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Therapeutics for Rare and Neglected Diseases

African boy getting a shot

The NCATS Therapeutics for Rare and Neglected Diseases (TRND) program is designed to encourage and speed the development of new drugs for rare and neglected diseases. TRND stimulates drug discovery and development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses. Read the TRND fact sheet.

TRND collaborations offer an opportunity to partner with TRND researchers and gain access to rare and neglected disease drug development capabilities, expertise, and clinical/regulatory resources in a collaborative environment with the goal of moving promising therapeutics into human clinical trials. To foster collaborations, TRND uses an application and evaluation process to select collaborators. Selected investigators provide the drug project starting points and ongoing biological/disease expertise throughout the project.

The drug development process is complicated and expensive. Innovations in drug discovery and development and management of development programs can substantially reduce the risks, time delays and costs of advancing basic research breakthroughs into treatments. Thus, in addition developing new candidate drugs for rare and neglected diseases, the TRND program is designed to advance the entire field of drug development by encouraging scientific and technological innovations aimed at improving success rates in the crucial pre-clinical stage of development.

woman scientist working in labTRND bridges the gap that often exists between a basic research discovery and the testing of new drugs in humans. To close the gap, the work of TRND includes the discovery, optimization and pre-clinical testing of therapies, with the goal to generate sufficient quality data to support successful Investigational New Drug (IND) applications to the U.S. Food and Drug Administration (FDA) and first-in-human studies as necessary. Therapeutic clinical candidates that reach this stage should be attractive to biotechnology and pharmaceutical companies to take into clinical development.

TRND expands upon the successes of the Molecular Libraries Program and the NCATS Chemical Genomics Center. The Molecular Libraries Probe Production Centers Network initiative has produced high-quality assay screening facilities across the United States that develops probes for exploring biology and disease. However, there is often a gap between fundamental exploration of biological processes and the point at which biotechnology and pharmaceutical companies adopt these research discoveries to develop therapeutic programs. Adoption of drug development programs for rare and neglected disease therapeutics is that much more difficult due to the limited market for such drugs. Through collaborative projects, TRND leverages both public- and private-sector investments to develop new drugs for patients with rare and neglected diseases.

Through the TRND program, NCATS scientists collaborate with researchers from a broad and diverse range of scientific disciplines and disease areas. TRND encourages investigators from both within and outside the NIH to submit projects for work within its intramural facility. These ongoing collaborations benefit researchers, and more importantly, patients with rare and neglected diseases.

TRND Active Projects Portfolio

Therapeutic Area/DiseaseOrganization Name(s)Partner Type(s)
Autoimmune pulmonary alveolar proteinosisCincinnati Children’s HospitalAcademic
Core binding factor leukemiaNational Human Genome Research Institute (NHGRI)NIH intramural labs
Creatine transporter defectLumos Pharma, Inc.Biotech
Fibrodysplasia ossificans progressivaMassachusetts General HospitalAcademic
HemoglobinopathiesPhoenicia Biosciences, Inc.Biotech
Hereditary inclusion body myopathyNew Zealand Pharmaceuticals, NHGRIBiotech, NIH intramural labs
HypoparathyroidismEli Lilly & Co.Pharmaceutical
LEOPARD syndromeBeth Israel Deaconess Medical CenterAcademic
MalariaLoyola University ChicagoAcademic 
Niemann-Pick disease type CAra Parseghian Medical Research Foundation, Niemann-Pick Type C Support of Accelerated Research (NPC-SOAR), Einstein College of Medicine, University of Pennsylvania, Washington University, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and National Human Genome Research Institute (NHGRI)Disease foundation, academic, NIH intramural labs
Retinitis pigmentosaUniversity of California, IrvineAcademic
SchistosomiasisCoNCERT PharmaceuticalsBiotech

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TRND Information

Therapeutic Successes

Young boy with Proteous Syndrome

A study published online in Science Translational Medicine on Feb. 25, 2015, found that NCATS’ TRND and BrIDGs programs have led to the reduced cost of developing new drugs and reduced financial risks. Read the full feature.


Image of the cerebellum of the brain affected by Niemann-Pick type C1 at the near-end stage of the disease (Eunice Kennedy Shriver National Institute of Child Health and Human Development Photo)

On Jan. 7, 2015, NIH announced that NCATS and other NIH researchers are collaborating with Vtesse, Inc., to develop treatments for Niemann-Pick disease type C and other rare lysosomal storage disorders. Read the NIH news release.

New TRND Projects

Mosquito on finger

On Oct. 15, 2014, NCATS announced three new pre-clinical drug development projects aimed at finding treatments for malaria, Lassa fever, and two rare blood disorders: beta-thalassemia and sickle cell disease. Read the announcement.