Support the Mission
Move gene therapy from the laboratory toward patients.
Your gift helps power the Mighty Mito Mission—a long-term $10 million philanthropic effort to accelerate COX20 gene-therapy development and help move promising science through the critical steps between the laboratory and clinical development.
Responsible stewardship
We direct philanthropic support toward the specific research and development milestones needed to advance the COX20 program.
Transparent progress
We report on milestones openly, so you can see where the program stands, what your support is helping make possible, and what comes next.
Scientific rigor
Our priorities are grounded in the best available evidence and informed by researchers and experts working across mitochondrial disease and gene therapy.
Every gift helps move the program forward
Developing a gene therapy is a carefully sequenced process. Support helps make the work required at each stage possible.
Research
Building the scientific foundation
Preclinical
We are hereEvaluating efficacy, dosing, delivery, and safety
Manufacturing
Developing the processes needed to produce a clinical-grade therapy
Regulatory
Preparing the data and materials needed for regulatory review
Clinical Trial
Testing the therapy in people, if and when the program is ready
Patients
The goal: therapies that can ultimately reach the children who need them