Preclinical Studies Of Gene Therapy Products: Latest Trends

A significant number of gene therapy (GT) products have progressed from preclinical to clinical development, with delivery approaches broadly categorized into viral vectors, where modified viruses carry the genetic material, and nonviral vectors, which utilize lipids, metals, or polymers. Each method offers distinct challenges and opportunities, particularly when translating therapies into animal models such as nonhuman primates (NHPs).

This presentation will explore key challenges and innovative solutions related to GT delivery in NHPs, including how to model an immunosuppressive regimen, considerations for study designs, methods for investigating germline integration through sample collection and PCR techniques, and the importance of age categories in selecting NHP models.

The session will be of particular interest to biotechnology and pharmaceutical companies developing GT products, as well as academic researchers in the field. Presenters include Norbert Makori, BVM, MSc, Ph.D., DABT, Vice President of Toxicology at Altasciences, and Kaylin Koenig, Ph.D., Principal Scientist at Altasciences.