Analysis of genotype and phenotype data from the UK Biobank to characterize genes and pathways impacting human disease, predict efficacy, and potential adverse outcomes of Ionis drug targets.
Approved Research ID: 94121
Approval date: December 6th 2022
Drug targets with evidence of efficacy in humans, such as obtained from analyzing human genetic data, have a significantly higher likelihood of becoming approved, successful treatments. Hence it is now a necessity that pharmaceutical companies incorporate human genetics evidence when designing new therapeutics. We are applying to access the extensive health and medical data and genetic data collected by the UK Biobank in order to integrate human genetic findings into the drug discovery and development pipeline at Ionis. We will analyze human genetic data to identify novel drug targets, prioritize Ionis targets in development with supporting human genetic evidence or by discovering potential adverse effects, and to identify additional diseases that could benefit from existing Ionis drugs. Our analyses will include diseases in the cardiometabolic, renal, neuroscience, ophthalmology and immunology fields. Leveraging human genetics to identify targetable genes/pathways implicated in genetically complex, common diseases will help a much greater number of patients benefit from the therapeutic technologies that Ionis has successfully used for treatment of rare genetic disorders. We hope that incorporating human genetic evidence in our therapeutic pipeline will help streamline and accelerate the development of successful and safe therapies at Ionis, thus benefiting more patients, and faster.