National Center for Advancing Translational Sciences (NCATS)


NCATS' mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

NCATS is improving health through smart science. The goal is to get more treatments to more patients more quickly. The Center develops, demonstrates and disseminates innovations that reduce, remove or bypass system-wide bottlenecks in the translational process. NCATS focuses on what is common across diseases and collaborates with other government agencies, including other NIH ICs; industry; academia; and patient support organizations.

Important Events in NCATS History

December 2011 — NCATS was established on Dec. 23 as part of the Consolidated Appropriations Act, 2012 (P.L. 112-74), which amended the Public Health Service Act.

March 2012 — NCATS teamed with Eli Lilly and Company to assess biological profiles of medicines and molecules that may enable biomedical researchers to better predict treatment outcomes, improve drug development, and lead to more specific and effective approaches.

May 2012 — NCATS and Eli Lilly and Company jointly released an online Assay Guidance Manual. The manual provides researchers with step-by-step guidance through the complex process of turning a basic research finding into an assay that will start the process of discovering pharmacological tools and drugs.

May 2012 — NCATS launched the Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program to advance therapeutic development by creating partnerships between pharmaceutical companies and the biomedical research community.

July 2012 — A research collaboration including scientists from NCATS and the University of Wisconsin–Madison helped identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease, a genetic neurological disease for which no treatments currently exist.

July 2012NIH awarded 17 grants for projects designed to create 3-D chips with living cells and tissues that accurately model the structure and function of human organs, such as the lung, liver and heart. These awards are funded and administered by NCATS. In September 2012, NIH awarded two additional tissue chip grants, administered by NCATS but funded by other NIH Institutes and Centers (ICs).

August 2012 — A team that includes nine NCATS researchers identified compounds that delay tumor formation in mice. The compounds target a specific form of pyruvate kinase, called PKM2, that governs how cancer cells use glucose.

August 2012 — NCATS announced the members of its inaugural Advisory Council and Cures Acceleration Network Review Board.

September 2012 — NIH Director Francis S. Collins, M.D., Ph.D., announced the appointment of Christopher P. Austin, M.D., as director of NCATS. View Image.

September 2012 — NIH researchers, including those from NCATS, launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM).

November 2012 — Researchers from NCATS designed a novel drug discovery method that uses two co-expressed reporter genes rather than one to increase the odds of identifying candidate compounds with true activity against biological or disease targets.

January 2013 — Research in NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program led to an NIH clinical trial for a possible treatment for Niemann-Pick disease type C1 (NPC1). View Image.

March 2013 — To investigate new ways to treat glaucoma, NCATS partnered with the Johns Hopkins School of Medicine in Baltimore. The team identified several compounds that appeared to stop the death of retinal ganglion cells, the neurons in the back of the eye that, when damaged in glaucoma, lead to vision loss and blindness.

June 2013 — As part of a pilot initiative, NCATS announced New Therapeutic Uses program awards to find new treatments for patients in eight disease areas.

June 2013 — The National Academies of Sciences, Engineering, and Medicine (formerly known as the Institute of Medicine) released The CTSA Program at NIH: Opportunities for Advancing Clinical and Translational Research.

November 2013 — Scientists at NIH used RNA interference (RNAi) technology to reveal dozens of genes that may represent new therapeutic targets for treating Parkinson’s disease. The findings also may be relevant to several diseases caused by damage to mitochondria. Researchers at the National Institute of Neurological Disorders and Stroke (NINDS) collaborated with NCATS researchers to discover a network of genes that may regulate the disposal of dysfunctional mitochondria, opening the door to new drug targets for Parkinson’s disease and other disorders. View Image.

December 2013 — Pamela M. McInnes, D.D.S., M.Sc.(Dent.) was named deputy director of NCATS, effective Jan. 12, 2014.

December 2013 — NCATS collaborated with Life Technologies Corporation to make gene-silencing data on the biochemical makeup of small interfering RNA molecules publicly available. View Image.

February 2014 — NCATS and National Cancer Institute (NCI) scientists published the first results from a test of a combination drug screening platform.

February 2014 — NCATS and the NIH Clinical Center hosted Rare Disease Day at NIH to spotlight the challenges encountered by those affected and the significant research and collaboration activities that are helping to make a difference in the development of new diagnostics and treatments. Since 2012, NCATS has played a major role in each annual event.

April 2014 — NCATS released its first annual report (2012-2013).

May 2014 — NCATS Advisory Council working group released findings on a 2013 report about the Clinical and Translational Science Awards (CTSA) Program released by the National Academies of Sciences, Engineering, and Medicine (formerly known as the Institute of Medicine).

May 2014 — CTSA Program institutions participating in institutional review board (IRB) reliance networks showed that efficient and centralized oversight can accelerate translational science.

June 2014 — NIH and NSF collaborated on an Innovation Corps (I-CorpsTM) pilot program to train business-minded biotech researchers. NCATS, the NCI, the National Heart, Lung and Blood Institute (NHLBI), and NINDS participated in the program.

July 2014 — Baxter International acquired Aes-103, a potential treatment for sickle cell disease advanced by NCATS’ TRND program researchers and collaborators. This was the first time a company has acquired a drug candidate developed with TRND resources. View Image.

July 2014 — NCATS launched a chemical toxicity data model competition. The Toxicology in the 21st Century (Tox21) Data Challenge 2014 was a crowdsourcing competition to develop computational models that can better predict chemical toxicity.

July 2014 — NIH announced a three-year pilot project called Illuminating the Druggable Genome (IDG). The awards primarily are funded through the NIH Common Fund. Other NIH ICs, including NCATS, are supporting this effort.

