Archemix technology is based on the in vitro selection of nucleic acids. In the same way combinatorial chemistry produces millions of compounds that feed the drug discovery pipeline, in vitro selection of nucleic acids yields compounds (nucleic acids) with useful biological activities. Through this process, enormous libraries (up to 1016 unique compounds) are screened to identify individual compounds best suited to a particular application. This method allows Archemix to generate powerful new compounds, Aptamers and RiboReporters™, that meet specific commercial needs.
Aptamers can be thought of as the nucleic acid antibodies. Archemix’s primary focus is to develop aptamers as a novel source of therapeutics for the treatment of acute and chronic diseases. Like antibodies, aptamers may be also be employed to address critical bottlenecks in the drug discovery process, including target validation and drug screening.
RiboReporters™ are unique sensors in that they are able to directly convert molecular recognition into a detectible signal. Archemix is pursuing the use of RiboReporter™ Sensors in a range of applications.
Aptamers are oligonucleotides that bind to molecular targets in a manner conceptually similar to antibodies. Through the SELEX process, aptamers have been identified against numerous target types including growth factors, enzymes, immunoglobulins, receptors, viral proteins and others. The Archemix preclinical portfolio includes aptamers directed to a wide range of validated therapeutic targets (see Examples). Aptamers are similar to therapeutic antibodies, and as such, have a number of desirable characteristics for use as therapeutics, including biological efficacy, high specificity and affinity, and excellent pharmacokinetic properties. In addition, they offer specific competitive advantages such as:
- aptamers are produced by an entirely in vitro process, which allows for the generation of initial therapeutic leads in as little as two weeks. In vitro selection allows the specificity and affinity of the aptamer to be tightly controlled, including selection against both toxic and non-immunogenic targets.
- aptamers are able to disrupt protein-protein interactions
- aptamers as a class have demonstrated little or no immunogenicity or toxicity.
- aptamer therapeutics can be administered subcutaneously.
- aptamers are chemically synthesized, allowing for lower cost and easily scaled production.
- aptamers can be stored at room temperature as lyophilized powders.