Agilis Biotherapeutics develops DNA therapeutics for patients with rare diseases of the central nervous system (CNS) that single-gene defects cause. Its technology enables precise targeting and restoration of lost gene function while avoiding unintended off-target effects. Agilis Biotherapeutics also provides DNA therapeutics for patients withAADC Deficiency, a rare CNS disorder arising from reductions in the enzyme aromatic L-amino acid decarboxylase (AADC) that causes the inability to develop any motor strength and control resulting in breathing, feeding, and swallowing problems. Moreover, it offers DNA therapeutics for Friedreich’s Ataxia, a rare and life-shortening neurodegenerative disease caused by a single defect in the FXN gene, which causes reduced production of the frataxin protein, and DNA therapeutics for Angelman Syndrome (AS), a rare genetic, neurological disorder characterized by severe developmental delays. Agilis Biotherapeutics has a strategic partnership with Intrexon Corporation. It began its operation in 2013, with its headquarters in Lynnfield, Massachusetts.

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Agilis Biotherapeutics

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