July 2014 — The Michael J. Fox Foundation funded a research project that showcases how NCATS' chemical screening resources can advance development of potential therapeutics for a broad range of diseases.

August 2014 — NCATS and National Human Genome Research Institute (NHGRI) scientists developed a potential treatment for patients with Gaucher disease, a rare, inherited condition marked by enlargement of the liver and spleen, anemia, nose bleeds, easy bruising and bleeding, bone problems, and occasionally neurological problems.

September 2014 — NIH funded the next phase of the NCATS-led Tissue Chip for Drug Screening program to integrate tissue chips and test drug effects.

October 2014 — NIH awarded $29 million to expand the Rare Diseases Clinical Research Network (RDCRN) to study more than 200 rare diseases.

November 2014 — NCATS experts, academic researchers, a patient advocacy group and a pharmaceutical company collaborated to identify a potential drug target for an inherited neurological disorder called Charcot-Marie-Tooth disease.

November 2014 — An NCATS repurposing test identified 53 drugs that may block Ebola infection.

January 2015 — NCATS and the Eunice Kennedy Shriver National Institute of Child Health and Human Development entered into an agreement with biotechnology company Vtesse, Inc., to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal storage disorders. View Image.

January 2015 — NCATS announced the winners of the Tox21 Data Challenge 2014, a crowdsourcing competition that attracted contestants from 18 countries to design computational models to better predict chemical toxicity.

March 2015 — Supported in part by NCATS, scientists found that a compound originally developed as a cancer therapy potentially could be used to treat Alzheimer’s disease.

April 2015 — NCATS released two funding opportunity announcements for Collaborative Innovation Awards, designed to stimulate team-based research across the CTSA Program consortium.

April 2015 — NCATS launched an initiative to wash and reuse plastic plates used during high-throughput robotic drug screening.

April 2015 — NCATS-supported researchers found that an over-the-counter drug indicated to treat allergy symptoms limited hepatitis C virus activity in infected mice.

May 2015 — An NCATS Chemical Genomics Center project team constructed a 3-D model of ovarian cancer metastasis using cells from patients.

June 2015 — NCATS released two CTSA Program funding opportunity announcements, one for Recruitment Innovation Centers and the other for Trial Innovation Centers. Both are aimed at overcoming key roadblocks to multisite clinical trials.

July 2015 — NCATS released its 2014 annual report, featuring the Center’s major milestones, programs and initiatives.

July 2015 — NCATS awarded nearly $3 million to support four academic research groups in testing a selection of pharmaceutical industry assets for new therapeutic uses.

July 2015 — NCATS-supported research enabled five men with complete motor paralysis to voluntarily generate step-like movements thanks to a new strategy that non-invasively delivers electrical stimulation to their spinal cords.

August 2015 — The White House announced that NIH is expanding its I-Corps™ training program to accelerate the commercialization of biomedical technologies developed with federal Small Business Innovation Research (SBIR) and Small Business Technology Transfer funding.

August 2015 — Supported by NCATS’ Tissue Chip for Drug Screening program, a team of scientists from Northwestern University, Charles Stark Draper Laboratory and the University of Illinois at Chicago designed a miniaturized 3-D representation of the female reproductive tract and liver — called EVATAR™ — on a handheld, interconnected platform for use in drug testing and to study the basic biology of female reproduction.

September 2015 — NCATS researchers and collaborators from the NIH’s National Institute of Allergy and Infectious Diseases (NIAID), Georgetown University, and the University of California, San Francisco, released a large dataset of potential drug combinations for malaria.

September 2015 — NCATS announced it would spearhead the second phase of several NIH ExRNA Communication program projects to test and validate exRNA molecules for their potential as disease biomarkers and treatments. The ExRNA Communications program is supported through the NIH Common Fund.

September 2015 — An NIH initiative expanded NCATS’ Tox21 program to investigate the effects of environmental chemicals on human development using robotic screening of cultured cells.

December 2015 — An NCATS-supported study revealed new genetic clues to age-related macular degeneration.

January 2016 — NCATS, the U.S. Environmental Protection Agency, and NIH's National Toxicology Program within the National Institute of Environmental Health Sciences announced a challenge that would award up to $1 million to improve the relevance and predictivity of data generated from automated chemical screening technology used for toxicity testing.

March 2016 — NCATS’ small business funding helped launch a new platform for rare diseases drug discovery.

March 2016 — RDCRN researchers partnered with The LAM Foundation, culminating in the first U.S. Food and Drug Administration (FDA)–approved treatment for lymphangioleiomyomatosis (LAM).

May 2016 — NCATS introduced plans for its new single IRB reliance platform for multisite clinical studies.

May 2016 — An NIH-funded team, supported in part by NCATS, found ketamine lifts depression via a byproduct of its metabolism.

May 2016 — NCATS scientists contributed to an NIH study that visualized proteins involved in cancer cell metabolism with an approach that may have an impact on drug discovery and development.

June 2016 — CTSA Program-supported scientists produced the clearest-ever image of telomerase, an enzyme involved in cancer and aging. This achievement could lead to the development of anti-cancer and anti-aging therapies.

June 2016 — NCATS-supported researchers developed a clinical-grade stem cell line with the potential to accelerate the advancement of new medical applications and cell-based therapies.

July 2016 — NCATS scientists collaborated with NHGRI and NINDS researchers to identify and test a molecule that shows promise as a possible treatment for the rare Gaucher disease and the more common Parkinson’s disease.

August 2016 — NCATS researchers identified compounds that potentially can be used to inhibit Zika virus replication and reduce its ability to kill brain cells.

September 2016 — NCATS released its 2015 annual report, featuring the Center’s major milestones, programs and initiatives.

