We wish to use the UK BioBank to identify disorders that are particularly well suited for developing new treatments based on oligonucleotide therapeutics. Oligonucleotide therapeutics are a new type of drug that significantly expands our treatment capabilities for disorders that have a genetic cause (where a genetic cause consists of changes in the DNA sequence). For these disorders, the genetic cause can be discovered by analyzing the UK Biobank data. This work may lead to the development of new treatments for disorders with an unaddressed need. We anticipate a period of 36 months to analyze the UK BioBank data and discover targetable disorders, review the literature for these disorders, if necessary investigate experimentally additional details. After this stage, the therapeutic design and development process may take 4-5 years and lead to a clinical trial (which is the clinical testing of the drug safety and efficacy). A clinical trial may last 3-6 years, and a successful clinical trial may lead to public access for the new therapeutic agent.
