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Therapeutics for Rare and Neglected Diseases

Considerations for TRND Collaborators

TRND supports projects across phases of development that include pre-clinical and Investigational New Drug (IND) application-enabling studies. Individual projects can begin at various stages, depending on the treatment type. TRND does not support direct entry into clinical testing.

TRND projects typically represent one of the following general stages or types:

  • Lead Optimization: In this stage, researchers aim to identify and develop a potent, specific development candidate. These early-stage candidates must include:
    • Clear structure-activity relationships in at least two structurally distinct chemical series or a well-defined biological lead;
    • Reproducible activity in primary and orthogonal assays;
    • Efficacy in an accepted animal model (or, when not available, cellular model) of the disease;
    • Initial indications of favorable absorption, distribution, metabolism and excretion properties; and
    • Favorable head-to-head comparisons to the prior art.
  • New Molecular Entity or New Biologic Entity: This type of project involves an advanced lead molecule, requiring completion of IND-enabling studies. At minimum, the selected candidate molecule will include clear efficacy data, good drug metabolism and pharmacokinetic properties, and initial non-Good Laboratory Practice safety studies demonstrating no gross toxicities. Development candidates may require completion of IND-enabling pharmacokinetics, pharmacodynamics, toxicology or formulation studies.
  • Gene or Cell-Based Therapy: Gene or cell-based therapies use technologies to deliver genes or living, intact cells to patients to treat or prevent certain diseases. TRND only considers late-stage gene or cell-based therapy projects at this time. At minimum, these projects should have an identified delivery vector, a selected curative gene construct and a validated animal disease model(s).
  • Repurposing: A repurposing candidate represents a drug previously approved by the Food and Drug Administration to treat another disease that has been shown to also be effective in an animal model (or, when not available, cellular model) of a rare condition or neglected tropical disease. Relying heavily on the previous indication data package, a repurposing candidate will be more advanced, in need of formulation, dose-finding, disease-specific toxicology or other studies to enable clinical testing.
  • Platform Technology: Through these projects, investigators seek to develop a therapeutic candidate directed toward a specific rare condition or neglected tropical disease, but the technology represents a platform with the potential to address a wider range of additional disorders and that can enable more efficient future development of other therapeutics. Both early- and later-stage projects are considered, as described above.
Last updated: 07-17-2017
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