BIO 2025: New Perspectives on RNA Editing

As the Biotechnology Innovation Organization (BIO) International Convention meets in Boston from June 16–19, 2025, BioPharm International® caught up with Ram Aiyar, PhD, CEO and president of Cambridge, Mass.-based Korro Bio.

In the interview, Aiyar explains the broader implications of Korro’s OPERA platform, short for “Oligonucleotide Promoted Editing of RNA.” Increased precision, he said, is the key to addressing a broader range of diseases.

“This specific technology is really with that focus in mind—how do you increase the biological pathway?” Aiyar says in the interview. “So how we think about targets and how we think about indications is really focused on, what is the biology that we want to affect, that we want to increase or upregulate, and what is the biological benefit that we can get from that? The beauty here is that because the way we target RNA is so specific, we can learn from population statistics based on genetics, outcomes based on genetics, and also prior pharmaceutical experiences in terms of what targets can be upregulated. And so that's what we bring to bear.”

Aiyar will be one of the speakers in a session at BIO 2025 entitled “Cracking the Code: Effective Biotech and Pharma Partnerships Unveiled,” to be presented on Tuesday, June 17.

Korro Bio’s Chief Medical Officer Kemi Olugemo, MD, will also be representing the company that day in another presentation at BIO 2025, “Innovative Patient-Focused Models for Accelerating Rare Drug Development.”

Click the above video for Part 1 of our interview with Ram Aiyar.

Click here for all of our BIO 2025 coverage and check back soon for Part 2 of this interview.

*Scroll below for a full transcript of the video.

About the speaker

Ram Aiyar, PhD, is CEO and president at Korro Bio.

Transcript

Editor's note: This transcript is a direct, unedited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.

​Hi, I'm Ram Aiyar, CEO of Korro Bio. I've been at Korro for approximately five years. My background is primarily in healthcare, with a little bit of technology development early in my career. I have over two decades of experience in healthcare across multiple verticals, starting in research at Johnson & Johnson, moving into investment banking at JP Morgan, and then at an investment firm in Paris called Sofinnova Partners, investing in medical devices as well as therapeutics. And then for the last 12 years, I've been back in the US building companies. And so Korro is my seventh company that I'm affiliated with.

This is one of the few ones that I didn't start from scratch, but I'm super excited about its mission and goal, and why I'm really here, which is to bring medicines to patients.

So just taking a step back at Korro Bio, what we do, is modify a single alphabet on RNA. So we convert an A, or an adenosine, to an I or an inosine. And primarily that conversion is done through a protein that already exists in every segment, we just deliver a drug that's highly specific to affect that change.

The way that change manifests itself is in two ways. One is you can change and modify or modulate the expression of a protein, or you can actually change the protein to make it more functional. So as we think about what the basis of that technology are and where you can deliver things with high specificity, with our platform, which we call OPERA, and our drug candidate that we call CHORDs, no pun intended.

And the idea there is, you know, making a transient specific re-doseable titratable modification, that will provide biological benefit. So we use this platform. You know, most drugs, when they are administered, they're usually to knock down a certain biological pathway. So think of cholesterol, you know, LDL cholesterol is bad.

You want to remove it from the system. So there are many ways to sort of shut down that pathway to make bad cholesterol. But there is no really good way to increase good cholesterol, for example. Right? And so using that as an example, there are many other biological pathways that we can think about where you want to increase, like increase metabolism, increase energy.

And so this specific technology is really with that focus in mind, how do you increase the biological pathway? So how we think about targets and how we think about indications is really focused on, what is the biology that we want to affect, that we want to increase or upregulate, and what is the biological benefit that we can get from that?

The beauty here is that because the way we target RNA is so specific. We can learn from population statistics based on genetics, outcomes based on genetics, and also prior pharmaceutical experiences in terms of what targets can be upregulated. And so that's what we bring to bear. So providing a very specific titratable re-doseable way to activate a biological pathway.

So that's a very broad question, right? Because when we think about drug development these days. It's not like, our grandparents' drug development companies. You don't, you are not a chemist that's sitting, and making chemical modifications or, chemical compounds and then trying on animals and seeing if it works or not, and then think about, okay, you know, maybe this works, maybe it doesn't work, and you take a shot on goal.

These days, we have the benefit of technologies that understand how genetics work or what is the relationship between your genes and the environment that you are in and how that will work for individual people. So the idea of precision medicine or personalized medicine is really coming to bear.

But now if you take one level above, so not on an individual basis, but on a population basis, that still stratifies patients in terms of what can be done or not. So the technologies that have come along the way to understand how the environment affects your body and how it's dictated by the genes or proteins that you make is immense.

That's one component of it. The second component of it is, when you think about specificity of these drugs, right? So when you think about a pill, you don't really know entirely the mechanism, you know, three decades ago or four decades ago. You have a sense of what it can do, but these days you have a molecular understanding of what every drug actually does.

We know a lot more about biology. There's still a lot to be discovered, but we know much more, than before. And so we can be very specific. In how to target that. as an example, you know, when we think about OPERA, which is Korro's technology, we can target a single alphabet on RNA. I mean, let me put that in perspective, your transcriptome or when your genes get translated, you make millions.

The number is immense in terms of the amount of RNA that you make. About 25% of your body, inside the cells is made up of an A that forms these RNA to target one specific A in one specific gene, is unbelievable when you think about it. And so, the specificity of these technologies have come a long way.

Then the last component I would say is that, you know, technology from computational methods, technology from what one would call artificial intelligence, machine learning, computational tools, they have come a long way to design these drugs. So when we think about our drugs from, or CHORDs from OPERA there are a string of alphabets that are chemically modified.

We can modulate, and we can model what those alphabets could be and how they impact that specific target. So as I said, when you think about the gamut of technologies that we can use, and how far we've come over even the last few decades, it's pretty significant the amount of progress we've made and the confluence of technologies that can come together to aid in drug development.