CELL-TARGETING APTAMER LIGANDS!
The Problem: Drug candidates that prove positive in “test tube” (or in vitro) assays may face substantial problems when tested in animals, a necessary prerequisite before human clinical trials. These problems include absorption, distribution, metabolism, excretion, and toxicity.
The Solution: Aptagen’s technique, involving molecules called aptamers, overcomes the limitation of the traditional pharmaceutical drug discovery process. The unique chemistry of modified aptamers, unlike other forms of drugs currently used, permits the selection of drug candidates in whole animal models, bypassing the “test tubes” entirely, and taking drug development one step closer to human clinical trials. By using an animal model with the disease state of interest, specific knowledge of the pathology or disease condition is not needed. An added benefit is the significant reduction in the number of animals needed for drug evaluation before entering into human clinical trials.
Using the current antibody method; for every drug that makes it to market, hundreds of promising candidates that worked in-vitro (on the lab bench) fail during animal ADMET studies (absorption, distribution, metabolism, excretion, and toxicity). From lab bench to pharmacy shelves it takes from seven to fifteen years and costs $4B with an average failure rate of 80%. Half of this time and approximately 70% of all associated biopharmaceutical R&D costs are spent on in-vitro development.
Because aptamers (synthetic antibodies) are an in-vivo (directly tested in the animal model) approach, they avoid the majority of the bench testing, saving several years and approximately 35% of the R&D cost. The unique chemistry of aptamers, unlike other forms of drugs currently used, permits the natural selection of drug candidates in whole animal models, bypassing the test tube entirely. By using an animal model with the disease state of interest, Aptagen need not possess specific knowledge of the pathology or disease condition in question. As an added benefit, because this approach reduces the false starts, there are actually fewer animals needed for drug evaluation.
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