Primera Therapeutics develops gene editing therapies to correct mutations in mitochondrial DNA, the root cause of many inherited diseases. Their therapies aim to provide a curative approach for severe childhood diseases, chronic kidney disease, and metabolic disorders linked to mitochondrial dysfunction.
Funding
Funding not disclosed
Founders
Product
Problem
Mitochondrial diseases, affecting approximately 1 in 5,000 individuals, are caused by mutations in mitochondrial DNA (mtDNA). These mutations disrupt energy production within cells, leading to a range of severe childhood diseases, chronic kidney disease, metabolic disorders, and age-related conditions. Current treatments for these inherited mitochondrial diseases primarily address symptoms without targeting the underlying genetic cause.
Solution
Primera Therapeutics is developing gene editing therapies designed to correct mutations in mtDNA, addressing the root cause of mitochondrial diseases. Their approach utilizes a customizable TALE-based system to specifically target and repair mutant DNA within the mitochondrial genome. This enables precision gene editing at the mitochondrial level, offering the potential for disease-modifying treatments. The initial clinical focus is on editing renal progenitor cells from patients with the m.A3243G mutation who are in the early to mid stages of chronic kidney failure.
Target Audience
The primary target audience includes patients with inherited mitochondrial diseases, particularly those with the m.A3243G mutation and chronic kidney disease, as well as individuals suffering from other mitochondrial dysfunction-related disorders.
Features
- First-in-class gene editing platform targeting inherited mutant mitochondrial DNA
- Customizable TALE-based system for precise targeting and repair of mtDNA
- Focus on editing renal progenitor cells for chronic kidney disease treatment
- Potential application for a range of mitochondrial diseases, including severe childhood diseases, metabolic disorders, and age-related conditions