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DNA Double Helix

Gene Therapy for Rare Condition Closer to Market

NCATS and Agilis researchers collaborated to bring a gene therapy for the rare pediatric condition aromatic L-amino acid decarboxylase deficiency closer to market.


Work with Us

The TRND program currently is seeking new collaborations. Proposals are due Sept. 30, 2017. Learn more and contact TRND.

Therapeutics for Rare and Neglected Diseases (TRND)

The TRND program supports pre-clinical development of therapeutic candidates intended to treat rare or neglected disorders, with the goal of enabling an Investigational New Drug (IND) application. Learn more.

Find NCATS Programs & InitiativesAccess NCATS Expertise & Resources

About TRND

TRND supports pre-clinical development from lead optimization through IND application.

Scientific Capabilities

TRND offers world-class in-house experts and connections to external resources.

TRND Projects

Explore active and completed therapeutic development projects supported by TRND, including projects with clinical activities.

Why TRND Matters

There are more than 6,500 identified rare and neglected diseases, yet only about 250 treatments are available for these conditions. One reason is that limited numbers of patients can make gathering information and designing drug studies difficult. As a result, scientists often know little about the symptoms and biology of these conditions. Another obstacle is that some private companies may find it difficult to justify the cost of developing drugs for such small rare disease markets. Even in the case of infectious diseases — such as malaria — that inflict health burdens on large numbers of people in the developing world, the private sector often neglects therapeutic development because of insufficient economic incentives. Learn more about TRND.