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

BOSTON–(BUSINESS WIRE)–Emulate, Inc., a leader in next-generation in vitro models, today announced the launch of its new adeno-associated virus (AAV) transduction application for the liver chip, which enables gene therapy researchers to assess the delivery efficiency and safety of AAV vectors in a validated, human-relevant liver and liver model testing gives you results in weeks, not months like animal models. This technology allows them to rapidly iterate on the AAV design to optimize gene therapy delivery and accelerate development.

“The ability to more quickly optimize AAV design with this application is a game changer for the gene therapy industry,” said Jim Corbett, CEO of Emulate. “We are pleased to offer this novel application to researchers in biopharmaceutical, academic and government settings around the world who are exploring gene therapy as a potential to treat a broad spectrum of diseases such as cancer, cystic fibrosis, heart disease, diabetes, etc. This is a metabolic disease.”

Gene therapy involves replacing a faulty or missing gene by adding a new gene into the body’s cells to treat or prevent a genetic disease or disorder. Currently, this technique is mainly available in clinical trials testing its potential to treat hereditary diseases, 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 because it efficiently targets different cell and tissue types and has been shown to be safe and well-tolerated.

Due to the lack of suitable non-clinical models, when developing new AAV vectors, scientists often face difficulties in ensuring that the vector will effectively and safely deliver the therapy to the right cells in the right organ. Animal models are slow, expensive, 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. Also conventional in vitro Models limit the number of AAVs that can be tested because they are just a single cell type in a static petri dish and do not accurately reflect how cells behave in the body.

“The Emulate Liver chip provides the specific 3D multicellular architecture, physiological functions and mechanical forces required to recapitulate the relevant aspects of the liver,” said Lorna Ewart, PhD, Emulate’s chief scientific officer. “Now we have shown that it is a more human-relevant model that allows researchers to assess and discriminate the transduction efficiency of different AAV-based gene therapies in a concentration- and time-dependent manner, as well as to assess toxicity. ”

Information on the Liver chip’s Emulate AAV transduction application is available on the company’s website and will be featured in a live webinar on October 6thth.

About Emulate, Inc.

Emulate ushers in a new era of 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 – providing researchers with innovative technology designed to predict human response with more precision and detail than traditional cell culture or animal-based experimental tests. Developed at the Wyss Institute for Biologically Inspired Engineering at Harvard University and supported by Northpond Ventures, Founders Fund and Perceptive Advisors, the organ-on-a-chip technology empowers researchers in academia, pharmaceutical and government industries with its predictive power and human capabilities Recreate lifelike biology. To learn more visit emulatebio.com or follow us on LinkedIn and Twitter.

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