Scientific Glossary
Terminology definitions for human-relevant medicine technologies
Biomarker
A measurable indicator of biological state or condition. Used to monitor disease progression, drug response, and treatment efficacy.
CAR-T (Chimeric Antigen Receptor T-cell)
Genetically modified T cells engineered to recognize and attack cancer cells. A revolutionary immunotherapy approach for certain blood cancers.
CRISPR
A gene-editing technology that allows precise modifications to DNA. Used for creating disease models, developing therapies, and understanding gene function.
Digital Twin
A computational model that creates a virtual representation of a physical system—in healthcare, a patient's physiology—to predict responses to treatments and interventions.
DILI (Drug-Induced Liver Injury)
Liver damage caused by medications. A leading cause of drug failure and withdrawal. Human liver models can predict DILI with higher accuracy than animal tests.
FDA Modernization Act 2.0/3.0
U.S. legislation (2022-2023) that eliminates the requirement for animal testing before human clinical trials, enabling use of NAMs and other human-relevant methods.
In Silico
Experiments performed via computer simulation or modeling. Includes computational drug discovery, molecular dynamics, and AI-driven predictions.
In Vitro
Experiments performed outside a living organism, typically in test tubes, petri dishes, or cell culture systems. Contrasts with in vivo (in living organisms).
iPSC (Induced Pluripotent Stem Cell)
Adult cells reprogrammed to an embryonic-like state, capable of differentiating into any cell type. Enables patient-specific disease modeling and drug testing.
Microfluidics
Technology for manipulating small volumes of fluids in microscale channels. The foundation of organ-on-chip devices and lab-on-chip systems.
Microphysiological System (MPS)
Engineered platforms that recreate human organ function at microscale. Includes organ-on-chip devices and tissue chip systems.
NAMs (New Approach Methodologies)
Non-animal technologies for drug safety assessment including in vitro, in silico, and organ-on-chip methods recognized by FDA Modernization Act 3.0.
Organ-on-Chip
A microfluidic device lined with human cells that recreates the physiological environment of human organs, enabling drug testing in human-relevant conditions.
Organoid
Self-organizing 3D cellular structures derived from stem cells that replicate the architecture and function of human organs. Used for disease modeling and drug testing.
PBPK (Physiologically Based Pharmacokinetic)
Mathematical models that predict drug behavior in the body based on physiological parameters. Used for dose optimization and drug-drug interaction prediction.
PK/PD (Pharmacokinetics/Pharmacodynamics)
PK describes how the body processes a drug (absorption, distribution, metabolism, excretion). PD describes how the drug affects the body.
Spheroid
A 3D cell culture that forms a sphere-like structure. Simpler than organoids but provides more realistic drug response than 2D cultures.
Tissue Chip
An organ-on-chip device specifically designed to model tissue-level function. Term used by NCATS Tissue Chip Program.
Toxicology
The study of harmful effects of substances on living organisms. In drug development, assessing safety and identifying potential adverse effects.