Emulate Announces New Gene Therapy Application Enabling Accelerated Development of Treatments for Genetic Diseases with Organ-on-a-Chip Technology

The new Emulate adeno-associated virus (AAV) transduction application for the Liver-Chip offers the quickest path to AAV optimization and design iteration

BOSTON--(BUSINESS WIRE)--Emulate, Inc., a leading provider of next-generation in vitro models, today announced the launch of its new adeno-associated virus (AAV) transduction application for the 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, not months, as with animal models. This technology allows them to rapidly iterate on AAV design to optimize delivery of gene therapies and accelerate development.

“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. “We are excited to offer this novel application to researchers in biopharmaceutical, academic, and government entities around the world who are exploring gene therapy as a potential to treat a wide range of diseases such as cancer, cystic fibrosis, heart disease, diabetes, and metabolic disease.”

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, PhD. “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.”

Information about the Emulate AAV transduction application for the Liver-Chip is available on the company’s website and will be presented in a live webinar on October 6^th.

About Emulate, Inc.

Emulate is igniting a new era in human health with industry-leading Organ-on-a-Chip technology. The Human Emulation System provides a window into the inner workings of human biology and disease—offering researchers an innovative technology designed to predict human response with greater precision and detail than conventional cell culture or animal-based experimental testing. Pioneered at the Wyss Institute for Biologically Inspired Engineering at Harvard University and backed by Northpond Ventures, Founders Fund, and Perceptive Advisors, Organ-on-a-Chip technology is assisting researchers across academia, pharma, and government industries through its predictive power and ability to recreate true-to-life human biology. To learn more, visit emulatebio.com or follow us on LinkedIn and Twitter.