In 2019 Nature published a landmark paper introducing a new gene editing tool that has the potential to correct nearly all disease-causing DNA. The work of Dr. David Liu and Dr. Andrew Anzalone and their team, this new generation of genetic editing, known as prime editing, has the potential to change medicine as we know it today. Imagine a world where we could cure, halt, or prevent most genetic diseases.
Dr. Liu describes prime editing as a word processor which can insert, delete, and replace multiple bases without cutting the DNA. Unlike earlier iterations of gene editing, which function like genetic scissors, as in the case of CRIPSR, or base editing, which acts like a pencil to erase and replace single amino acids in the DNA, prime editing can "search and replace" to restore normal genetic function almost anywhere in the genome. In principle, prime editing can correct up to 89% of known genetic variants associated with human diseases.
Prime Medicine, a company founded in 2019 by Dr. Liu to advance prime editing into therapeutic applications, launches today. I've been closely involved with the company since inception, and helped recruit Keith Gottesdiener to lead the team as CEO. In this video, Keith and I discuss the potential of prime editing to change the way we treat genetic disorders, and Prime Medicine's mission to bring this transformational technology to patients.
"When you look at what prime editing can do, in many ways it seems to me to be limitless," says Keith. Prime editing makes all types of DNA corrections, at the site of mutation, resulting in a permanent change, leaving the gene under native control. It edits with high fidelity and specificity; with minimal off-target editing elsewhere in the genome. And it works on all types of cells: rapidly dividing and quiescent; mammalian and non-mammalian, and on a wide range of organs.
Prime editing can address the fundamental cause of disease and potentially provide patients a long-lasting cure. Keith and his team of physician-scientists are driven by their motivation to help people with genetic diseases. "The whole purpose is to make a difference to patients," he says. "We feel this goal is an overpowering one, and we have to move quickly. Still, we know there are challenges ahead, and it will be years before this can be tested in the clinic."