Aditya Raguram

Early on as a PhD student at Harvard, Aditya Raguram, 27, helped develop two groundbreaking tools based on the genetic engineering technique CRISPR that both have the potential to treat a wide range of genetic disorders. But these tools—made of large proteins that can erase and rewrite segments of DNA—are hard to deliver safely into cells of the body. Modified viruses, a common delivery mechanism for vaccines and other therapeutics, aren’t great couriers of proteins. And packaging these tools as DNA or mRNA, which would then synthesize the proteins once inside the cells, comes with risks that edits could be made in undesired places.

Prior to completing his PhD, Raguram devised a fix. Working with Samagya Banskota (one of our 35 Innovators in 2022), he developed a new type of engineered virus-like particles that not only managed to handle bulky protein cargo, but could release it at just the right moment when in contact with a target cell. The team used these particles to deliver gene-editing therapies to mice, including one that corrected a mutation causing blindness; it was able to partially restore vision.

Today, practitioners are beginning to use CRISPR-based techniques to treat disorders in humans by editing stem cells in a lab and then transplanting them back into the body. Raguram’s innovation marks a major step toward using gene editing to make fixes to the body directly—perhaps with a single injection.