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Approved Research

Identification of genetic modulators and therapeutic targets for RNA base editing

Principal Investigator: Dr Howard Stern
Approved Research ID: 85426
Approval date: June 24th 2022

Lay summary

Korro Bio is an early-stage pharmaceutical company focused on developing treatments for rare diseases caused by genetic mutations as well as common diseases without a simple genetic cause.

Certain diseases are caused by genetic mutations in DNA. However, establishing a strong connection between a genetic mutation and a disease is not always an easy task. The UK BioBank offers a unique combination of data that helps to establish connections between genetic mutations and disease features, including a person's DNA, their medical history and lifestyle, disease symptoms, and measurements from their blood or tissues. With this combination of information Korro would look for connections between genetic mutations in DNA and which diseases or symptoms they lead to. Some genetic diseases are very rare, or are not easy to diagnose, so both the breadth of the UK BioBank data and the large number of participants are important for working on rare diseases.

Korro would use UK BioBank data to strengthen existing understanding of the relationships between genetic mutations and the diseases that they cause as well as establish new connections between genetic mutations and diseases. Korro's technology allows us to correct genetic mutations by periodically administering a corrective drug. With better knowledge of the genetic mutations that cause diseases, we can work on treatments that correct those mutations. The data from UK BioBank would also help us address questions such as: At what age do patients begin to show symptoms of the disease? In which organs of the body does the genetic mutation cause problems? When would be the right age to begin treatment?

More common diseases without a simple genetic explanation could also be treated by Korro's technology. Korro could develop a drug that would change the properties of a protein involved in disease.  As an example, the protein amyloid is involved in Alzheimer's disease, and Korro's technology might be able to correct the protein, so it is less likely to cause disease.  The data from the UK BioBank would help us identify which protein corrections might help mitigate various diseases and which protein corrections would be most amenable to Korro's technology.

The public health impact of this work is potential new drugs to mitigate suffering from difficult-to-treat diseases, many of which do not have meaningful treatments available today.