Agilis Biotherapeutics

About:

Agilis Biotherapeutics designs and engineers DNA-based therapeutics for genetic disorders.

Website: http://www.agilisbio.com

Twitter/X: agilisbio

Top Investors: Sands Capital Ventures

Description:

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 with AADC 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.