At the end of 2023 we witnessed an important moment in medical history: For the first time, the US Food and Drug Administration approved a therapy that uses Crispr gene editing. This new therapy was developed by Crispr Therapeutics and Vertex Pharmaceuticals to treat sickle cell anemia, a disease caused by a single letter mutation in the genetic code that has long been understood but has been neglected by the research community for decades.
This is an important milestone for gene editing in medicine, and in particular for the sickle cell community, which has long awaited better treatment options. The outlook for this therapy is better than we could have hoped. Victoria Gray, one of the first patients in the US to receive the therapy in a clinical trial, is symptom-free four years later. Indeed, it may turn out to be not only therapy, but also a cure.
There are more Crispr-based therapies hot on its heels, treating conditions like high cholesterol, inflammatory diseases and chronic infections. But it’s not time for a victory lap in the field of gene-editing therapies: the race is just beginning.
Let me put this in context. When my colleagues and I published how Crispr could be used for genome editing in 2012, we could hardly have imagined that just 11 years later there would be an approved therapy on the US market. In the scheme of medical research, that paper-to-patient timeline is incredibly fast. But “fast” depends on your perspective. Every week I get emails from people all over the world who hope that Crispr can help them, their children, their parents, their friends. Because Crispr can easily adapt to different regions of the genome, it gives new hope to people with rare and neglected genetic diseases. One therapy in 12 years is not fast enough if you are the one waiting.
Sickle cell therapy is projected to cost more than $2 million per patient, and only a small number of facilities in the US have the technological capacity to provide it. We see a certain cycle over and over again: The first wave of new technology that hits the market is expensive and out of reach for most people. Fifteen years ago, a smartphone was a luxury item; now 85 percent of the planet owns one. Similarly, laptops and tablets, once only for the rich, are now everywhere in the world.
But life-saving medicine cannot be treated as a luxury, and we cannot simply wait to let market forces drive down prices over time. In 2024 we’ll see more expensive first-wave therapies on the market, but researchers are already looking to the second wave: therapies designed to be affordable and accessible. New technologies enabling in vivo delivery of gene editing therapies and improved manufacturing will be key to lowering prices, as will unique partnerships between universities, government and industry, united with affordability as a common goal. It is not enough to simply make the tools. We must ensure that they reach those who need them most.
