Progress
Science doesn't move overnight. It moves milestone by milestone.
Developing a gene therapy is a long, carefully sequenced process. This is a living record of where the COX20 gene-therapy program stands, and we will update it as the work advances.
- 2026
Diagnosis, research connections, and Mighty Mito
Genetic testing identified variants in the COX20 gene, giving Felix a diagnosis of COX20-related mitochondrial complex IV deficiency. His family began connecting with researchers working to understand COX20 deficiency, and Mighty Mito was founded to help accelerate that work and support the path toward treatment.
- 2026In progress
Preclinical gene-therapy development
Researchers are advancing a COX20 gene-therapy approach through preclinical development, including work to evaluate efficacy, dosing, delivery, and safety. This is the current focus of the program.
- Future milestones
From preclinical development toward clinical translation
The next stages will depend on the results of the ongoing preclinical work and the requirements for advancing toward regulatory preparation and, ultimately, clinical development. We will add milestones and projected timelines as they are established with our research partners.
Watching the program advance
Next milestone
Complete the critical preclinical studies needed to determine whether and how the COX20 gene-therapy approach can advance toward regulatory preparation.
What it will take
Moving from promising preclinical data toward human treatment requires sustained support for efficacy, safety, dosing, delivery, manufacturing, and other development work.
What we're learning
Every study brings us closer to understanding how a COX20 gene-therapy approach could work—and what will be required to move it from the laboratory toward patients. We will share the progress, challenges, and next steps along the way.
Every milestone is powered by donors.
Progress on an ultra-rare disease depends on sustained, patient support. Help keep the program moving.