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Pretzel is tackling the roots of mitochondrial disease with an initial focus on mtDNA upregulation

Primary Mitochondrial Diseases

Mitochondria are “the powerhouse of the cell,” responsible for making cellular energy. Mitochondrial disease is caused when cells are not able to make the energy required. There are no effective or curative treatments available for mitochondrial disease; current approaches focus on supportive care and symptom management.

  • Prevalence Approx.
    1 in 5,000 people
  • Age of onset Varies
  • Causes Genetic

mitochondrial DNA
depletion syndromes

A sub-set of primary mitochondrial diseases (PMD), called mitochondrial DNA depletion syndromes (MDDS), are caused by severely low levels of mitochondrial DNA (mtDNA). When these syndromes manifest in childhood, MDDS have high rates of mortality and reduced life-expectancy, with a broad range of debilitating symptoms.

Pretzel is developing small-molecule medicines designed to upregulate levels of healthy mtDNA across multiple organs, including the brain, with the goal of improving survival, function and quality of life for those with mitochondrial disease.