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Approved Research

Leveraging whole genome sequencing to identify genes with protective expression increase variants

Principal Investigator: Dr Daniele Merico
Approved Research ID: 83180
Approval date: March 21st 2022

Lay summary

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 contributing genetic factor (where a genetic factor consists of changes in the DNA sequence). For these disorders, protective genetic factors can be discovered by analyzing the UK Biobank data and exploited to design oligonucleotide therapeutics that mimic their effect. 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 3-4 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.