HUMANOID CoRE Mission

Reducing translational gap in drug discovery

Phase '0'

Clinical trials are failing patients. Drugs must go through a pipeline of triphasic testing before they can be approved by the US FDA: phase 1 to primarily assess safety; phase 2 to confirm safety and assess some efficacy; and phase 3, the pivotal efficacy trial. Most drugs fail somewhere along this pipeline, many in the last phase because of a primary lack of efficacy.

HUMANOID ^TM is pioneering a new approach to human clinical trials by introducing phase '0'. Before proceeding to patients in the clinic, phase 0 tests efficacy in multi-dimensional human organoid-based disease models-in-a-dish, where ineffective compounds can be rejected early in the process. Currently, most drugs are validated in two-dimensional (2D), cultured cells, which usually represent just one cell type. This simplistic model fails to recapitulate the multi-dimensional nature of human tissues where multiple cell types (epithelial, stromal, immune) interact in 3-dimensions within the extracellular matrix, and some tissues have additional dimensions such as coexisting microbes. Using patient-derived stem cells to grow organoids, HUMANOID ^TM seeks to change the game by reverse-engineering these complex multi-dimensional healthy and diseased human tissues, one cell at a time.

Organs-On-A-Dish

HUMANOID ^TM isolates, expand, and biobanks 3D organoids from healthy and diseased human tissues, isolates and biobanks primary cells and develops models to study organ physiology and human disease. Any tissue with stem cells can be grown as an organoid. Likewise, if a model requires microbes, immune cells, or non-immune support cells, HUMANOID ^TM researchers can incorporate them, constantly refining the model to achieve greater complexity, as needed, to emulate key aspects of the disease. These models are informed by human pathophysiology and validated by PreCSN's disease maps, translating engineered tissues/organoids into appropriate models to screen for drug efficacy and toxicity. Because organoids can be specifically derived from multiple patients in any disease subtype, these organoid-based models represent human organs-on-a-dish, enabling human phase 0 clinical trials.

Accelerate Drug Discovery

Early efforts have validated patient-derived organoids as effective platforms to screen for novel therapeutics in inflammatory bowel disease, microbe-driven polyposis in the colon, hereditary colorectal cancer syndromes, chemical-induced lung injury, and gastric cancer, as well as therapies that reprogram stem cells within solid tumors. Ongoing efforts are developing models for NASH/fatty liver and Alzheimer's disease. These models are rigorously vetted to validate their ability to replicate human disease. The next step will be multi-organ models to further capture human biology.

HUMANOID ^TM's organoids replicate critical aspects of human disease, offering more efficient ways to test potential drugs for toxicity and efficacy and giving researchers and clinicians new tools to conduct more efficient clinical studies. These organoids can also test existing drugs or multi-drug cocktails to help individual patients. Results take only days, giving physicians an incredible resource to prescribe personalized medicines for each patient's unique disease.

HUMANOID ^TM's organoid-based models provide a head start on efficacy, giving researchers an incredible tool to separate useful therapies from dead ends. This is how we accelerate the drug discovery process and bring new hope to patients.