Development of Next Generation Therapeutics for NAFLD/NASH

Summary

• Non-Alcoholic Fatty Liver Disease (NAFLD) and its progressed form, Non-Alcoholic Steatohepatitis (NASH), affects ~100 million people of all ages in the United States
• The Englander Institute of Precision Medicine (EIPM) and Weill Cornell Medicine have developed multi-disciplinary efforts to improve the diagnosis, treatment, and prevention of NALFD/NASH
• WCM has developed a highly effective research-oriented, multidisciplinary treatment center to treat NALFD/NASH and provide the samples needed to make advances in NALFD/NASH research
• The EIPM has developed an innovative deep target mining platform to discover novel targets using state of the art single cell sequencing and spatial-omics

Technical Overview

• NALFD/NASH is a multi-system disease
• Targeting cells in the liver has the potential to treat other conditions associated with NALFD/NASH
• The EIPM will use patient samples at different stages of disease progression and resolution in order to identify key drivers
• This approach combined with spatial profiling technology at EIPM will also allow the team to elucidate the contribution of specific immune cell types to disease progression
• The EIPM has also developed a state-of-the art patient-derived 3D multi-cell type liver model, that accurately represents the physiological behavior of liver cells, to do high throughput screening of novel targets
• All together this approach will be a powerful tool to identify key mediators of NAFLD/NASH, assess therapeutic potential of identified targets and evaluate potential therapy response

Market Opportunity

• The Englander Institute offers clinical and research expertise in the multi-system complexity of metabolic, multisystem liver diseases
• The EIPM’s cutting-edge technology enables innovative deep target mining using single cell, spatial-omics, genomics, imaging, and high-throughput screening
• The EIPM has access to a unique clinical and patient sample biobank and unprecedented preclinical models to test and validate new targets
• This potentially transformational approach will not only impact NALFD/NASH treatment, but it has therapeutic implications for other disorders as well

Partnering Opportunity

Weill Cornell Medicine is seeking an industrial partner with deep domain expertise in the liver space to leverage the EIPM’s infrastructure and platform for the development of novel therapeutics

Supporting Data / Figures

Figure 1: Overview of deep target mining and validation platform.