Journal of Genetic Syndromes & Gene Therapy
Open Access

Study of rare disease utilizing bioinformatics approach

2^nd World Congress on Rare Diseases and Orphan Drugs

June 29-30, 2017 London, UK

Weida Tong

National Center for Toxicological Research - US FDA, USA

Posters & Accepted Abstracts: J Genet Syndr Gene Ther

Abstract :

A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population. Among ~7000 rare diseases reported, only ~500 orphan drugs are available. The U.S. Food and Drug Administration (FDA) has implemented various policies to improve treatment options for rare diseases. As a result, in the last 10 years, over one-third of all new drug approvals by FDA were for rare diseases. There are a number of challenges to develop orphan drugs for rare diseases. For example, the lack of knowledge about the natural history of rare diseases is one important challenge for orphan drug development. Bioinformaticsbased approaches have the potential to offer systematic insights into the complex relationships among drugs, targets and rare diseases necessary for better understanding the natural history of rare disease and seeking for potential treatment options. We have employed diverse bioinformatics approaches with integrated data related to rare diseases to uncover the underlying mechanism of rare diseases and explore the potential application of the existing marketed drugs for the treatment of rare diseases. This presentation will describe several projects relating to the development of a bioinformatics framework for drug repositioning in rare diseases, deciphering miRNA transcription factor feed-forward loops to identify drug repurposing candidates for Cystic Fibrosis, and potential reuse of oncologic drugs for the treatment of rare diseases.

Biography :

Email: weida.tong@fda.hhs.gov