Esme Asaad '26, Jack Simes '26, Dave Olugemo '26: Korro Bio

Alpha-1 Antitrypsin Deficiency, or AATD, is a rare disease that affects an estimated 100,000 patients in the United States and a similar number in Europe. AATD is caused by a genetic mutation that creates malformed Alpha-1 Antitrypsin protein. Instead of protecting the lungs from damage and disease as they are supposed to, these abnormal proteins get stuck in the liver, leading to COPD and liver failure among other serious complications. Advances in RNA editing technology at the Cambridge pharmaceutical start-up Korro Bio have created promise for a treatment to mitigate the effects of AATD by reversing the mutation in the RNA and enabling the production of healthy Alpha-1 Antitrypsin protein.

This summer, we had the opportunity to work with Korro Bio’s clinical team as they conducted a clinical trial to test the safety and efficacy of their new treatment to improve the quality of life of patients living with AATD. While technologies like CRISPR, which can edit DNA, have earned significant attention in recent years, DNA editing therapies carry considerable risks because changes made to the DNA are difficult to reverse. However, Korro’s treatment uses a protein already present in the body, called ADAR, to edit the mRNA instead. By editing the mRNA, the treatment repairs the shape of the resulting protein, but because mRNA is regularly discarded and rebuilt, the change is not permanent. 

We began our time at Korro with Alex Loboda, the Director of Clinical Quality, who ensures that the clinical trial is conducted according to federal regulations and the medical community’s Good Clinical Practices (GCP) guidelines. We spent the first day completing an online course to become certified in GCP. We learned about regulations on the recruitment of patients, oversight of the trial, and reporting of side effects, which are in place to protect the safety of trial participants and the quality of the data collected. We then put together a presentation on GCP and the history of regulation in clinical trials. Alex is responsible for auditing clinical trial sites and other parties that Korro works with to ensure they are complying with established procedures, so we had the opportunity to mock-audit some members of the clinical team to learn about their individual roles and about the auditing process. In addition, we assisted Alex in identifying potential sites for the next stages of the study by reviewing ongoing and past studies that have involved AATD and using an Excel sheet to analyze the trends in enrollment rate and eligibility criteria at these study sites.

Esme, Jack, and Dave meeting with Alex.

We spent our second week with Rune Aabo Christensen, who works in Clinical Operations and coordinates the ongoing trial. We completed activities that mimicked the problems he must solve every day to keep the trials moving forward. For example, we had to figure out how to condense an address into a limited number of lines and characters while retaining all of the important information. We also learned how to use Excel to create our own patient trackers.

We were each assigned a desk and computer.

In the third week, we worked with Peter Aquino, the pharmacovigilance physician, who is responsible for monitoring the side effects of the drug as they appear and weighing them against the treatment’s potential benefits. We researched and learned from him about historical disasters in drug safety, why drugs are withdrawn from the market, and how to read a prescription information label. 

In our fourth week, we had the opportunity to work with Jon Fischer from the Strategy and Portfolio Planning team to help evaluate whether a specific disease would be ideal for the Development Team to target. We weighed various considerations, including the number of patients currently affected by the disease, the efficacy of existing treatments, the degree of unmet need, the market opportunity, and applicable regulatory pathways. We then presented our findings and our recommendation on whether Korro should pursue a treatment for the disease to the Strategy and Portfolio Planning Team (S&PP). 

Throughout our internship, we joined weekly Core Team meetings with the entire clinical and regulatory team and had the opportunity to see business, legal, medical, and logistical perspectives come together to form a complete picture of the intricate process of drug testing and approval.

We joined weekly meetings with the Core Team.

We also had the opportunity to talk to the researchers at Korro about their roles as they develop other drugs with this same novel RNA editing technology. Towards the end of our internship, we had a chance to tour the labs and see the machines and processes that the researchers use to develop and test new treatments.

Cassidy, Mafatta, Dave, Jack, Katie, and Esme touring the NGS room.

Nam showing us the robot she uses to isolate mRNA.

Mafatta demonstrating her multichannel pipette.

At Korro, we have learned about the pharmaceutical industry and the diversity of career paths in biotech, as well as what it is like to work in a business setting. Thank you to Kemi Olugemo, Alex Loboda, Rune Aabo Christensen, Peter Aquino, Brianna Cassidy, Mr. Schlenker, and Dr. Bloch, and everyone else who helped to provide us with the opportunity to learn and experience so much at Korro. 

To conclude, we each have a few individual thoughts to share.

Esme: I appreciated the opportunity to learn about the world of pharma and the multitude of careers in this field, as well as the deep care that is given to every stage of the development of a drug. I am grateful to everyone at Korro who was quick to welcome us and took the time to talk to us about their roles at the company, the paths that led them there, and their advice as we look to our own futures.

Dave: I especially enjoyed learning about the work of Jon and the S&PP team. Seeing the two sides of biology and business come together was very intriguing and allowed me to develop a new understanding of the vast biotech world. We were able to evaluate the market potential for a specific indication while also making sure the mechanism of action that Korro uses is compatible with the indication as well. I am beyond grateful to everyone at Korro who worked with us and allowed us to learn from their experiences!

Jack: One of the highlights of our time with Korro was meeting with Dr. Aquino and learning about drug safety and pharmacovigilance. It was fascinating to see how side effects are monitored and balanced with potential benefits, and I liked learning how to read prescribing information and understand how it's used in real-world settings. This experience gave me a deeper appreciation for the safety side of clinical trials and opened my eyes to a side of biotech I hadn’t considered before.