AIM
SUMMA is aimed to advance microRNA mediated cardiac gene therapy to stimulate cardiac regeneration in mice towards commercial proof-of-concept. During this project, different Tasks are formulated to perform (1) efficacy studies in a large animal pig model of cardiac ischemia of this new experimental drug, (2) perform market research, (3) IP strategy development and (4) business development activities to maximize the value of the project’s results.
RESULTS
A subtype 6 AAV vector (AAV6) was created as an optimal intra-cardiac tool to deliver the miR-106b gene in a porcine model of chronic ischemia. Therapeutic benefit was measured by serial echocardiography, left ventricle catheter-based hemodynamic measurements and magnetic resonance imaging. Post-mortem analysis included gross organ examination, infarct size determination, histological parameters for proliferative markers (Ki67, phospho-histone H3, Aurora B), lectin for cardiomyocyte cross sectional area, CD45 for inflammation and virology (AAV DNA, microRNA DNA and RNA). Clinical chemistry, immunohistochemical data, liver and kidney sections of the porcine model were used as a first indication for absence of toxicological side effects. A clear regenerative potential was observed from delivering this microRNA cluster to the injured adult myocardium and no signs of unwanted toxicity effects were evident from the analyses performed. Additionally, the primary data were patented (WO/2017/036811) and a freedom to operate (FTO) analysis was completed to evaluate where our future products would potentially infringe. Finally, the most optimal marketing strategy was analysed and chosen that would secure the highest potential of commercial success, and a final business model was chosen with the most suitable success rate as the best route to commercialisation, yielding a business plan that is currently used for discussions with investors.
CONCLUSION
Our initial aim to demonstrate a commercial proof-of-concept in large animals to regenerate the infarcted heart was demonstrated in a pig model, the data were IP protected and a marketing strategy for optimal potential to commercial success documented in a business plan for a spin-out biotech company. This commercial potential has been added to list of major lead compounds in the spin-out biotech company Mirabilis Therapeutics BV.
