Analysis of UK Biobank Cohorts to Accelerate Therapies to Treat Neurological Disease
Approved Research ID: 93490
Approval date: November 15th 2022
Ribonucleic acids (RNA) mediate the action of chromosomes in every cell in the body, and the RNA from a specific gene is key in turning that gene's instructions into actual function. A number of diseases are caused by abnormalities in genes that are turned into abnormal RNA. For example, Huntington's Disease is caused by lengthening of a specific gene on chromosome 4, and this abnormality is reflected in the corresponding RNA. If one could degrade this pathological RNA throughout the brain, one might arrest Huntington's Disease. Alternatively, in some neurological diseases there may be health benefit to reducing the expression of normal genes that may be harmful, analogous to genes responsible for high levels of low-density lipoproteins (LDL) that are linked to risk of cardiac disease. The body has a natural physiological process for regulating RNA levels, called RNA interference (RNAi), discovery of which was awarded the Nobel prize in 2006. Artificial RNAi, then, offers the opportunity for specific regulation of individual genes to attack a number of diseases.
Atalanta Therapeutics is a biotechnology company based in Boston, Massachusetts, USA, dedicated to developing therapeutics for neurological and neurodegenerative disease with short interfering RNA (siRNA) technology. Our version of siRNA was engineered to have a selective, durable, and well-tolerated action throughout the brain and central nervous system. Many rare genetic diseases of the nervous system (like Huntington's) have no effective treatments, and likewise common diseases including Alzheimer's Disease are in need of truly transformative treatments that attack the root cause of disease. Accordingly, Atalanta's siRNA technology could be applied to a number of diseases, if key unknowns can be addressed.
The data and infrastructure of the UK BioBank will provide biological understanding of human disease that is key to making actual therapeutics and bringing therapeutics to patients. The UK Biobank will help us select the safest targets for our siRNA, and also help discover any easily measurable biomarkers associated with action of our siRNA that can be used to test the dose at which a potential therapeutic works. The health of persons carrying natural genetic variants in the target of interest will be analyzed, to better understand potential patient populations and to give some measure of the overall prevalence of a specific disease. Results will be made public per UK Biobank policies.