Novel Ex Vivo Organoid Model Platform for Drug Development in Cancer and Cardiovascular Disease

Summary

• A key roadblock in successful drug development is the inability to model human or patient specific conditions in animal models
• The ability to replicate key biological features of human organs in the form of ex vivo organoids has enabled the creation of valuable models for studying human biology and disease
• The Englander Institute for Precision Medicine (EIPM) is launching the next phase of its Ex Vivo Models Platform
• This platform will synergize existing research platforms, innovative tools and computational biology expertise to fast track pre-clinical breakthroughs in new drug development in cancer and cardiovascular disease
• The EIPM is comprised of leading KOLs and experts who have developed this impressive platform and continue to build on its potential to fast track pre-clinical breakthroughs

Technical Overview

• The EPIM will leverage its unique resources to develop a novel organoid platform to model human diseases. Key features include:
• An expansive, living biobank-A comprehensive and unique tumor organoid biobank (250+ models) provides a discovery/validation platform complemented by first-in-class, clinical-grade genomic assays for interrogating tumor samples and their landscape
• Ability to perform high throughput drug screening-high-throughput screening methods, robotic automation, and AI will assist rapid testing and testing of thousands of drug candidates and prediction of drug-target interactions for more efficient drug development
• Generation of immune-competent organoids-EIPM is developing immune-competent human cancer models for functional and mechanistic discovery that contributes to treatment response vs resistance
• Assessment of mechanisms of resistance to treatment-EIPM Investigators can use CRISPR gene editing on human organoid models to manipulate specific features, insert genomic variants and replicate precise conditions in order to model specific disease conditions and identify novel vulnerabilities to target

Market Opportunity

• The ability to manipulate ex vivo organoids models with relative ease has created a powerful, potentially unlimited tool that will shape the future of drug development and medical discovery
• The FDA Modernization Act 2.0 was signed in December 2022, expanding the types of models allowed for drug testing and approval beyond animal models, which could include organoids
• Big pharma (including notable strategics such as Roche) have recently invested in the development of organoid models in a broad set of indications in order to speed up drug discovery and development
• The EIPM is in a position to deploy and/or provide this platform in the framework of a dedicated research collaboration in order to drive new drug development in a cost-effective manner

Partnering Opportunity

Weill Cornell Medicine is seeking an industrial partner with deep domain expertise and a strong presence in the personalized medicine and ex vivo organoid space to advance the development of this platform

Figure 1: Breast tumor organoid *Sshah74. “Heterogeneous breast tumor in 3D” ( Source ). License: CC BY SA 4.0.