Event Detail

BSTP & IATP Educational Webinar: Translational Considerations of NonClincial Findings in AAV-vector Gene Therapy

Monday, May 13, 2024

6:00 – 7:30 AM US PDT
9:00 – 10:30 AM US EDT
2:00 – 3:30 PM UK
3:00 – 4:30 PM Europe
6:30 – 8:00 PM India
9:00 – 10:30 PM China
10:00 – 11:30 PM Japan/Korea

This webinar will provide a detailed look at the nonclinical studies employed to characterize the emergent nonclinical, nonhuman primate microscopic finding of dorsal root ganglia toxicity in a preclinical program and how these findings influenced clinical monitoring and product label for the AAV9 gene therapy product onasemnogene abeparvovec indicated for spinal muscular atrophy. The natural of history of spinal muscular atrophy will be briefly reviewed along with the components of the AAV9 gene therapy construct and the nonclinical program developed to support its development. Finally, we will review, from a translational medicine and toxicology perspective,  the potential for and considerations of potential clinical translation of nonclinical nonhuman primate microscopic DRG findings.

Speakers: Emily Meseck and Tukov, Francis Fonyuy