September 2016 — CTSA Program-supported researchers developed a new, ultrasensitive assay for diagnosing diseases, including thyroid cancer, HIV and type 1 diabetes.

September 2016 — NCATS announced the Bench-to-Clinic Repurposing initiative and related funding opportunity to support investigators in repurposing existing experimental drugs or biologics, as well as FDA-approved therapies already on the market.

October 2016 — NCATS and the Center for the Advancement of Science in Space announced a collaboration to use tissue chip technology for translational research at the International Space Station U.S. National Laboratory.

October 2016 — NCATS partnered with the Stimulating Peripheral Activity to Relieve Conditions program to support research on the peripheral nervous system in hopes of finding new ways to treat conditions such as asthma, diabetes and nausea.

October 2016 — Scientific experts from 11 institutions began collaborating to assess the feasibility of developing the Biomedical Data Translator. Through this effort, NCATS integrated existing biomedical data to help reveal new relationships within those data and identify novel opportunities for research.

October 2016 — NCATS announced approximately $6 million in new awards for fiscal year 2016 to establish three testing centers for the Tissue Chip for Drug Screening program.

October 2016 — An NCATS-supported study found that a wearable patch that delivers small amounts of peanut protein through the skin showed promise for treating children and young adults with peanut allergy.

October 2016 — NCATS-supported Zika research in male mice revealed reproductive implications for human males.

November 2016 — NCATS released the Center’s strategic plan, organized into four overarching themes: translational science, collaboration and partnerships, education and training, and stewardship. The themes are captured in the strategic goals and collectively provide an overview of what the Center plans to accomplish to achieve its mission.

November 2016 — NCATS and NIAID researchers created a rapid screening test to identify drugs and drug combinations that may potentially be useful in combating infections that are resistant to many different antibiotics.

January 2017 — NCATS scientists, working with collaborators from the University of New Mexico and the University of Miami as part of the IDG Knowledge Management Center, developed the Target Central Resource Database (TCRD), which collates many heterogeneous gene/protein datasets, and Pharos, a multimodal web interface that presents the data from TCRD.

January 2017 — An unprecedented trans-NCATS collaboration enabled the rapid advancement of a rare lung disease therapy to human trials. Academic and industry researchers collaborated with NCATS TRND scientists on preclinical testing. CTSA Program support enabled researchers to begin an early study of the experimental drug in patients. View Image.

March 2017 — NCATS and NINDS-supported scientists discovered a urinary biomarker that may help track amyotrophic lateral sclerosis.

March 2017 — NCATS announced that all CTSA Program sites signed on to the NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) IRB authorization agreement. This agreement enabled all participating study sites to rely on the ethics review of one IRB for each study, making it possible to initiate multisite studies within weeks instead of months.

March 2017 — Patient-focused translational science collaboration among government and academic scientists, patients, and patients’ advocates advanced a potential treatment for the rare disease NPC1.

April 2017 — Teams led by NCATS and University of Tokyo scientists identified the first inhibitor of an enzyme long thought to be a potential drug target for fighting disease-causing parasites and bacteria. View Image.

April 2017 — Through NCATS’ Trial Innovation Network, CTSA Program-supported investigators worked collaboratively — including with the CTSA Program Trial Innovation Centers and the Recruitment Innovation Center, as well as with other NIH ICs — to tackle inefficiencies in clinical trials.

April 2017 — NCATS-supported researchers found that an experimental treatment cured 100 percent of guinea pigs and rhesus monkeys in late stages of infection with lethal levels of Marburg and Ravn viruses, relatives of the Ebola virus.

April 2017 — NCATS CTSA Program mentoring support sparked an interdisciplinary translational research team that developed a potential therapy for brain cancer.

May 2017 — Researchers from NCATS, NHLBI and the University of Tokyo identified the first inhibitor of an enzyme long thought to be a potential drug target for fighting disease-causing parasites and bacteria.

May 2017 — The NCATS SMART IRB team launched an Online Reliance System to help streamline the ethics review conducted by IRBs. View Image.

June 2017 — NCATS and the University of Nevada, Reno School of Medicine researchers demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy. View Image.

June 2017 — NCATS and the Center for the Advancement of Science in Space announced five grants to support research on human physiology and disease onboard the International Space Station U.S. National Laboratory. View Image.

June 2017 — NCATS hosted an inaugural event NCATS Day — to help identify patients’ needs and brainstorm ways to foster engagement at every step of the translational science spectrum. The Center hosted the event in 2018 and 2019.

June 2017 — NCATS partnered with Eli Lilly and Company on the NCATS-Eli Lilly scholars’ externship program. The ongoing effort pairs scholars, trainees and investigators from NCATS’ CTSA Program with an Eli Lilly project team for up to one year.

June 2017 — An NCATS-supported multidisciplinary team addressed translational research hurdles — including ethics review and informed consent processes — to add genetic tests to newborn screenings and improve early diagnosis of fragile X syndrome and other rare genetic diseases.

July 2017 — NCATS-supported scientists demonstrated how an investigational drug works against a rare, fatal genetic disease, NPC1.

July 2017 — Through its Small Business Innovation Research program, NCATS supported the development of a technology to use electronic health records data to advance value-based and enhanced patient care.

August 2017 — Supported in part by NCATS, a team of scientists examined the maturation of astrocytes in 3-D structures grown in culture dishes to resemble human brain tissue. This research addresses a significant gap in human brain research by providing an invaluable technique to investigate the role of astrocytes in both normal development and disease.

