The Therapeutics for Rare and Neglected Diseases (TRND) Program is a new, congressionally-mandated NIH program to develop drugs for rare and neglected diseases. The work of TRND will include the discovery, optimization, and pre-clinical testing of therapies, with the goal to generate sufficient, quality data to support successful Investigational New Drug Applications to the FDA. Therapeutic clinical candidates that reach this stage should be attractive to biotechnology and pharmaceutical companies to take into clinical development. TRND will build on advances made through the chemical genomics efforts of the NIH Roadmap and will work to develop scientific paradigms and technologies to improve the success rates in the preclinical stage of drug development generally. Through collaborative projects, TRND will leverage both public and private sector investments to develop new drugs for patients with rare and neglected diseases.