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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

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.

Explore All Areas of Our Impact

Impact Stories

Researchers Create 3-D Model for Rare Neuromuscular Disorders, Setting Stage for Clinical Trial

3-D bioengineered model that mimics the biology of two rare neuromuscular diseases. In both disorders, the brain’s messages to muscles are disrupted by the body’s immune system.

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

An NIH study in rats shows that star-shaped brain cells, called astrocytes (red) may play an active role in breathing.

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.

Thumb and pointer finger holding tissue chip

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.

Illustration of an adeno-associated virus carries a gene into a cell.

Platform Vector Gene Therapy (PaVe-GT)

Our scientists are using the same gene delivery system and manufacturing methods for multiple rare disease gene therapies.

Scientist in the lab

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

Leveraging Turncoat Immune Cells to Combat Cancer

June 3, 2022 - Grantee/Partner News

NCATS BioPlanet: A Resource for Discovery

March 23, 2022 - NCATS News

Researchers can better study how compounds and drugs affect cells with an NCATS resource that combines all pathways in human cells into one database.

New Dental Tool Prototype Can Spot the Acidic Conditions That Lead to Cavities

March 8, 2022 - Media Coverage