September 2017 — A collaboration between researchers from NCATS’ TRND program and Agilis Biotherapeutics, Inc., of Cambridge, Massachusetts, resulted in a gene therapy for the rare pediatric condition aromatic L-amino acid decarboxylase deficiency moving closer to market years ahead of schedule. View Image.

September 2017 — Through its Tissue Chip for Drug Screening program, NCATS awarded $15 million to support development of 3-D microphysiological system platforms that model human disease. View Image.

September 2017 — Supported in part by NCATS, researchers found that children exposed to high indoor levels of pet or pest allergens during infancy have a lower risk of developing asthma by 7 years of age. The findings may provide clues for the design of strategies to prevent asthma from developing.

September 2017 — Supported in part by NCATS, research conducted in mice provided evidence that highly lethal brain tumors, called high-grade gliomas, stop growing when deprived of a specific molecule naturally produced when brain cells fire.

November 2017 — Supported in part by NCATS, scientists identified a potential drug that could lead to new approaches for preserving brain cells after an ischemic stroke.

November 2017 — NCATS CTSA Program-supported investigators collaborated to validate biomarkers intended to monitor the response to therapy for NPC1.

December 2017 — A team of NIH-supported researchers explored the use of a highly sensitive technique to counteract hearing loss caused by cisplatin, a powerful drug used to treat cancer. The team, which included NCATS scientists, measured and mapped cisplatin build-up in the inner ear and found a region that could be targeted to prevent hearing loss.

December 2017 — NCATS-supported researchers demonstrated the value in a community-based research evaluation model of consultation services that is helping to translate clinical practice observations into research-supported treatment approaches.

January 2018 NIH launched the Somatic Cell Genome Editing (SCGE) program aimed at removing barriers to slow the adoption of genome editing for treating patients. This program is supported by the NIH Common Fund and is managed by a trans-agency working group, led by NCATS.

January 2018 — NCATS’ TRND program collaborated with scientists at Duke University’s Clinical and Translational Science Institute to advance a gene therapy for a rare neuromuscular disorder toward clinical testing. View Image.

January 2018 NCATS scientists and collaborators demonstrated that blocking the activity of a certain enzyme reduced the effects of Huntington’s disease in cell and animal models. View Image.

February 2018 Scientists from NCATS and Sweden’s Karolinska Institutet developed a potential new approach to fighting cancer by breaking down a defense system used by cancer cells. The defense system involves an enzyme, thioredoxin reductase 1 (TrxR1), which supports cancer cell survival. View Image.

February 2018 — NIH’s National Institute of Dental and Craniofacial Research collaborated with NCATS through the CTSA Program to provide translational research training opportunities to oral, dental and craniofacial scientists early in their careers. View Image.

March 2018 Through its CTSA Program, NCATS established the National Center for Data to Health, through which informatics experts are addressing operational and institutional barriers to sharing data.

March 2018 — NCATS supported an innovative platform technology to precisely deliver nutrients and hormones to cells for a preclinical therapeutics testing program.

March 2018 Tox21 program partners from NCATS and other collaborators published a new strategic and operational plan to broaden the scope of their research activities to address new challenges. View Image.

April 2018 NCATS-supported researchers made significant strides in reliably engineering human heart muscle that mimics the intricacies of a human heart, which could serve as a better model for testing the effects of toxic substances than current tissue-engineered heart models. View Image.

April 2018 NCATS-funded researchers used stem cells originally derived from a person’s skin to recreate interactions between blood vessels and neurons that may occur early in the formation of the fetal human spinal cord, which may potentially be used to test personalized treatments of neurological disorders. View Image.

April 2018 Through its New Therapeutic Uses program, NCATS awarded funding for three new projects to repurpose compounds that have already undergone significant research and development by industry, including testing in humans.

May 2018 Scientists at NIH, Northwestern University and their collaborative research partners identified a compound that blocks the spread of pancreatic and other cancers in various animal models. This research was in-part funded by NCATS.

May 2018 — NCATS Biomedical Data Translator teams implemented a unique and collaborative approach to advance biomedical data sharing. View Image.

May 2018 NIH's RDCRN-supported research helped overcome a translational roadblock by charting how several rare, inherited disorders progress in patients over time, which enabled a potential rare disease gene editing treatment into clinical trials. View Image.

July 2018 NCATS-supported researchers developed an innovative device that detects internal bleeding and monitors a patient’s response to blood loss. View Image.

August 2018 — Researchers at Cedars-Sinai Medical Center and the University of California, Los Angeles, part of NCATS’ CTSA Program, paired pharmacists with barbershops to help prevent heart attacks and strokes. View Image.

August 2018 NCATS scientists created a simple, fast and automated process to make 3-D tissues transparent, which enables many types of observations within 3-D growth environments. View Image.

August 2018 NCATS created a new online tool — NCATS Inxight: Drugs — that aggregates reliable, curated drug development data from multiple existing sources to help researchers find the information they need more easily.

August 2018 NCATS and the FDA co-hosted a two-day workshop about gene therapy for rare diseases.

September 2018 NCATS launched the Rare Diseases Are Not Rare! Challenge seeking creative ways to help raise awareness about rare diseases. Winners and honorable mentions were announced in December 2018. In February 2020, NCATS ran the Challenge again and announced winners and honorable mentions in August 2020. View the gallery of 2020 winning entries.

October 2018 — NCATS’ CTSA Program support enabled the early development and “de-risking” of an innovative device that may help patients avoid complications of heart valve replacement. View Image.

October 2018 NIH awarded $86 million to support research aimed at improving methods to edit the human genome. These grants were the first to be awarded through the SCGE program, which is managed by staff from multiple NIH ICs, with leadership from NCATS. View Image.

November 2018 — Joni L. Rutter, Ph.D., joined NCATS as the Center’s new deputy director. View Image.

