At the end of 2023, we witnessed an important moment in the history of medicine: 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 disease, an ailment caused by a single-letter mutation in the genetic code that has been long understood but was neglected by the research community for decades.
This is a major milestone for gene editing in medicine, and specifically for the sickle cell community, who have 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, this may prove to be not just a therapy but a cure.
There are further Crispr-based therapies coming close on its heels, treating conditions such as high cholesterol, inflammatory disease, and chronic infections. But it’s not time for a victory lap for the field of...