Our Impact on Tools and Technologies
We create solutions that work for many diseases, speeding more treatments for all people more quickly.
Making Research Faster for Many Diseases at a Time
The University of Pittsburgh Drug Discovery Institute Microphysiology Systems Database is designed in part to capture experimental information on organ models and the data generated from them. (University of Pittsburgh Drug Discovery Institute Photo/Richard DeBiasio)
There are more than 10,000 known diseases, but only 5% of them have a treatment or cure. If we focus on developing new drugs for one disease at a time, it will take thousands of years to find therapeutic answers for every disease.
To speed new treatments, NCATS has created therapeutic tools and technologies that work for many diseases. Our experts look for what’s similar among groups of diseases to find shared solutions for all. For example, different rare diseases can share a common genetic or biochemical pathway that goes awry. We are using that knowledge to streamline the development of approaches with the potential of treating many diseases. These include cell-based treatments, gene-targeted treatments and new uses for existing treatments.
We also overcome scientific barriers common to all diseases, such as research models that poorly predict how a promising medicine will work in people. Our approaches for making more predictive drug screening models have spurred the evolution of the field, led to new models for dozens of diseases, and has started to open up bottlenecks in the drug development pipeline.
Impact Stories
Researchers Create 3-D Model for Rare Neuromuscular Disorders, Setting Stage for Clinical Trial
NCATS-supported researchers created a 3-D model that mimics the biology of two rare neuromuscular diseases.
Efficient Approach Creates Astrocytes Faster From Stem Cells for Drug Development and Regenerative Medicine
NCATS scientists used stem cells and a novel shortcut to create large supplies of astrocytes to speed research on therapies for neurodegenerative diseases.
Tools and Technologies Activities
Through collaboration, we develop research solutions that work across projects and diseases.
Tissue Chip for Drug Screening
This program funds the development and testing of models built from human cells for predicting drug safety and toxicity before clinical testing in people.
Platform Vector Gene Therapy (PaVe-GT)
Our scientists are using the same gene delivery system and manufacturing methods for multiple rare disease gene therapies.
Stem Cell Translational Laboratory (SCTL)
Our experts develop methods and standards to use stem cell technology (using cells derived from skin or blood) to advance treatment approaches.
Tools and Technologies News
Electrifying Invention Can Save Young Lives and Ease Chronic Lung Conditions
February 5, 2024 - NCATS News
- Our Impact on Tools and Technologies
- Small Business (SBIR)
NCATS Scientists Turn Stem Cells Into Vast Supply of Neurons to Better Predict Pain Therapies’ Effectiveness
June 14, 2023 - NCATS News
- Our Impact on Tools and Technologies
NCATS researchers and collaborators test possible new pain-relieving drugs in the laboratory on nerve cells.
Leveraging Turncoat Immune Cells to Combat Cancer
June 3, 2022 - Grantee/Partner News
- Our Impact on Tools and Technologies
NCATS BioPlanet: A Resource for Discovery
March 23, 2022 - NCATS News
- Our Impact on Big Data
- Our Impact on Tools and Technologies
Researchers can better study how compounds and drugs affect cells with an NCATS resource that combines all pathways in human cells into one database.