By Healthcare Brew Staff
less than 3 min read
Definition:
Gene therapy is a process that modifies a patient’s genes to treat or cure diseases, according to the FDA.
It can be used to:
- Swap a disease-causing gene with a healthy copy.
- Remove a disease-causing gene that isn’t performing properly.
- Add a new or modified gene into the body to treat a disease.
What is gene editing?
Gene editing is a type of technology that allows scientists to make changes to patients’ physical features, such as eye color, as well as address disease risk. The technology acts like a pair of scissors, cutting the DNA in a specific spot and removing or replacing the DNA.
One of the most notable gene editing tools is CRISPR, which was first publicized in a 2012 study that focused on the clustered regularly interspersed short palindromic repeats (CRISPRs—and it essentially started a genetic revolution.
In 2023, the FDA approved the first CRISPR-based medicine, Casgevy, to treat sickle cell anemia. There are other CRISPR-based treatment trials in progress for illnesses like Hepatitis B, lymphoma, and urinary tract infections.
What risks do gene therapy pose?
Gene therapy can be a pricey endeavor, with the Institute for Clinical and Economic Review estimating the average cost to be between $1 million and $2 million per dose. This raises a health equity issue in regards to who can access these sometimes life-saving therapies.
In March 2025, a Duchenne muscular dystrophy patient died after taking Sarepta Therapeutics’s Elevidys gene therapy medication. And though the community continued to express support for the treatment, a second patient died in June 2025, leading to an FDA investigation. There were initial concerns about the treatment’s accelerated FDA approval, but the director of the FDA’s Center for Biologics Evaluation and Research overruled them.
