variety of disease and bring benefit to patients in need.
• Rational Design. Antisense drug discovery is more rational than any other type of drug discovery because:
• The rules for creating antisense drugs are known. They bind to target RNA.
• The building blocks of antisense technology are constant across all antisense drugs. It is the order of the building blocks that direct our drugs to a specific target.
• The distribution and metabolism of antisense drugs are very similar from drug to drug, resulting in a common and often times, predictable, safety profile across all antisense drugs.
• Efficiency. Our antisense platform is much less costly and time consuming at the drug discovery and early development stages than traditional small molecule drugs. We can translate what we learn from the testing of one antisense drug to future antisense drugs and reduce the potential for failure in early stages of development, thus creating significant competitive advantage for the platform.
• Manufacturing. The advances we have made in process chemistry have resulted in dramatic reductions in the cost of our drugs. It is a simple and cost effective process in which our antisense drugs are chemically synthesized.

We have successfully used our drug discovery platform to generate a large pipeline of first-in-class antisense drugs, each unique to a specific RNA target, and each to treat a disease where there are patients with significant unmet medical needs.