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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