First-in-Class Gene Editing Platform
Primera Therapeutics is focused on addressing the root cause of mitochondrial diseases by developing a first-in-class gene editing platform to target inherited mutant mitochondrial DNA. Primera’s first approach uses a fully customizable TALE-based system to specifically target and repair the mutant DNA in the mitochondrial genome. This application enables precision gene editing to be deployed at the mitochondrial level for the first time.
Primera is leveraging mtDNA gene editing therapies developed in the Mayo Clinic lab of Dr. Steven Ekker and Dr. Karl Clark and an innovative toolbox with the support of key strategic partners including Cellectis, Mayo Clinic, the Children’s Hospital of Philadelphia, the United Mitochondrial Disease Foundation and others. With a solid scientific and technical foundation, Primera seeks to accelerate the development of mitochondrial disease cures, be first to the clinic and rapidly support this undeserved patient population.
While there are hundreds of mitochondrial mutations that have been described, most adults with mitochondrial diseases have a m.A3243G mutation, which alters the code for a mitochondrial leucine tRNA. The mutation in this tRNA causes random amino acid misincorporation throughout the mitochondrial proteome, resulting in a range of symptoms in numerous organ systems.
Many adults with the m.A.3243G mutation will eventually develop chronic kidney diseases, typically resulting in the need for dialysis and eventually requiring kidney transplantation. Primera’s initial clinical focus is on editing renal progenitor cells from patients having the m.A3243G mutation that are in the early/mid stages of chronic kidney failure.
Inherited mitochondrial disorders caused by genetic changes in the mtDNA afflict adults and young children, and have very limited or have no treatment options. They can present across a large number organs and systems and often result in death. Primera will seek treatments for additional follow-on indications, such as diabetes and numerous CNS and musculo-skeletal disorders, in the future.