December 2018 — NCATS announced it would support three new funding opportunities and a prize challenge through the NIH Helping to End Addiction Long-termSM Initiative, or the NIH HEALSM Initiative. View Image.

December 2018 NCATS partnered with the ISSNL to send tissue chips into space for research initiatives leveraging the unique microgravity environment. View Image.

December 2018 — CTSA Program-supported investigators devised and piloted an innovative approach called Real-Time IRB that reduces the time between study submission to the institutional review board and final approval by 70 percent.

December 2018 Researchers supported by NCATS’ Tissue Chip for Drug Screening program developed a 3-D working model of a human heart chamber. View Image.

December 2018 NCATS researchers overcame a translational roadblock by developing tests to identify compounds that disrupt a driver of several cancers. Their work, which involved using NCATS’ high-throughput screening technology to evaluate thousands of compounds at once, provided a template for other scientists to discover new compounds that could be useful as therapies. View Image.

January 2019 NCATS examined the biological activities of natural products to evaluate the compounds’ possible uses in treating a variety of diseases. The findings make up the Canvass library of natural products.

February 2019 — NIH launched the Platform Vector Gene Therapy (PaVe-GT) initiative, led by NCATS, to test whether it is feasible to use the same gene delivery system and manufacturing methods for multiple rare diseases in gene therapy clinical trials.

February 2019 NCATS, the National Institute on Drug Abuse and NINDS co-sponsored The Opioid Crisis and the Future of Addiction and Pain Therapeutics: Opportunities, Tools, and Technologies Symposium.

April 2019  Scientists from the Extracellular RNA Communication Consortium published a landmark collection of papers on the biology and possible clinical applications of exRNA. The ExRNA Communication program is funded by the NIH Common Fund and led by staff from the Common Fund, NCATS and other NIH ICs.

May 2019 The fifth edition of the International Congress on Research of Rare and Orphan Diseases was co-organized in collaboration with NCATS to bring together scientists to share research and exchange ideas.

May 2019 NCATS-supported researchers sent four tissue chip projects into space to speed the study of aging-related conditions and the development of treatments for them. View Image.

May 2019 NCATS and other members of an international group called Translational Together identified qualities that distinguish translational scientists. View Image.

June 2019 NCATS sponsored a Workshop on CNS Immunogenicity Considerations for adeno-associated virus (AAV)-mediated Gene Therapy that brought together the scientific and medical expert community for AAV-mediated therapies to focus on issues related to the central nervous system administration and immunogenicity of these products.

July 2019 — NCATS, the NCI and the National Institute of Biomedical Imaging and Bioengineering (NIBIB) co-sponsored a workshop to provide experts and the community the opportunity to share their perspectives on current issues associated with the incorporation of Artificial/Machine Intelligence systems into health care settings.

August 2019 NCATS announced winners of the 2018 ASPIRE Design Challenges, which were launched as part of the NIH HEAL Initiative to encourage innovative and catalytic approaches toward solving the opioid crisis.

August 2019 NCATS scientists and their colleagues created a one-of-a-kind database that details the body’s biological pathways, which can enable researchers to more readily study gene activity, gather insights on disease mechanisms, shed light on when and how compounds are toxic to cells and more.

September 2019 Scientists from NIH, including NCATS and Cincinnati Children’s Hospital Medical Center devised a potential treatment against a common type of leukemia that could have implications for many other types of cancer. View Image.

September 2019 NIH awarded approximately $89 million to advance additional projects for genome projects through the SCGE Program. The program is supported by the NIH Common Fund and led by NCATS. View Image.

September 2019 — NCATS collaborated with The Children's Inn at NIH, the Amateur Radio on the International Space Station and the International Space Station U.S. National Laboratory to host Ask an Astronaut: Biomedical Science Edition, which provided children receiving care at NIH the opportunity to talk to an astronaut in space about science.

September 2019 NCATS’ Director Christopher Austin, M.D.  joined NIH Director Francis S. Collins, M.D., Ph.D., and several other Institute and Center directors on Capitol Hill to discuss investments in medical research at NIH.

October 2019 - NIH awarded approximately $31 million in grants to increase the percentage of rare diseases that have FDA approved treatments. The grants support consortia that form the RDCRN, which is led by NCATS and  NCATS Office of Rare Diseases Research. View Image.

November 2019 NCATS and several other NIH ICs co-sponsored the NIH Inaugural Rural Health Seminar, which explored how innovations in clinical and translational science could improve rural health outcomes.

November 2019 NCATS researchers and colleagues identified a drug pair that worked together to both kill cancer cells and counter the effects of a genetic mutation that causes the group of deadly childhood brain cancers. View Image.

December 2019 NCATS hosted Repurposing Off-Patent Drugs: Research & Regulatory Challenges to discuss challenges around finding new uses for drugs that are already on the market but lack commercial and regulatory incentives for research and development.

February 2020 — Researchers from NCATS, NCI and Tuskegee University showed in laboratory experiments that a novel type of immune therapy can reprogram immune cells to kill cancer cells and halt tumor growth in several types of cancer. View Image.

March 2020 — NIH-supported researchers sent heart and gut tissue chip projects into space to speed the study of aging-related conditions and the development of treatments for them. View Image.

April 2020 — NCATS collaborated with NIAID, NIBIB and NCI to conduct a study to determine how many adults in the United States without a confirmed history of SARS-CoV-2 infection have antibodies to the virus. View Image.

May 2020 — NCATS scientists developed the COVID-19 OpenData Portal to share data from a large panel of experiments to test approved drugs against targets in or related to SARS-CoV-2.

