AmplifyBio company

Optimizing Preclinical AAV Safety - Harmonization and Discovery

woman giving speech in hotel ballroom

Download the slides from Dr. Victoria Best’s talk at Optimizing AAV Safety 2022 about assessing preclinical safety signals derived from AAV gene therapies.  

Synopsis: Opportunities to Expand AAV Safety Data, Benchmarking, and Harmonization at the Preclinical phase

  • Issues the FDA is going to pay more attention to soon. Lab developed test validation, identifying correlates and biomarkers of safety

  • Opportunities within the preclinical and clinical community to harmonize their methods for analytical testing

  • Overlooked opportunities for gaining insight into preclinical data to inform the design and monitoring protocols that occur in the clinic

  • Overlooked opportunities for collaboration in the community, particularly amongst industry partners (data mining/sharing)