Leveraging the Gut to Effectively Treat Disease
The gut is a critical system that plays a central role in many diseases by signaling to other organs how to respond to a variety of factors such as a meal, the microbiome or even pathogens.
UNDERSTANDING THE GUT
Our understanding of the gut or gastrointestinal (GI) tract is evolving on an almost daily basis, and the emerging view is that it is a critical and active sentinel organ that transmits signals to other organs throughout the body on how to respond to a meal, the microbiome or even pathogens.
We have created a unique discovery and design platform that integrates two critical concepts: (i) our proprietary chemistry capabilities that enable us to design and optimize gut-restricted compounds, which can provide a higher margin of safety than systemically absorbed compounds, and (ii) our stem cell-based translational technology called the Ardelyx Primary Enterocyte and Colonocyte Culture System, or APECCS, that enables us, at the lab bench, to discover the many targets the GI tract uses to control health and disease processes. Through this system, we are able to understand the biology that our targets control and thereby the mechanisms by which our compounds work to affect the physiological benefits we see in patients. Our platform can be applied across the entire GI tract, allowing for the broadest evaluation of disease targets to develop the best medicines for specific diseases.
The predictive ability of our platform enables us to better assess, at a very early stage, the potential for a small molecule compound to treat a disease.
HOW OUR PLATFORM WORKS
Why is this important? Gut-restriction chemistry is a body of proprietary techniques, technology and know-how that we have developed in order to restrict the mechanism of the drug to targets within the GI tract, reducing absorption, which we believe will provide a safety benefit.
Stem Cell Technology
The Ardelyx Primary Enterocyte and Colonocyte Culture System (APECCS) is an in-vitro capability, derived from human GI stem cells obtained from healthy and diseased patients, we’ve developed that mimics key aspects of the human GI system.
WHAT IS APECCS?
We have developed a unique cell-culture system that simulates the gut in-vitro (outside the body).
We call this component of our discovery platform Ardelyx Primary Enterocyte and Colonocyte Culture System, or APECCS. APECCS involves obtaining biopsies of various segments of the human gut from both healthy subjects and those with a disease of interest. We then grow those cells under proprietary conditions emulating the way that they would naturally grow in the body. We have developed this into a miniaturized format that allows us to utilize it for cell-based drug screening, target validation, and to understand at the cell and molecular level how our drugs work and how identified compounds may interact with specific gut tissues.