Shankar Ramaswamy, MD, on Bringing Gene Therapy to Common Diseases

“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.”

Gene therapy, particularly adeno-associated virus vector-based gene therapy, first gained its foothold in the field of medicine by targeting relatively rare inherited disorders like Duchenne muscular dystrophy and hemophilia A, both of which are now among the indications for the 6 FDA-approved AAV vector-based gene therapies. Indeed, this trend has continued, with many companies and academic institutions continuing to pursue the development of AAV vector-based gene therapies for rare and even ultrarare genetic disorders. Because of the high cost and lengthy and complex manufacturing process for gene therapy products, targeting smaller patient populations for both clinical trials and commercial products is in some ways a good fit for the modality. That said, gene therapy may have the potential to treat much more common diseases, as well. One company currently seeking to bring AAV vector-based gene therapy to common diseases is Kriya Therapeutics. Kriya gave 6 presentations regarding their work on this endeavor at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.

At the conference, CGTLive® sat down with Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics, to learn more about the company’s efforts. Ramaswamy explained that gene therapy has significant potential to address unmet needs for relatively common diseases like type 1 diabetes and geographic atrophy, but that bringing gene therapy to such indications will require overcoming challenges such as scaling manufacturing, reducing costs, and characterizing products. He noted that Kriya is currently working on technologies and methods to address these concerns, and gave his view that gene therapy is on the precipice of reaching much broader use in clinical practice.

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