World’s First Crispr Drug Launches Slowly

Deshawn “DJ” Chow he waited a year to receive treatment that would change his life. The 19-year-old was born with sickle cell disease, which causes his red blood cells to become sticky and sticky. The misshapen cells build up and block blood vessels, cutting off oxygen to the body’s organs and causing excruciating pain. The disease affects about 100,000 people in the United States, most of whom are black.

The pain recurred for Chow in high school, landing him in the hospital many times. He missed school, birthday parties, and sleepovers with his friends. Sometimes the pain would last for days. He said: “It’s like my body is on fire.”

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

Due to manufacturing difficulties, insurance delays, and the extensive preparation involved for patients, few people in the US have taken Casgevy since it became commercially available. Slow release proves the challenge of marketing modern drugs and getting them to patients. Another sickle cell gene therapy, Lyfgenia, was approved last December, and the first patient was treated in September. Developed by Bluebird Bio, it uses an ancient 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 treatment so far. WIRED reached out to them all 34 US hospitals have agreed to offer it since December. Of the 26 that responded, City of Hope and Children’s National Hospital in Washington, DC, were the ones that offered Casgevy’s treatment. (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 beta thalassemia patient has been treated at Children’s National. Multiple sources told WIRED that Casgevy’s infusions will begin as early as 2025.

“The way to get this medicine is very different from just taking pills,” says Leo Wang, Chow’s hematologist at City of Hope. It is a one-time treatment that involves collecting and processing human cells. For the patient, it means a grueling round of chemotherapy before getting the cells, and a month in the hospital afterwards.