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TRND Scientific Capabilities

TRND has a world-class, in-house team of experts covering the range of preclinical and early clinical development.

Scientific Capabilities

The Therapeutics for Rare and Neglected Diseases (TRND) program leverages both internal capabilities and external contract resources to advance projects most efficiently. TRND is not a grant program. TRND provides in-kind resources to push forward preclinical drug development. 

TRND Expertise

The TRND program supports a world-class, in-house team of drug development experts and project managers with experience and knowledge across the field of drug discovery and development.

TRND staff can provide the following expertise, as needed:


  • Medicinal chemistry optimization
  • Evaluation of functional activity, potency, pharmacokinetics, pharmacodynamics and efficacy
  • Biomarker development
  • Definition or optimization of dose and schedule for in vivo activity
  • Development of pharmacology assays
  • Conduct of pharmacology studies with pre-determined assays
  • Acquisition of bulk substance (Good Manufacturing Practice [GMP] and non-GMP)
  • Development of suitable formulations
  • Development of analytical methods for bulk substances
  • Production of dosage forms
  • Stability assurance for dosage forms
  • Range-finding initial toxicity
  • Investigational New Drug (IND)–directed Good Laboratory Practice toxicology, with correlative pharmacology and histopathology
  • Planning of clinical trials
  • Regulatory support to increase the likelihood of satisfying U.S. Food and Drug Administration requirements
  • IND filing support         

Internal Resources

As part of the NIH Intramural Research Program, the TRND program also has access to shared resources on the NIH campus in Bethesda, Maryland, including small and large animal facilities and the NIH Clinical Center, the world’s largest facility dedicated to clinical research.

External Resources

The external TRND network includes organizations across the rare diseases patient community and, through close collaboration with the U.S. Food and Drug Administration, regulatory experts.

In situations where TRND internal resources are insufficient to generate key materials or data, such as Good Manufacturing Practice production and multi-specifies Good Laboratory Practice toxicology studies, NCATS leverages its government contracting capability to work with expert contract research organizations.


Last updated on November 7, 2023