Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors

This article describes our current understanding of fundamental biological principles and possible procedures (emphasizing those related to toxicology and toxicologic pathology) needed to support research and development of in vivo GTx products. This article is not intended to provide comprehensive guidance on all GTx modalities but instead provides an overview relevant to in vivo GTx generally by utilizing recombinant adeno-associated virus-based GTx-the most common in vivo GTx platform-to exemplify the main points to be considered in nonclinical research and development of GTx products.PMID:34657529 | DOI:10.1177/01926233211041962

Source: Toxicologic Pathology - October 18, 2021 Category: Pathology Authors: Julie A Hutt Basel T Assaf Brad Bolon Joy Cavagnaro Elizabeth Galbreath Branka Grubor Lisa M Kattenhorn Annette Romeike Laurence O Whiteley Source Type: research