Accelerating gene therapy for ultra-rare mitochondrial disease
Turning mitochondrial science into treatments for patients.
Mighty Mito is working to accelerate gene therapy research for ultra-rare mitochondrial diseases, beginning with COX20 deficiency.
For too many children, a genetic diagnosis comes without a treatment.
Mitochondrial diseases are a diverse group of genetic disorders that can affect how cells produce energy.
For families living with ultra-rare mitochondrial diseases, the path from diagnosis to treatment can be long, uncertain, and difficult to navigate.
But genetic medicine is changing what is possible.
The why
Meet Felix
Felix is four years old. He is funny, determined, endlessly curious, and completely himself.
He also has an ultra-rare genetic disorder caused by changes in the COX20 gene, which is essential for mitochondrial function.
Currently, no approved gene therapy exists for COX20 deficiency. But researchers are working toward one.
The gap we're trying to close
From diagnosis to treatment, too much time can pass.
Promising science can take years to become a treatment. For ultra-rare diseases, that path can be even harder.
Mighty Mito exists to help close the gap between scientific discovery and the children waiting for what comes next.
We are building a focused, science-driven nonprofit dedicated to accelerating the development and translation of therapies for ultra-rare mitochondrial diseases—beginning with COX20 deficiency.
The opportunity
What if a child's genetic diagnosis could become the starting point for a treatment?
Gene therapy may offer a path forward.
Gene therapy aims to address disease at its genetic source by delivering a functional copy of a gene that is not working properly.
Researchers at the University of Massachusetts Chan Medical School are developing and testing a gene-therapy approach for COX20 deficiency.
Mighty Mito's role is to help move promising science toward patients.
The Mighty Mito mission
Felix is one child. The mission is much bigger.
Mighty Mito was founded in response to one family's experience with an ultra-rare mitochondrial disease—but our mission extends far beyond one child.
We believe promising genetic therapies should have a straightforward path from the laboratory to the patients who need them.
Our nonprofit is dedicated to accelerating the development and translation of therapies for ultra-rare mitochondrial diseases, beginning with COX20 deficiency.
Our vision
A future where a rare genetic diagnosis does not mean a lifetime without treatment.
The Mighty Mito Mission
Help us move gene therapy from the laboratory to patients.
Mighty Mito is building a $10 million philanthropic campaign to accelerate COX20 gene-therapy development through the critical steps between promising research and clinical treatment.
Our immediate goal is to fund the next critical stage of preclinical development and help build the foundation for eventual clinical translation.
Every milestone brings us closer to a future in which children with COX20 deficiency can have a treatment option.
Where your support goes
Your support moves science forward.
Scientific research
Supporting critical studies needed to advance COX20 gene therapy.
Preclinical development
Supporting efficacy, safety, dosing and other studies required before human treatment.
Manufacturing
Supporting development and production of clinical-grade gene therapy materials.
Regulatory preparation
Supporting the work required to prepare for FDA review and clinical development.
Clinical development
Supporting the infrastructure necessary to conduct a clinical trial when the therapy is ready.
Mighty Mito is committed to responsible stewardship, scientific rigor and transparency.
The science
Beautiful, understandable science.
COX20 helps cells assemble part of the machinery mitochondria use to produce energy. In COX20 deficiency, that process is disrupted.
The scientific hypothesis behind gene therapy is simple to state: deliver a functional copy of the gene, and cells may be able to produce functional COX20 again.
COX20 Community
Have COX20? We'd love to hear from you.
Whether you're newly diagnosed, have been navigating COX20 deficiency for years, or are simply looking for others who understand, you're welcome here.
Connect with the Mighty Mito communityThere are many ways to move the mission forward.
Whether you give, connect us with others, or help us build, every contribution brings a treatment closer.