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AAPS – PHARMSCI 360 2021

October 17, 2021 — October 20, 2021
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Synopsis

The American Association of Pharmaceutical Scientists (AAPS) is a professional, scientific organization of approximately 7,000 individual members and over 10,000 actively participating stakeholders employed in academia, industry, government, and other pharmaceutical science related research institutes worldwide. We’re thrilled to be joining virtually at this year’s AAPS Pharmsci 360 annual meeting so be sure to check out our on-demand presentation or submit a contact us form to learn more.

Speaker Spotlight

S. Jordan Kerns, PhD

Director, Applied Biosciences

Abstract

Presentation available on-demand from October 11th – 20th.

With favorable patient tolerability and the ability to transduce diverse cell types, recombinant AAV vectors are the leading platform for clinical gene delivery and show great promise in the treatment of a variety of human diseases. Recent advances in capsid design and optimization of genetic payloads have further improved both the efficiency of transduction and clinical outcomes of AAV treatment. Despite this progress, the interaction between AAVs and host-specific cellular factors, key determinants of engineered AAV therapeutic potential, are poorly understood because of the lack of preclinical model systems that recapitulate clinical observations. To overcome these challenges, Organs-on-Chips incorporate primary cell-types into precisely controlled microenvironments that enable better prediction of human responses to therapies. Here we present proof-of-concept results from our Human Liver-Chip, a co-culture of primary hepatocytes and liver sinusoidal endothelial cells, treated with AAV constructs expressing a reporter GFP transgene. We observe a dose and time-dependent increase in GFP signal and confirm that the route of administration is an important determinant of transduction efficiency. Additionally, we will show that AAV treatment does not significantly affect levels of albumin production or transaminase release indicative of tolerability and a positive clinical safety profile. The Human Liver-Chip has previously been shown to predict drug-induced liver injury, and can now be similarly used to better understand host-specific regulation of engineered AAV therapeutics.

About Emulate

Emulate Inc. is a privately held company that creates advanced in vitro models for understanding how diseases, medicines, chemicals, and foods affect human health. Our lab-ready Human Emulation System® includes three components: Zoë® Culture Module, Organ-Chips, and analytical software applications. The platform provides a window into the inner workings of human biology and disease—offering researchers a new technology designed to predict human response with greater precision and detail than conventional cell culture or animal-based experimental testing. Each of the Emulate proprietary Organ-Chip models—including the liver, intestine, and kidney—contains tiny hollow channels lined with tens of thousands of living human cells and tissues and is approximately the size of an AA battery. An Organ-Chip is a living, microengineered environment that recreates the natural physiology and mechanical forces that cells experience within the human body. Our founding team pioneered the Organs-on-Chips technology at the Wyss Institute for Biologically Inspired Engineering at Harvard University. Emulate holds the worldwide exclusive license from Harvard University to a robust and broad intellectual property portfolio for the Organs-on-Chips technology and related systems. For more information, please visit emulatebio.com.

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