CAMBRIDGE, Mass. & MADISON, Wis.--(BUSINESS WIRE)--Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative gene therapies for rare genetic diseases that affect the central nervous system (CNS), and Waisman Biomanufacturing, a non-profit gene and cell therapy development and manufacturing group located at the UW-Madison Waisman Center, (Waisman) announced today that the companies have entered into an exclusive partnership agreement for the production of Agilis’ novel gene therapy product, AGIL-FA, for the treatment of Friedreich’s ataxia (FA). FA is a debilitating multi-system disease arising from mutation of the FXN gene. It is the most common inherited ataxia, with 1 in every 100 people being carriers of a mutated FXN gene. FA typically arises between the ages of 5 and 15 and manifests as difficulty with balance and coordination. Over time, the disease progresses to an array of neurological symptoms and life-altering changes in mobility, energy, speech, hearing, and other body systems including the cardiovascular system, which collectively reduce longevity in many cases.
