Enabling next generation cardiac therapeutics with genetic engineering and novel in vivo models for cardiomyocyte transplantation
The development of cellular therapeutics is acutely dependent on the ability to evaluate the functional characteristics of the cells in predictive animal models. This forms the basis of key pre-clinical data packages that are key for regulatory submissions preceding human clinical trials. The development of appropriate model systems, the execution of the surgical techniques to deliver cells to the target tissue, and the techniques to functionally analyze these cells in situ are technically challenging. Michael Laflamme's laboratory is focussed on the development of protocols for making and testing human stem cell derived cardiomyocytes in animal models and is the world leader in this field. BlueRock Therapeutics (BRT) is actively developing a clinical and commercial pipeline of cell therapies for cardiac indications. This proposal bridges development work being done in the Laflamme lab and at BRT. The data that will be generated in the project will be instrumental in shaping BRT's development pipeline. Further, the project will provide Wahiba, the proposed trainee, with an opportunity to work with BRT staff to propose a business case and implementation plan for the internalization of this highly specialized skill set within the company.