We develop new treatments by combining our expertise in pharmaceutical research, biomedicine, neurosciences and veterinary sciences. Our research spans from molecular level to social sciences and will be strengthened in the crucial phases of the drug development pipeline, from drug discovery and drug delivery, to drug action and therapy, allowing us to find efficient novel regenerative and disease modifying drugs.
Our major aim within drug discovery research theme will be to discover smart small molecule compounds to stimulate body’s regeneration capacity to heal itself. This is encouraged by recent observations concerning cardiac regeneration.
Our research includes the identification of intracellular metabolic pathways critical for cell regeneration. In addition, we will identify and characterize potential drug targets and drugs by stydying metabolic signatures aggravated in experimental disease animal models (e.g. cardiac ischemia and stroke model) and by the identification of aberrant metabolic pathways in patient-derived pluripotent stem cells. We also screen libraries of large compounds, in collaboration with the University of Helsinki HiLIFE unit Finnish Institute of Molecular Medicine. The newly discovered metabolites and pathways will serve as a starting point to design and synthesize novel metabolite analogs with therapeutic activity. In this, we utilise our expertise in leading-edge pharmaceutical analysis, metabolomics, chemoinformatics and medicinal chemistry.
Our challenge will be the development of pharmaceutical formulations capable of delivering small molecular and biological drugs precisely to the site of action in the body, for example to the tissue needing regeneration. Delivery of an experimental compound to its site of action is a common bottleneck in drug development as this limitation prevents a thorough evaluation of the value of a new therapeutic principle.
Translation from basic biomedical discoveries towards treatments requires development of special site-specific and timely regulated delivery technologies for DNA, RNA and protein-based macromolecules. By building on our research on advanced drug delivery, we will focus on treatment of cardiovascular conditions and macular degeneration, the latter being a major cause of blindness in the elderly.
Our research efforts possess great potential for resolving pharmaceutical problems such as delivery of small molecule drugs to the brain and other “hard-to-reach” tissues, precision delivery of regenerative drugs to diseased organs and, as a result, avoiding severe adverse effects in other tissues; and the use of poorly soluble small molecule drugs by embedding them in nanocarriers.
Our research will be directed towards regenerative drug therapy, which means disease-modifying therapies of ageing-related diseases. Among them, heart failure and post-stroke recovery are major diseases with unmet medical needs.
Our aim is to solve the mechanism of action of experimental compounds that would possess regenerative capacity. We want to identify the signal cascades modified by the drugs that mediate their effects on tissue repair as well as validate the previously unknown drug targets by applying existing research tools and developing new reporter assays. We also aim to study the effects of a defined number of novel compounds on disease progression and tissue recovery in experimental models of heart failure and stroke.
We will analyze drug effects in humanized research models such as cardiac cells derived from induced pluripotent stem cells (iPSCs) from heart failure patients. We will also apply new knowledge on human genome and CRISPR/Cas9 technologies to improve the value of target validation and translation of preclinical research and drug development towards disease-modifying drugs.