LHON Opportunity Next Steps

LHON Collective has the opportunity to become the global hub of a sustainable, interdisciplinary, patient-centric research ecosystem. Four high-priority opportunities where philanthropic investment and partnership could have a transformative impact have been outlined. Important to the development of LHON Collective’s strategy is understanding how the components of each opportunity might build on one another. For example, while biorepositories are natural pipelines for iPSC model development, there is an opportunity to build partnerships between existing repositories, for example, BRAIN UK and RARE-X to support patient-derived tissue and cell models while also simultaneously supporting the development of zebrafish models, which are not dependent on patient samples. 

This would shorten the runway to drug screening and therapeutic development by producing important foundational research and drug development tools as the LHON research ecosystem is capable. The below graphic outlines the anticipated timeline and order of program build based on the foundational opportunities presented in this opportunity map. Determining which partners to leverage and when, as LHON Collective leads the build-out of a research and therapeutic development pipeline, will be another key part of the strategy. Leveraging existing frameworks and developing partnerships to align on the opportunities presented will accelerate progress with the potential to increase the scope and scale of the research ecosystem.