From the Director | What's New at NCATS? | Research Opportunities Volume 03 • Issue 04 • July 24, 2014

Director's Message

Christopher Austin

I often cite the essential components of translational science — collaboration, public-private partnerships, innovative research models — as common denominators of all NCATS initiatives. One of the most exciting parts of our work is when research teams demonstrate that these elements can produce breakthroughs in translation. Our latest accomplishment is truly a historic one, producing two "firsts": For the first time, efforts of researchers from NCATS' Therapeutics for Rare and Neglected Diseases program have culminated in the acquisition of a drug candidate by a biopharmaceutical company, Baxter International, to complete clinical development. And also for the first time, a drug targeting the underlying cause of sickle cell disease — the first genetic condition ever defined at the molecular level — has advanced into late-stage clinical development.

Read more of the latest Director's Message.

Christopher P. Austin, M.D.

What's New at NCATS?

Industry Acquires First Drug Candidate from TRND Program

NCATS Launches Chemical Toxicity Data Model Competition

Research Project Leverages NCATS Chemical Screening Approach and Resources

Small Businesses: Don't Miss Upcoming Application Deadlines

Upcoming Events

NCATS in the News

Collaborate with NCATS Scientists

Image of sickle cell with other cells

Industry Acquires First Drug Candidate from TRND Program

On July 9, 2014, NIH announced that biopharmaceutical company Baxter International has acquired a drug candidate developed by NCATS' Therapeutics for Rare and Neglected Diseases (TRND) researchers and collaborators. The small molecule, Aes-103, is designed to treat sickle cell disease, a genetic blood disorder that affects millions worldwide, including approximately 100,000 people in the United States — among them, 1 in 500 African-Americans. This is the first potential treatment specifically developed to target the underlying molecular mechanism of the disease as well as the first time industry has acquired a drug candidate developed with TRND program resources. Read the NIH news release.

Graphic of DNA and associated code

NCATS Launches Chemical Toxicity Data Model Competition

On July 16, 2014, NCATS launched the Toxicology in the 21st Century (Tox21) Data Challenge 2014, a crowdsourcing competition to develop computational models that can better predict chemical toxicity. The Tox21 initiative is designed to improve current toxicity assessment methods, which are slow and costly. The challenge model submission deadline is Nov. 14, 2014, 11:59 p.m. ET. NCATS will showcase the winning models in January 2015. Read the full announcement and register today!

RNAi screen of Parkin

Research Project Leverages NCATS Chemical Screening Approach and Resources

A key NCATS approach to solving translational problems is to focus on improving scientific methods and tools that can speed the translation of laboratory discoveries into new treatments for patients. With support from the Michael J. Fox Foundation for Parkinson's Research, James Inglese, Ph.D., director of NCATS' Assay Development and Screening Technology Laboratory, and Richard Youle, Ph.D., of the National Institute of Neurological Disorders and Stroke, are leading a project that showcases how NCATS' chemical screening resources can advance development of potential therapeutics for a broad range of diseases. Read the full feature.

Small Businesses: Don't Miss Upcoming Application Deadlines

Next SBIR/STTR Application Deadline: August 5, 2014

With $17.6 million in funding annually, NCATS seeks to increase small business participation in federally supported research and development as well as private-sector commercialization of technology developed with federal support. The NCATS Small Business Innovation Research (SBIR) and Technology Transfer (STTR) programs supports these entrepreneurs. Read more about NCATS' SBIR/STTR Topics of Interest.

NIH hosted a pre-submission webinar about its SBIR/STTR programs on March 6, 2014. Access the webinar recording, PowerPoint slides and transcript to learn about the registration and submission process, common submission errors, eligibility requirements and recent changes, venture capital support, switching between SBIR and STTR, the SBIR Direct Phase II opportunity, and other relevant program updates.

I-Corps at NIH Application Deadline: August 7, 2014

Active NCATS SBIR/STTR grantees are eligible to apply for I-Corps at NIH, a collaborative funding and mentorship opportunity with the National Science Foundation's I-Corps program. This nine-week immersion program is designed to help participants commercialize their innovations. For the first time, this innovative and transformative entrepreneurial training program is available to biomedical and life sciences companies.

