Joshua Todd, PhD, National Institute of Neurological Disorders and Stroke at the NIH, discusses the clinical development of adeno-associated virus (AAV)-based gene therapies for neuromuscular disorders.
The development of new genetic therapies is essential to treating the underlying cause of disease. AAV-based therapies are expected to grow in the following years with a focus on rare and neglected diseases since most of those conditions have a clear genetic pathophysiology. However, those same conditions are small in number and are most at risk for not being commercially viable therapies.
Currently, genetic therapy research and development is siloed across the NIH’s many centers. However, the concept of the Core for Advanced Genetic Therapies (CAGT) aims to centralize and vertically integrate clinical trial development. The objective is to optimize the design and execution of genetic therapy trials at the NIH to improve the lives of patients.
Through this program, in addition to centralization, an NIH Intramural Research Program (IRP) workforce would be assembled. These participants would be experts in genetic therapy research and development. Operational and budgetary efficiencies would be improved as well lowering the risk in clinical trials. Finally, researchers and clinicians would be trained in the design and conduct of gene therapy trials.
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Bönnemann Lab – Neuromuscular and Neurogenetic Disorders of Childhood Section: https://research.
NINDS Clinical Trials Unit (CTU): https://research.ninds.nih.
To learn more about rare neuromuscular disorders, visit https://checkrare.com/diseases/musculoskeletal-diseases/