Emulate Launches Liver-Chip Application to Accelerate Testing of AAV Gene Therapies

Emulate launched its new adeno-associated virus (AAV) transduction application for its Liver-Chip that enables gene therapy researchers to test the delivery efficiency and safety of AAV vectors in a validated, human-relevant model of the liver and get results in weeks rather than months.

Photo: Lorna Ewart, Emulate Chief Scientific Officer

“The ability to more rapidly optimize AAV design using this application is a game-changer for the gene therapy industry, said Emulate CEO Jim Corbett.

Gene therapy involves replacing a faulty or missing gene by adding a new one inside the body’s cells to treat or prevent a genetic disease or disorder. Currently, this technique is primarily available in clinical trials, testing its potential to treat inherited disorders, cancer, and HIV/AIDS. The AAV vector is the most versatile and popular viral vector that researchers use as a delivery vehicle for gene therapy, as it efficiently targets different cell and tissue types and has been demonstrated to be safe and well-tolerated.

Due to the lack of suitable non-clinical models, scientists often struggle when designing new AAV vectors to ensure that the vector effectively and safely delivers the therapy to the right cells, in the right organ. Animal models are slow, costly, and tightly regulated, which limits the number of AAV delivery vehicles that can be tested and the ability to look at the individual contribution of each cell type. In addition, conventional in vitro models restrict the number of AAVs that can be tested as they are only a single cell type in a static petri dish and do not accurately reflect how cells behave inside the body.

“The Emulate Liver-Chip provides the specific 3D multicellular architecture, physiological functions and mechanical forces necessary to recapitulate the relevant aspects of the liver,” said Emulate Chief Scientific Officer Lorna Ewart. “Now we have demonstrated it to be a more human-relevant model that researchers can use to assess and discriminate between the transduction efficiency of various AAV-based gene therapies in a concentration- and time-dependent manner, as well as evaluate the toxicity.”

Author: Rare Daily Staff