May 2020 — The RDCRN, led by NCATS in collaboration with nine other NIH ICs, launched a research survey to find out how COVID-19 is affecting individuals with rare diseases, their families and their caregivers. View Image.

June 2020 — Created through a collaboration between FDA and NCATS, CURE ID enabled the crowdsourcing of medical information from health care professionals to facilitate the development of new treatments using repurposed drugs for difficult-to-treat infectious diseases.

June 2020 — NIH launched a centralized, secure enclave as part of the National COVID Cohort Collaborative (N3C) effort to enable researchers to study vast amounts of medical record data from people diagnosed with COVID-19. View Image.

June 2020 — NCATS launched its first online translational science course, which focused on teaching key principles of translational science and how these principles have been operationalized in a real-world project. 

July 2020 — NCATS introduced the I Am Translational Science video series, which shares stories by researchers and others about what they do, what inspires their work and why they find translational science exciting. The initial stories featured people involved in COVID-19 efforts.

September 2020 — NCATS’ CTSA Program supported NIH in several efforts to speed the discovery and delivery of COVID-19 treatments and vaccines to those in greatest need. View Image.

September 2020 — Through its CTSA Program, NCATS expanded enrollment in two randomized, placebo-controlled clinical trials funded by NIH to further evaluate convalescent plasma as a treatment for people hospitalized with COVID-19. View Image.

September 2020 — NIH awarded 10 grants to support researchers’ efforts in using tissue chips — tiny, bioengineered models of human tissues and organ systems — to study diseases and test drugs. The awards are administered through the new Clinical Trials on a Chip program, led by NCATS in conjunction with other NIH ICs. View Image.

October 2020 — As part of the Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) partnership, NCATS coordinated and oversaw an adaptive Phase 3 clinical trial to evaluate the safety and efficacy of three immune modulator drugs in adults hospitalized with COVID-19. View Image.

October 2020 — NCATS and NIAID co-sponsored the NIH Virtual SARS-CoV-2 Antiviral Therapeutics Summit, which brought together experts from academia, industry and government to discuss the state of antiviral therapeutics development for COVID-19.

November 2020 — NCATS co-hosted the Virtual Workshop on Systemic Immunogenicity Considerations for AAV-Mediated Gene Therapy, which brought together scientists, therapeutic developers and patient representatives to focus on immunogenicity issues related to the systemic administration of AAV gene therapy products and identify solutions for those issues.

December 2020 — Three NCATS-supported tissue chip research projects were sent to the International Space Station to explore how to prevent osteoarthritis after joint injuries, what drives muscle wasting and whether heart tissue damage can be reversed.

NCATS Legislative Chronology

December 23, 2011 President Obama signed into law the Consolidated Appropriations Act, 2012 (P.L. 112-74), enabling NIH to establish NCATS. This law also transferred authority over the Cures Acceleration Network (CAN) to NCATS. Authorized to reduce significant barriers to successful translation and accelerate the development of high-need cures, the CAN provides NCATS with flexibility in how it funds projects. Implementation of this authority is guided by the CAN Review Board.

December 13, 2016 — The 21st Century Cures Act changed NCATS annual report requirement to a biennial report and modified NCATS’ restriction on supporting clinical trials to allow support for all phase II clinical trials and support for phase III clinical trials on rare diseases.

March 27, 2020 — President Trump signed the Coronavirus Aid, Relief and Economic Security (CARES) Act, providing funding to NCATS and several other ICs to advance coronavirus research.

Biographical Sketch of NCATS Acting Director Joni L. Rutter, Ph.D.

Joni L. Rutter, Ph.D.Joni L. Rutter, Ph.D.

Joni L. Rutter, Ph.D., is the acting director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). Rutter oversees the planning and execution of the Center’s complex, multifaceted programs that aim to overcome scientific and operational barriers impeding the development and delivery of new treatments and other health solutions. Under her direction, NCATS supports innovative tools and strategies to make each step in the translational process more effective and efficient, thus speeding research across a range of diseases, with a particular focus on rare diseases. By advancing the science of translation, NCATS helps turn promising research discoveries into real-world applications that improve people’s health. In her previous role as the NCATS deputy director, Rutter collaborated with colleagues from government, academia, industry and nonprofit patient organizations to establish robust interactions with NCATS programs.

Prior to joining NCATS, Rutter served as the director of scientific programs within the All of Us Research Program, where she led the scientific programmatic development and implementation efforts to build a national research cohort of at least 1 million U.S. participants to advance precision medicine. During her time at NIH, she also has led the Division of Neuroscience and Behavior at the National Institute on Drug Abuse (NIDA). In this role, she developed and coordinated research on basic and clinical neuroscience, brain and behavioral development, genetics, epigenetics, computational neuroscience, bioinformatics, and drug discovery. Rutter also coordinated the NIDA Genetics Consortium and biospecimen repository.

Throughout her career, Rutter has earned a national and international reputation for her diverse and unique expertise via more than 50 publications in journals. She has received several scientific achievement awards, including a SmithKline Beecham Student Award in Pharmacology, a Janssen Research Foundation Young Investigator Award and a Fellowship Achievement Award from the National Cancer Institute (NCI).

Rutter received her Ph.D. from the Department of Pharmacology and Toxicology, Dartmouth Medical School, Hanover, New Hampshire, and completed a fellowship at NCI within the Division of Cancer Epidemiology and Genetics.

