Rare Genetic Mitochondrial Diseases
Rare genetic mitochondrial diseases such as Barth syndrome and [POLG-related] mitochondrial myopathy are associated with severe muscle weakness, which can impact all muscles in the body and lead to debilitating fatigue. Mighty Therapeutics developed the first, and currently only, approved therapy for Barth syndrome. There are currently no FDA or EMA approved treatments for POLG-related mitochondrial myopathy.
Ophthalmic Diseases
Dry age-related macular degeneration, or dry AMD, which is the leading cause of blindness among older adults in the developed world, is characterized by symptoms such as distorted or blurred vision, reduction in low luminance visual acuity and reduced overall visual acuity. We estimate that dry AMD impacts more than 10 million individuals in the United States. There is a significant unmet medical need for safe and less invasive therapies that permit earlier intervention in the trajectory of this progressively blinding disease to protect and potentially improve visual function.
Neurodegenerative Diseases
Some neurodegenerative diseases, including Parkinson’s disease, Leigh syndrome, and Friedreich’s ataxia, are linked to mitochondrial dysfunction. These diseases are progressive, debilitating, and affect multiple organ systems. Leigh syndrome is fatal, with most children passing away by age 3, and treatment is focused on palliative care. Treatment options for Parkinson’s disease and Friedreich’s ataxia are primarily limited to symptom management.
