OHIO STATE UNIVERSITY
A Safety and Efficacy Study of AAV2-hAADC for AADC Deficiency
A Safety and Efficacy Study of AAV2-hAADC for AADC Deficiency is a funding opportunity from OHIO STATE UNIVERSITY, up to 2242039, closing August 31, 2027.
Aug 31, 2027
(396d left)Overview
The goal of this proposal is to further evaluate the safety and feasibility of gene transfer to provide aromatic L- amino acid decarboxylase (AADC) enzyme into the midbrain of patients with AADC deficiency and continue Biologics License Application (BLA)-enabling studies as per FDA recommendations. AADC deficiency is…