NCATS Directors

Name In Office from To
Thomas R. Insel (Acting) December 23, 2011 September 22, 2012
Christopher P. Austin September 23, 2012

April 15, 2021

Joni L. Rutter (Acting) April 16, 2021


Major Programs

NCATS’ programs and initiatives span the entire spectrum of translational science.
They are as follows:

  • 3-D Tissue Bioprinting Program. In this program, NCATS experts are applying the techniques of 3-D bioprinting to develop tissue models that mimic the organization and physiology of cells in the tissues of living organisms, in a microplate format for drug screening.
  • A Specialized Platform for Innovative Research Exploration (ASPIRE). By addressing long-standing challenges in the field of chemistry, including lack of standardization, low reproducibility and an inability to predict how new chemicals will behave, ASPIRE is designed to bring novel, safe and effective treatments to more patients more quickly at lower cost.
  • Assay Development and Screening Technology (ADST) program. ADST is designed to advance therapeutic development through research and development of innovative assay (test) designs and chemical library screening methods. Program experts work to optimize assays requested or submitted by the biomedical research community for high-throughput small-molecule screening.
  • Assay Guidance Manual. The Assay Guidance Manual initiative provides best-practices resources and training devoted to the successful development of robust, early-stage drug discovery assays. Investigators worldwide can use the manual to design biologically and pharmacologically relevant assays for high-throughput screening and lead optimization to evaluate collections of molecules that modulate the activity of biological targets, pathways and cellular phenotypes.
  • Biomedical Data Translator. NCATS launched the Biomedical Data Translator (Translator) program to accelerate biomedical translation for the research community. Through this program, NCATS will integrate multiple types of existing data sources and reveal potential relationships across the spectrum of data types. This multiyear, iterative effort will culminate in the development of a comprehensive, relational, N-dimensional Biomedical Data Translator that integrates multiple types of existing data sources, including objective signs and symptoms of disease, drug effects, and intervening types of biological data relevant to understanding pathophysiology.
  • Bridging Interventional Development Gaps (BrIDGs) program. BrIDGs supports research collaborations to advance candidate therapeutics for both common and rare diseases into clinical testing. Investigators selected through an application process partner with NCATS experts to generate pre-clinical data and clinical-grade material through government contracts for use in Investigational New Drug applications to a regulatory authority such as the FDA.
  • Chemistry Technology program. Chemistry technology experts at NCATS develop small molecules and screening approaches that other scientists can use to pursue innovations in therapeutic development. The aim is to provide cutting-edge resources to enable the broader biomedical research community to pursue basic and translational studies in a faster and more in-depth manner.
  • Clinical and Translational Science Awards (CTSA) Program. The CTSA Program supports an innovative national network of medical research institutions that work together to improve the translational research process to get more treatments to more patients more quickly. CTSA Program research centers serve as hubs locally and regionally to catalyze innovation in training, research tools and processes.
  • Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes (CTR) Grants Program. The CTR grants program supports projects focused on collecting the data needed to move promising rare disease therapies and diagnostics to clinical trial.
  • Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. Through this innovative program, NCATS aims to improve the process of developing new treatments and cures for disease by finding new uses for assets that already have cleared several key steps along the development path (also known as drug repurposing). NCATS designed its New Therapeutic Uses program to align with one of its primary goals: to foster approaches that improve the translational research pipeline and ultimately accelerate the pace at which discoveries are turned into new preventions, treatments and cures for human diseases. In October 2017, NCATS released a funding opportunity to explore a potential new use of an existing experimental drug or biologic through its Bench-to-Clinic Repurposing initiative, which supports preclinical studies to test the utility of an independent crowdsourcing effort, computational algorithm or big dataset from patient records to predict new uses of a drug or biologic.
  • Early Translation Branch (ETB). ETB researchers advance small molecule therapeutic development through assay (test) design, high-throughput screening and medicinal chemistry. Small molecule chemical compounds, which can be used to test or “probe” the effects of increasing or decreasing the activity of a biological target in cells or animals, are some of the most powerful tools for target validation, which is the process of demonstrating that engaging a target provides meaningful therapeutic benefit. Probes enable researchers to investigate protein, cell functions and biological processes. If appropriate, probes can be optimized to become potential drug candidates.
  • Extracellular RNA (exRNA) Communication program. Through this NIH Common Fund program, scientists are beginning to understand the potential exRNA research may hold for improving understanding, diagnosis, prognosis and treatment of a wide variety of diseases and conditions, such as cancer, bone marrow disorders, heart disease, Alzheimer’s disease and multiple sclerosis. ExRNA communication is a recently discovered cell-to-cell signaling process that holds enormous promise for improving our understanding of a wide variety of diseases.
  • Functional Genomics Lab. NCATS’ Functional Genomics Lab is designed to develop and improve RNAi screening approaches to better understand gene function and identify treatment targets. Gene silencing through RNAi has emerged as a powerful tool for understanding gene function. Over the past several years, high-throughput RNAi screens have illuminated a wide variety of biological processes, ranging from genes that affect the activity of therapeutic agents to novel components of signaling pathways.
  • Genetic and Rare Disease Information Center (GARD). This NCATS collaboration with the National Human Genome Research Institute offers comprehensive information on rare and genetic diseases to patients, their families, health care providers, researchers and the public. The online GARD database provides accurate, up-to-date information about ongoing research, symptoms, treatment options and other details.
  • The Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM. NCATS is one of several NIH Institutes and Centers participating in the NIH HEAL Initiative. With Initiative support, NCATS will accelerate the process of developing new treatments for opioid misuse and addiction and for pain.
