Antisense is an innovative platform for drug discovery. Platform technologies combine all the elements necessary to rapidly and efficiently create a stream of new products.
The targets for antisense drugs are RNA molecules. RNA is an optimal drug target for several reasons:
- RNA is universal. It is vital to all living things, as it affects the expression of all genes.
- RNA is a simpler molecule than proteins in that it is composed of four fundamental building blocks called nucleotides (A,C,U,G). Proteins are made from 20 different building blocks called amino acids.
- RNA-based drug discovery is a rational and efficient process. We design our antisense drugs to interact precisely with a specific sequence of RNA.
Many of the antisense drugs in our pipeline bind to protein-coding RNAs, including messenger RNAs (mRNAs), and inhibit the production of disease-causing proteins. Recently the scientific community has discovered many new types of RNAs, including microRNAs and long non-coding RNAs, which are involved in the regulation of protein production within the cell. Our antisense technology is uniquely suited to exploit these exciting new RNA targets, as we have done with microRNAs through Regulus Therapeutics.
Our antisense technology is efficient and enables us to create a robust pipeline of antisense drugs to treat 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 the scientific community considers “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. We focus our drug research on diseases that are associated with RNA targets found in these tissues. Because antisense drugs distribute to many tissues, antisense drugs could treat a wide variety of diseases and bring benefit to patients in need.
- Rational Design. Antisense drug discovery is more rational than any other type of drug discovery because:
- We know the rules for creating antisense drugs. 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 a 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. Manufacturing our drugs 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 for which there are patients with significant unmet medical needs.