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An Overview of Nonclinical Considerations in Translating AAV Gene Therapies to the Clinic 

02-24-2021 11:16 AM

An Overview of Nonclinical Considerations in Translating AAV Gene Therapies to the Clinic

February 17, 2021


Laura Dill Morton, DVM, PhD, DABT, DACVP

About the Speaker:
Dr. Dill Morton received her bachelor’s degree and DVM from The Ohio State University and her PhD in Veterinary Pathobiology from the University of Illinois. She is a Diplomate of both the American College of Veterinary Pathologists and the American Board of Toxicology. After two years as an Assistant Professor at the University of Illinois in Urbana-Champaign, Dr. Dill Morton then transitioned into the pharmaceutical industry where she worked for more than 20 years at Baxter Healthcare, TAP Pharmaceuticals, Pharmacia, Pfizer Global R&D, and Novartis Institutes for Biomedical Research in a variety of interdisciplinary roles. In 2015, Dr. Dill Morton joined Aclairo Pharmaceutical Development group where she serves as a Senior Consultant, focusing primarily on cell and gene therapy and complex biologics.

Adeno-associated viral vectors are currently the most commonly used vectors for gene editing and gene replacement approaches in both CNS and ocular indications. The first part of the presentation will cover case by case considerations for making decisions about nonclinical program design to enable clinical trials of AAV-mediated gene therapies. These will include communication with clinical team members; interactions with regulatory authorities; planning for formulation and delivery of test articles; planning for analytical methods to support toxicology studies; decisions about model selection and justification; dose selection; and study design.
In the second part of the presentation, general regulatory expectations for nonclinical testing of a CRISPR-based gene editing therapeutic will be discussed, including expectations for evaluation of genotoxicity including off-target editing, characterization of gross chromosomal changes, and evaluation of tumorigenicity.

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February 17, 2021 Signature Webinar

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