Award
National Institute of Neurological Disorders and Stroke 5R01NS094292-09
A Safety and Efficacy Study of AAV2-hAADC for AADC Deficiency
Recipient
Ohio State University
Award Amount
$2,242,039.00
Ceiling
$2,242,039.00
Awarded
July 23, 2025
Identifier
5R01NS094292-09
This award funds a clinical trial led by Ohio State University to study the safety and efficacy of AAV2-hAADC gene therapy for patients with AADC deficiency, aiming to improve neurological function and pave the way for therapy registration.
Description
A multi-center clinical trial to evaluate the safety, tolerability, and efficacy of AAV2-hAADC gene therapy for AADC deficiency, including long-term safety assessment and optimized dose delivery procedures.