Bringing Gene Therapy to Common Diseases

Shankar Ramaswamy, MD

Thus far, adeno-associated virus (AAV) vector-based gene therapy has largely made headway in the realm of rare disease. That may be changing, however, as some companies seek to apply the modality to more common diseases that affect much larger patient populations.

One such company is Kriya Therapeutics. CGTLive® recently spoke to Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics, at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD, to learn more about the company's plans for the future.

CGTLive: Can you give some background about Kriya Therapeutics?

Shankar Ramaswamy, MD: We're a company focused on bringing one-time gene therapies to more common, highly prevalent diseases. Our mission is really to expand the reach of this modality to tackle, let's say, diseases in mainstream medicine. A lot of what we focus on is enabling the technology to ultimately achieve that goal in terms of reach, throughput of producing material, bringing down the cost, characterizing products, actually developing them in a robust way that could potentially reach millions of patients one day. A lot of what we presented at ASGCT this year was really around many of the core technologies and methodologies that we're using to engineer our products, characterize them very thoroughly with some of the next generation techniques, produce them at scale, and really support the packages that are required, ultimately, for these products to enter the clinic and go to patients.

Are there any results the company presented at ASGCT this year that you would like to highlight?

I think we had some very interesting presentations on methods for characterization of products. This is something where it's really been a pain point in the field of gene therapy historically, where what you actually put into the patient may be very different than what you put into, let's say your animal studies and what you ultimately commercialize. Continuity and characterization of product is really critical to understanding what you're actually delivering to patients, which is what matters. We presented on some of our tools and methods to understand that at a very precise level—at say under different conditions of testing—how our products actually perform. That's something that I think will start to set a new standard for where the field needs to be to achieve that ultimate goal of bringing gene therapy to the masses.

How would you summarize the key takeaways for doctors and the broader healthcare community?

Within the halls of ASGCT, this is really a forum where you have some cutting edge technologies and presentations around the latest breaking data. That's very exciting for the audience that's here, we love to hear those updates and participate in that.

But I think some of the challenges that we often see from clinicians that aren't here is understanding when those technologies will actually be ready to treat the patients that they're seeing on a daily basis. I think the message that I would sort of convey is that we are closer to that reality than we've ever been before. I think that's core to Kriya's mission and reason for existence as a company. But I think collectively as a field, we're actually on the precipice of bringing gene therapy products in rapid fashion to patients around the world that are affected by some of the most common diseases afflicting millions of individuals worldwide. So just continue to have that optimism that this is not a science fiction sort of technology, this is really on the verge of being introduced to broader mainstream audiences. We have an obligation as a gene therapy community to really educate and inform our clinical colleagues that aren't here at this meeting and are actually seeing patients on the ground.

Are there any specific areas of interest for future research for the company that you can discuss?

Kriya's pipeline, again, is really focused on gene therapies for common, highly prevalent diseases. We have 3 core therapeutic areas that we're advancing. We have gene therapies, which are one-time gene therapies, in ophthalmology, metabolic disease, and neurology. An just to double-click on that for a second, we have a program in geographic atrophy, a program in thyroid eye disease, in type 1 diabetes, in metabolic-associated steatohepatitis, and a few neurology programs on focal neurological diseases. It's a broad portfolio.

I think, as we continue to bring those programs into the clinic in the next phase of our company, we will continue to present important information updates not just at ASGCT, but also at many of the scientific and medical conferences specific to those disease areas. As an example, we had a number of presentations at ARVO in Seattle, which is a big ophthalmology meeting, just a few days ago. We are very active in all of the disease communities in which we're engaged. You can expect to see more data as we complete a lot of our our work across the different phases of development and we look forward to sharing updates with the clinical community.

Is there anything else you would like to share?

Reiterating some points that I said before, maybe said differently: We're at a pretty exciting time in the gene therapy field, there's now 6 approved products that use AAV in the United States. This is not a future science fiction type of technology. This is a real technology where thousands of patients have received AAV either through commercial products or in clinical trials, and so the technology has really matured to a point where we can take that next leap as a field to bring gene therapies into a broader universe of diseases. I think we as a field need to figure out how we can scale the technology, how we can bring down the cost, and how we can improve throughput and quality of the products that we engineer and manufacture. But those are solvable challenges. There are companies like us that are working on solving those challenges. I think in the next decade, you're gonna see gene therapies sweep through a number of different areas of medicine, much like we saw with monoclonal antibodies back in the 90s. So I'm very excited to be a part of that and I think the broader clinical community should be aware of that potential really on the horizon.

This transcript has been edited for clarity.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.