AAV-frataxin

AAV-frataxin refers to a class of investigational gene therapies designed to treat Friedreich's Ataxia (FA), a rare neurodegenerative disorder caused by a deficiency of the mitochondrial protein frataxin. These therapies utilize adeno-associated virus (AAV) vectors, such as AAV9 or AAVrh10, to deliver a functional copy of the human FXN gene to affected tissues, primarily the heart and central nervous system. By restoring frataxin expression, the treatment aims to improve mitochondrial function, reduce oxidative stress, and prevent iron accumulation, thereby halting or reversing disease progression. Key clinical-stage programs include Lexeo Therapeutics' LX2006 and Rocket Pharmaceuticals' RP-A501, which focus on addressing the life-threatening cardiomyopathy and neurological symptoms associated with the disease.

Other names

AAV-frataxin gene therapyAdeno-associated virus frataxin