  • Illuminating the Druggable Genome (IDG). IDG originally was funded as three-year pilot program sponsored by the NIH Common Fund and designed to test a two-pronged approach for exploring the druggable genome. Support was first awarded in 2014. The current implementation phase of the IDG Program aims to build on the knowledge and tools developed during the pilot phase and disseminate these IDG-generated resources to the greater scientific community.
  • Informatics Research and Scientific Applications. Informatics at NCATS plays a key role in organizing, processing, analyzing and interpreting the large quantity and variety of data generated by translational research.
  • Matrix Combination Screening. NCATS experts use a technology called matrix combination screening to quickly narrow down a long list of potential drug combinations and find those with the most potential to help patients. The matrix combination screening platform can rapidly test the effect of different drug combinations on cellular, molecular or biochemical processes that are relevant to a disease of interest.
  • National COVID Cohort Collaborative (N3C). The N3C is a partnership among the NCATS-supported Clinical and Translational Science Awards (CTSA) Program hubs and the National Center for Data to Health (CD2H), with overall stewardship by NCATS. It aims to create a national resource of COVID-19 clinical data that the research community can use to answer critical research questions as the pandemic continues to evolve.
  • NCATS Toolkit for Patient-Focused Therapy Development. Launched in September 2017, the NCATS Toolkit for Patient-Focused Therapy Development was created to provide a collection of online resources that can help patient groups advance through the process of therapy development and provide them with the tools they need to advance medical research.
  • NIH Common Fund Programs. NIH Common Fund programs can provide a strategic and nimble approach to address key roadblocks in biomedical research that impede basic scientific discovery and its translation into improved human health. All NIH Institutes and Centers are involved with the NIH Office of Strategic Coordination in the design, implementation and evaluation of Common Fund programs.
  • OpenData Portal. The OpenData Portal is a resource created by NCATS to openly and quickly share COVID-19-related drug repurposing data and experiments for all approved drugs.
  • Platform Vector Gene Therapy (PaVe-GT). This NCATS-led pilot project will test whether it is possible to increase the efficiency of gene therapy clinical trial startup by using the same gene delivery system and manufacturing methods for multiple gene therapies.
  • Rare Diseases Clinical Research Network (RDCRN). NCATS’ RDCRN program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.
  • Rare Diseases Registry Program (RaDaR). RaDaR, formerly known as the Global Rare Diseases Registry Data Repository (GRDR) program, is designed to define best practices for patient registries. Through RaDaR, NCATS also strives to identify and adopt standards to support high-quality registries for rare diseases therapeutics development.
  • Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR). These programs support NCATS’ mission to transform the translational science process by helping small businesses develop and commercialize new technologies so that new treatments and cures for disease can be delivered to patients more efficiently. They serve as an engine of innovation, offering grants, contracts and technical assistance to small businesses and research organizations focused on advancing translational research and technologies that will improve disease prevention, detection and treatment.
  • Somatic Cell Genome Editing. The Somatic Cell Genome Editing program, led by NCATS, funded by the NIH Common Fund, and managed by a trans-NIH working group representing multiple institutes and centers, aims to develop quality tools to perform safe and effective genome editing in humans and then make these tools widely available to the research community to reduce the time and cost of developing new therapies.
  • Stem Cell Translation Laboratory (SCTL). NCATS is working to advance translational methods in stem cell research with support from the NIH Common Fund. Induced pluripotent stem cells are particularly useful because scientists can transform them into many different cell types to use for research or therapies. Through the SCTL, NCATS will provide researchers across various disciplines and organizations with the ability to establish collaborations to advance the translation of regenerative medicine applications.
  • Stimulating Peripheral Activity to Relieve Conditions (SPARC). SPARC is an NIH Common Fund program that focuses on understanding peripheral nerves — nerves that connect the brain and spinal cord to the rest of the body — and how their electrical signals control internal organ function. Modulation of these control signals is a potentially powerful way to treat common conditions and diseases such as rheumatoid arthritis and heart failure.
  • Therapeutics for Rare and Neglected Diseases (TRND) program. Through the TRND program, NCATS supports the preclinical development of therapeutic candidates intended to treat rare or neglected disorders, with the goal of enabling an Investigational New Drug application. The mission of the program is to encourage and speed the development of new treatments for diseases with high unmet medical needs. TRND stimulates therapeutic development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses.
  • Tissue Chip for Drug Screening (Tissue Chip) program. This NCATS collaboration with the Defense Advanced Research Projects Agency and FDA supports the development of bioengineered devices to improve the process of predicting whether drugs will be safe or toxic in humans. The focus is on 3-D platforms engineered to support living human tissues and cells, called tissue chips or organs-on-chips. Tissue chip devices are designed as accurate models of the structure and function of human organs, such as the lung, liver and heart. Once developed and integrated, researchers can use these models to predict whether a candidate drug, vaccine or biologic agent is safe or toxic in humans in a faster and more cost-effective way than current methods. The ultimate goal of the program is to accelerate the translation of basic discoveries into the clinic. By creating an integrated human body-on-a-chip, researchers can test the varied potential effects of a substance like a drug across the entire body before any testing in humans.
  • Toxicology in the 21st Century (Tox21) program. Tox21 is a federal collaborative effort among NIH — including NCATS and the National Toxicology Program at the National Institute of Environmental Health Sciences — the Environmental Protection Agency and the FDA. Tox21 researchers aim to develop better toxicity assessment methods to quickly and efficiently test whether certain chemical compounds have the potential to disrupt processes in the human body that may lead to negative health effects. Using the Center’s high-throughput robotic screening system, NCATS scientists are testing a collection of 10,000 approved drugs and environmental chemicals for their potential to disrupt biological pathways that may result in toxicity. The compounds undergo testing in NCATS’ high-speed robotic screening system.
  • Translational Approach to COVID-19. NCATS is supporting research activities spanning the translational science spectrum to address the novel coronavirus 2019 (SARS-CoV-2) and the disease it causes (COVID-19).

This page last reviewed on April 23, 2021