What is an aptamer?
Aptamers (synthetic antibodies) are (stable) single-stranded DNA, RNA, or peptide molecules capable of binding to its target antigen with high affinity and specificity. Aptamers (synthetic antibodies) have been developed against a wide variety of targets ranging from small organics to large proteins.
Aptamers Against Virtually Any Target!
Proteins. Organics. Toxins.
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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Aptagen's rapid fast-track approach to drug discovery bypasses the 'test tube' in pre-clinical development, taking it one step closer to human clinical trials.
In Vitro Assays -----> In Vivo Studies ------> Clinical Trials
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 aptabodies™ (patent pending), overcomes the limitation of the traditional pharmaceutical drug discovery process. The unique chemistry of aptabodies™, 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.
BENEFITS: overcome delivery issues * * identify unknown biomarkers * * tailor PK/PD properties * * rescue failed drug candidates from targeting/specificity issues
Spending too much time and capital on the trial-and-error approach to Drug Discovery? On average, only one in a thousand drug candidates make it to human clinical trials. The cost and time associated with the traditional method of drug development is expensive and quite slow (7 to 15 years and almost $1 Billion). However, Aptagen's technology significantly reduces the unpredictability, cost, and time for drug discovery and development.
Over a century ago, Paul Ehrlich described the ideal drug, famously known as a 'magic bullet,' in which it specifically targets the disease site and would not harm healthy tissue. However, finding these magic bullets with drug-like properties involves a trial and error approach and is extremely slow. Aptagen has developed the concept of a ‘replicating magic bullet’ called an aptabody™. The aptabody™ technology has a shorter development time compared to traditional methods of drug development, and can be used to increase the specificity and improve delivery of existing drug compounds resulting in the next generation of pharmaceuticals with improved pharmacodynamics and pharmacokinetics.
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It's not an antibody. It's not an aptamer.
It's an "aptabody".
What is an aptabody™? An aptabody™ is a supramolecular form of an aptamer ligand containing a wide variety of functional groups. The aptamer component acts as a scaffold to hold into place one or more functional groups such as amino acids, fatty acids, carbohydrates, small molecules and/or metals into a unique orientation in 3-D space. The order and proximity of these functional groups play a role in enhanced functionalities ranging from higher affinity and specificity over “naked” aptamers, to enzyme biomimetics, improved molecular targeting in vivo, cellular trafficking, pharmacokinetics/dynamics, and drug-like properties.
NOTE: Conventional antibodies have a chemical alphabet of the naturally occurring amino acids. Aptabodies™, by definition, have a chemical alphabet of all amino acids, fatty acids, sugars, as well as synthetic compounds, co-factors, metal ions, etc...