Skip to main content
HHS Logo U.S. Department of Health & Human Services NIH logo National Institutes of Health
Home
  • Site Map
  • |
  • Contact
  • Research
    • Preclinical Innovation
      • Improving the Drug Development Process
      • Repurposing Drugs
      • Testing & Predictive Models
      • Core Technologies
      Clinical Innovation
      • Clinical and Translational Science Awards (CTSA) Program
      • Rare Diseases Clinical Research Network (RDCRN)
      • Patient Registries
      • Clinical Research Toolbox
      Rare Diseases Research and Resources
      • Gene Therapy and Gene Editing Programs
      • Rare Diseases Clinical Research Network (RDCRN)
      • Therapeutics for Rare and Neglected Diseases (TRND)
      • Additional Rare Diseases Research and Initiatives
      Strategic Alliances
      • Alliances at NCATS
      • Licensing Opportunities
      • Forms & Model Agreements
      • Work with Strategic Alliances
      Patient/Community Engagement & Health Information
      • Genetic and Rare Diseases Information Center
      • NCATS Toolkit for Patient-Focused Therapy Development
      • Rare Diseases Registry Program (RaDaR)
      • Rare Diseases Community Resources
      Expertise & Resources
      • Preclinical Research Toolbox
      • COVID-19 OpenData Portal
      • Clinical Research Toolbox
      • PaVe-GT Pilot Project
      • National COVID Cohort Collaborative (N3C)
      NCATS' Role in the NIH HEAL Initiative
      • About NCATS’ Role in the NIH HEAL Initiative
      • Accelerating the Translation of Novel Compounds Toward INDs for Subsequent Clinical Testing
      • Fiscal Year 2019 Funded Projects and Prizes
      • NIH HEAL Initiative Funding & Collaboration Opportunities Led by NCATS
      NCATS Programs & Initiatives
  • Funding & Notices
    • Open Funding Opportunities
      • Closed Opportunities
      • Conference Grant Information
      • NCATS Program-Specific Funding Information
      Small Business Opportunities
      • About Small Business Opportunities
      • SBIR & STTR Research Priorities
      • Small Business Funding
      • Small Business Resources
      Funding Policy & Scientific Review
      • Funding Policy
      • PUBLIC NOTICE
      • Scientific Review
      • Cures Acceleration Network
      Grantee Information
      • Submission of Prior Approval Requests
      • Prior NIH Approval of Human Subjects Research Frequently Asked Questions
      NCATS Challenges and Prize Competitions Program
      • Bias Detection Tools in Health Care Challenge
      • LitCoin Natural Language Processing (NLP) Challenge
      • LitCoin Pilot Design Challenge
      • NCATS Rare Diseases Are Not Rare! 2018 Challenge Details
      • NCATS Rare Diseases Are Not Rare! 2020 Challenge Details
  • News & Media
    • News & Events
      • Releases & Announcements
      • Media Coverage
      • Events
      • Grantee & Partner News
      Stories & Publications
      • Features and Briefs
      • NCATS E-Newsletter
      • I Am Translational Science
      • Scientific Publications
      Multimedia
      • Photos
      • Videos
      • Infographics
      • Fact Sheets
      Connect with NCATS
  • About Translation
    • Translational Science Education & Training
      • Translational Science Principles
      • Translational Science Training at NCATS
      • Translational Science Training at Partner Institutions
      • Translational Science Training and Education Resources
      • About the NCATS Education Branch
      Translational Science Spectrum
      Issues in Translation
      • Artificial/Machine Intelligence
      • Clinical Research Efficiency
      • Collaboration and Partnerships
      • Data Transparency and Release
      • De-Risking Therapeutic Development
      • Predictive Efficacy and Toxicology
      Drug Discovery, Development and Deployment Maps
      Translational Science Resources
      Translational Science Skills
  • About NCATS
    • Center Overview
      • About the Center
      • Director’s Corner
      • NCATS Strategic Plan
      • NCATS Staff Profiles
      NCATS Programs & Initiatives
      • 3-D Tissue Bioprinting Program
      • Stem Cell Translation Laboratory
      • Assay Development and Screening Technology (ADST)
      • Biomedical Data Translator
      • Bridging Interventional Development Gaps (BrIDGs)
      • Chemistry Technology
      • Discovering New Therapeutic Uses for Existing Molecules
      • Genetic and Rare Diseases Information Center (GARD)
      • Matrix Combination Screening
      • Early Translation Branch (ETB)
      • A Specialized Platform for Innovative Research Exploration (ASPIRE)
      • A Translational Approach to Addressing COVID-19
      • Antiviral Program for Pandemics
      • Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes
      • Multidisciplinary Machine-Assisted, Genomic Analysis and Clinical Approaches to Shortening the Rare Diseases Diagnostic Odyssey
      • National COVID Cohort Collaborative (N3C)
      • The Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (BGTC)
      • NIH Common Fund Programs
      • Rare Diseases Registry Program (RaDaR)
      • Tissue Chip for Drug Screening
      • Toxicology in the 21st Century (Tox21)
      • Functional Genomics Lab
      • Therapeutics for Rare and Neglected Diseases (TRND)
      Work with NCATS
      • Contribute to NCATS Research
      Advisory Groups
      • NCATS Advisory Council
      • Cures Acceleration Network (CAN) Review Board
      • Council Concept Clearances
  • Research
    • Preclinical Innovation
    • Clinical Innovation
    • Rare Diseases Research and Resources
    • Strategic Alliances
    • Patient/Community Engagement & Health Information
    • Expertise & Resources
    • NCATS' Role in the NIH HEAL Initiative
    • NCATS Programs & Initiatives
  • Funding & Notices
    • Open Funding Opportunities
    • Small Business Opportunities
    • Funding Policy & Scientific Review
    • Grantee Information
    • NCATS Challenges and Prize Competitions Program
  • News & Media
    • News & Events
    • Stories & Publications
    • Multimedia
    • Connect with NCATS
  • About Translation
    • Translational Science Education & Training
    • Translational Science Spectrum
    • Issues in Translation
    • Drug Discovery, Development and Deployment Maps
    • Translational Science Resources
    • Translational Science Skills
  • About NCATS
    • Center Overview
    • NCATS Programs & Initiatives
    • Work with NCATS
    • Advisory Groups
  1. Home
  2. About NCATS
  3. NCATS Programs & Initiatives
  4. Therapeutics for Rare and Neglected Diseases (TRND)
  5. TRND Projects
  6. Active TRND Projects
  • About TRND
  • TRND Scientific Capabilities
  • Work with TRND
  • TRND Projects
    • Active TRND Projects
    • Completed TRND Projects
    • TRND Clinical Research Studies
    • Drug Repurposing Screening for Rare and Neglected Diseases
    • Gene Therapy Platform for Rare Diseases

