Gene Therapy Research & Drug Development

Gene therapy holds enormous promise for treating inherited and acquired diseases, but predicting human response is challenging due to complex mechanisms of action, limited clinical data, and a lack of predictive non-clinical models.

A key challenge gene therapy researchers are working to overcome is the development of gene delivery vehicles—including viral and non-viral vectors—that are both efficient and safe. Unfortunately, conventional models routinely fail to predict the potency and toxicity of these vehicles. The limited complexity of conventional in vitro models results in non-physiological response. Meanwhile, the species translation issue for animal models is profound, with two studies showing a ~20-fold higher transduction efficiency in mouse liver cells compared to human. Additionally, the long timelines, high costs, and rigid designs of non-human primate studies limit researchers’ ability to rapidly test and iterate on gene therapy delivery vehicles.

Obtain results more likely to translate to human response by assessing gene therapy vectors in a more physiologically relevant microenvironment.

With a variety of Organ-Chip offerings, researchers can evaluate the toxicity and tissue-specific tropisms of both viral and non-viral vectors—a key challenge when developing gene therapy. To model intravenous infusion of gene therapy vectors, the two-channel design of Emulate Organ-Chips can be used to assess gene therapy vector transport from the vasculature into the target tissue, as shown in proof-of-concept studies.