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FARAFARA Cure FA

Agilis Biotherapeutics Updates on Progress in CNS Gene Therapy Programs

Agilis Therapeutics announced the completion of the Company's Phase IIb clinical study of gene therapy for the treatment of Aromatic L-amino acid decarboxylase (AADC) deficiency and the preparation of a Biologics License Application (BLA) for submission to the FDA. In addition to being a milestone for the AADC community, advances in the AADC program are informative for the company's Friedreich's ataxia (FA) program. The Company also announced that the program in FA, AGIL-FA, an AAV-based vector for delivery of the human FXN gene intended to address the CNS manifestations of FA, is advancing rapidly through nonclinical, manufacturing and regulatory activities toward human clinical study.

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Jen Farmer

Jen Farmer

Executive Director

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