FARA is announcing a request for proposals aimed at supporting therapeutic development in FA, by providing key preclinical data to support the development of benchmarks to improve the likelihood of success of frataxin delivery or upregulation.
Many therapeutic interventions in FA are aimed at elevating the levels of frataxin protein with small molecules or delivering frataxin to the affected tissues by means of gene, protein replacement or stem cell therapy. However, there are critical pre-clinical studies that must be done to ensure that these interventions are successful.
Applications focused on addressing one or more of the following outstanding questions will be considered:
- What are the levels of frataxin that are needed to restore function in cells and tissues supportive of therapeutic benefit?
- What is the percent of cells that need to be targeted in a tissue to achieve a clinical benefit and what is the effect of frataxin increase/delivery in a subset of cells within a tissue?
- What is the contribution of non cell-autonomous effects on therapeutic benefit for relevant tissues?
- What are the temporal aspects of frataxin increase/delivery?