The goal of research and development at Vascular Research Group (VRG) is to develop new therapeutic or diagnostic molecules from combination libraries composed of animal toxins. One of the major strengths of the group is the unique, Global access to the animal toxins and a pharmaco-genomic drug discovery platform called Designer Toxin based on a special natural peptide toxin collection. Due to the Designer Toxin technology, the largest (> 1.5 million elements) target molecule oriented animal toxin-based combination library has already been created, which has resulted in patent of the new active ingredients. The VRG with its knowledge and resources focuses on developing lead molecules that are effective against a variety of diseases, such as cancer, autoimmune or other diseases or diseases can be targeted by gene therapy.
One of the main obstacles in drug discovery is to find new and high-potential lead molecules – identifying the compounds, future drug development can be based on. In addition, the lack of selectivity, weak mechanism of action or pharmacokinetics, and elimination of side effects of drugs currently available also constitute significant challenges for developers of new therapeutic agents. Accordingly, from cardiovascular disease to cancer, from autoimmune to nervous system diseases, there is a huge unmet need to develop new molecules with specific properties designed for the therapeutic need of diseases.
Animal toxins are unique molecules on Earth as they have been selected by evolution to kill a living creature within a minute. Toxins target vital receptors, binding with high affinity, are quite selective, and often have a therapeutically beneficial mechanism. Therefore, toxins offer unparalleled lead molecules to develop new drugs, diagnostic tools, and bioengineering applications. There are currently close to 20 animal toxin based drugs on the market (along with 500 animal toxins known) to treat heart attacks, high blood pressure, diabetes, chronic pain, and thrombosis. Yet we do not know the 20 million toxin molecules of 150,000 venomous animals on the planet.
The Designer Toxin technology platform creates a highly diverse family of compounds based on naturally occurring template molecules, and identifies the most promising ones based on the desired properties. It has been developed with state-of-the-art functional genomics and combinatorial chemistry for scalable large number of samples. Typically, for templates we take the series of animal toxins with characterized pharmacology. Then we use their diversity to develop lead compounds with completely new properties against new molecular targets. The key elements of the platform are the creation of more than a million molecular variants, evolutionarily “tested” molecular motifs, and building a library focusing on the target molecule.