The National Cancer Institute hosted an informational webinar about I-Corps at NIH on July 2, 2014. View the slides to learn more from experts with deep expertise in the biotechnology and life sciences industries.

Upcoming Events


National Conference on Engaging Patients, Families and Communities in all Phases of Translational Research to Improve Health

On Aug. 21–22, 2014, Duke Translational Medicine Institute will hold the 2014 Community Engagement Conference at the Bethesda North Marriott Hotel and Conference Center in Bethesda, Maryland. The goal of this conference is to present and compare perspectives and examples of methods of engagement in research that include individuals, including patients and families, to community organizations and disease advocates as well as clinicians and other health professionals.


NCATS Advisory Council/Cures Acceleration Network (CAN) Review Board Set to Meet September 19

On Sept. 19, 2014, NCATS will hold a joint meeting of the NCATS Advisory Council and the CAN Review Board on the NIH campus in Bethesda, Maryland. The meeting will feature reports from NCATS Director Christopher P. Austin, M.D., and others about the Center's initiatives, policies, programs and future direction. For more information, visit the NCATS Advisory Council page and the CAN Review Board page of the NCATS website.


Addressing Irreproducibility in Target Validation

On Oct. 23, 2014, the Novartis Institutes for Biomedical Research in Cambridge, Massachusetts, will host a one-day conference to address a growing concern from scientists and the public, which contend that the complex system for ensuring the reproducibility of biomedical research is failing and is in need of restructuring. The conference in planned around a series of talks and panels by industry, academia and government scientists, as well as editors from leading journals, to lend perspective and brainstorm practical solutions.

NCATS in the News

Collaborate with NCATS Scientists

NCATS researchers are seeking collaborators in the following areas:

Bridging Interventional Development Gaps (BrIDGs) Program

The BrIDGs program makes available, on a competitive basis, certain critical resources needed for the development of new therapeutic agents. The next opportunity to apply to the BrIDGs program tentatively is scheduled for January/February 2015. Visit the BrIDGs page for more information.

NCATS Chemical Genomics Center (NCGC)

NCGC is one of the centers in the Molecular Libraries Probe Production Centers Network (MLPCN), which is an NIH Common Fund initiative. Through the MLPCN, NCGC offers biomedical researchers access to large-scale screening capacity along with the medicinal chemistry and informatics expertise necessary to identify chemical probe molecules and to study the functions of genes, cells and biochemical pathways. For inquiries or to obtain NCGC probe molecules, contact Ajit Jadhav.

NCGC researchers also seek collaborators for assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics. Learn more.

NIH RNA Interference (RNAi) Initiative

The NIH RNAi initiative, administered by NCATS, provides state-of-the-art, high-throughput RNAi genome-wide screens for humans and mice. This resource is available only to NIH researchers. Scientists interested in performing high-throughput RNAi screens can contact Scott Martin, Ph.D., for more information.

Toxicology in the 21st Century (Tox21) Program

The Tox21 program aims to test 10,000 chemicals and evaluate their potential to cause health problems. Any investigator may propose the development of biological assays for high-throughput screening.

To suggest an assay, submit an assay nomination form to Menghang Xia, Ph.D. Proposed assays must be compatible with the high-throughput screening guidelines as described in the assay guidance criteria.

Research Opportunities and Announcements

Visit the NCATS Open Opportunities page for a complete list of funding and program announcements.

Onsite Tools and Technologies for Heart, Lung, and Blood Clinical Research Point-of-Care SBIR (R43/R44) • RFA-HL-14-011

Onsite Tools and Technologies for Heart, Lung, and Blood Clinical Research Point-of-Care STTR (R41/R42) • RFA-HL-14-017

Request for Information (RFI): Public-Private Partnerships for Organ Systems and Platforms Developed by Microphysiological Systems (MPS) Investigators • NOT-TR-14-008

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