Active TRND Projects

Click on the links below to view active TRND projects:

  • Development of Malaria Transmission-Blocking Drugs
  • A Novel Compound for Targeted Treatment of Core Binding Factor Leukemia
  • Repurposing of a Long-Acting Parathyroid Hormone Analog for the Treatment of Hypoparathyroidism
  • ​A Treatment for Patients with Jansen’s Metaphyseal Chondrodysplasia

Contact

TRND Program staff

Gene Therapy Advances to Clinical Trial

TRND Pompe image​NCATS and Duke University scientists help advance a gene therapy for Pompe into clinical testing for the first time.

Last updated: 02-09-2023

▲ Back to top
  • Connect with us:

  • Facebook Icon
  • LinkedIn Icon
  • Twitter Icon
  • YouTube Icon
  • Flickr Icon
  • Address Book Icon
  • E-Mail Icon
  • NCATS Home
  • Site Map
  • Privacy Notice
  • Comment Policy
  • Disclaimer
  • Accessibility
  • FOIA
  • OIG
  • HHS Vulnerability Disclosure

If you have problems viewing PDF files, download the latest version of Adobe Reader

For language access assistance, contact the NCATS Public Information Officer

National Center for Advancing Translational Sciences (NCATS), 6701 Democracy Boulevard, Bethesda MD 20892-4874 • 301-594-8966