= Members Only
ACT sponsors quarterly webinars on current topics in toxicology, including primarily drug and chemical safety assessments. Presenters are experts in their field, with backgrounds in government, industry or academia. The goal of the webinar series is to provide accessible education on topics in toxicology to ACT members and, more broadly, members of the toxicology community worldwide. ACT Webinars are reaching thousands, with collaborations spanning the globe while increasing individual and corporate ACT membership and participation. The ACT Signature Webinars are approximately 90 minutes, which includes questions and answers.
An Overview of Nonclinical Considerations
Jennifer Daly and Jeffrey Moehlenkamp
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.
There is no fee but advance registration is required.