Primera Therapeutics is pioneering innovative gene editing therapies specifically targeting mitochondrial diseases, which affect a significant number of individuals in the United States. The company is focused on addressing the root causes of these diseases by utilizing a first-in-class gene editing platform designed to target inherited mutations in mitochondrial DNA (mtDNA). Their approach employs a customizable TALE-based system that allows for precise editing of the mitochondrial genome, marking a significant advancement in the ability to correct dysfunctional mitochondria at a genetic level. This technology aims to provide new treatment options for conditions that currently have limited or no available therapies, particularly for inherited mitochondrial disorders.

The company is leveraging research and development efforts from esteemed institutions, including the Mayo Clinic, to enhance their gene editing capabilities. Their initial clinical focus is on patients with the m.A3243G mutation, which is linked to chronic kidney disease and other serious health issues. By targeting renal progenitor cells in patients experiencing early to mid-stages of chronic kidney failure, Primera aims to develop effective treatments that could significantly improve patient outcomes. In addition to kidney diseases, Primera plans to explore therapies for a range of other conditions, including diabetes and various central nervous system and musculoskeletal disorders, thereby broadening the impact of their gene editing technologies.

United States

Primera Therapeutics is a company that provides patient-centric gene therapies targeting the causes of mitochondrial disorders. The company focuses on developing treatments for rare mitochondrial diseases for which there are no approved therapies. Primera Therapeutics is pioneering a first-in-class gene editing platform to cure genetic diseases.

Primera Therapeutics

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