TREAT MITO Mission and Vision

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To organize and oversee a unified pipeline of therapeutic opportunities in mitochondrial disease by bringing together the various stakeholders required for effective therapeutic development. 

A world with a robust market of FDA-approved treatments and cures for mitochondrial disease, improved medical care, health outcomes, and quality of life for all patients with mitochondrial disease. 

TREAT MITO was created to bring multiple mitochondrial disease stakeholders together harnessing individual strengths and funneling community efforts into a unified, patient-centered approach to treating mitochondrial disease from bench to bedside. The primary objective of TREAT MITO is to convene, organize and oversee a unified research network for therapeutic development in mitochondrial diseases. This includes further developing and maintaining existing research programs such as a patient-populated registry, clinical research consortium, biorepository, genomic database, clinical care network and industry projects. The programs span early drug development efforts, translational research, clinical trial readiness activities such as developing patient-centered outcome measures for therapeutic studies, evaluating and supporting early clinical development of promising therapeutics, and facilitating multi-center clinical trials to streamline drug development. 

One of the pressing challenges that the mitochondrial disease community faces is the inability to date to translate rapidly growing interest from drug developers to approved therapeutics. Multiple failed clinical trials in the space have demonstrated the urgent need for a more integrated research network that can work toward shared, prioritized goals that are patient-centric. Faced with a tidal wave of pre-clinical activity prepared to translate, urgently building out a clinical trial consortium to oversee therapeutic development was identified as a priority. A unified effort to prioritize products with the highest yield of success and thoughtful trial planning is paramount for primary mitochondrial diseases. 

The development of a patient-prioritized research agenda in mitochondrial disease, from basic through to clinical, greatly benefits the community by ensuring research aligns with the most pressing needs and concerns of those affected. Engaging the research network and patient community in this effort involves soliciting input through surveys, focus groups and collaborative events. The resulting agenda enhances the relevance and impact of research, fostering a sense of community ownership, and creating a dynamic and responsive research network that directly addresses mitochondrial disease patient challenges.