The world’s first drug Crispr is off to a slow start

Deshon “DJ” Chow he waited a year to receive treatment that could change his life. The 19-year-old was born with sickle cell anemia, which makes his red blood cells crescent-shaped and sticky. The deformed cells accumulate and block blood vessels, cutting off oxygen to parts of the body and causing episodes of excruciating pain. The condition affects about 100,000 people in the United States, most of them black.

The pain became more frequent for Chow in high school, often landing him in the hospital. He missed school, birthday parties and sleepovers with friends. Sometimes the pain lasted for days. “It’s like my body is on fire,” he says.

A year ago, he learned of a new treatment called Casgevy that could end his years-long battle with pain. That’s it the first drug approved for using the Nobel Prize-winning technology known as Crispr, a type of gene editing. Chow received Kasgewi on Dec. 5 at City of Hope Cancer Center in Los Angeles. He is among the first patients in the US to receive the treatment from its approval in December 2023. It was also approved for beta thalassemia, a related blood disorder, this January.

Due to manufacturing complexity, insurance delays and extensive patient preparation, few people in the US have been dosed with Casgevy since it became commercially available. The slow uptake highlights the complex nature of commercializing cutting-edge medical treatments and getting them to patients. Another genetic treatment for sickle cell, Lyfgenia, won approval last December, and the first patient was treated in September. Made by Bluebird Bio, it uses an older technology that introduces a new gene to treat the disease.

Vertex Pharmaceuticals and Crispr Therapeutics, which developed Casgevy, have not publicly said how many patients have received the therapy so far. WIRED reached out to everyone 34 US hospitals have been approved to use it as of December. Of the 26 that provided responses, only City of Hope and Children’s National Hospital in Washington, D.C., said they used Casgevy. (Three hospitals declined to comment, and five others did not respond to multiple inquiries.) Chow is City of Hope’s first sickle cell patient, while a patient with beta thalassemia was treated at Children’s National Center. Several authorized centers told WIRED they will treat their first patients in early 2025.

“The process of getting this drug is very different from just taking a pill,” says Leo Wang, Chow’s hematologist-oncologist at City of Hope. This is a one-time therapy that involves harvesting and editing a person’s stem cells. For the patient, this means heavy chemotherapy before receiving the cells and a month in the hospital afterwards.