The World’s First Crispr Drug Gets a Slow Start

Deshawn “DJ” Week He waited a year to receive treatment that could have changed his life. The 19-year-old was born with sickle cell disease, which leaves his red blood cells crescent-shaped and sticky. Malformed cells can accumulate and block blood vessels, cutting off oxygen to parts of the body and causing severe pain. The condition affects about 100,000 people in the United States, the majority of whom are black.

During high school, Mr. Zhou’s pain became more frequent, often landing him in the hospital. He missed school, birthday parties and sleepovers with friends. Sometimes the pain lasts for several days. “My body was on fire,” he said.

A year ago, he discovered a new treatment called Casgevy that could end his years-long battle with pain. This is first approved drug Using Nobel Prize-winning technology Crispr, a type of gene editing. Chow received Casgevy on Dec. 5 at City of Hope Cancer Center in Los Angeles. He was one of the first patients to receive treatment in the U.S. Since approval in December 2023. In January this year, it was also approved to treat beta thalassemia, a related blood disorder.

Few people in the U.S. have taken Casgevy since it became available, due to manufacturing complexities, insurance delays and the amount of preparation patients need to do. The slow rollout highlights the complexities of commercializing cutting-edge medical treatments and getting them to patients. Another gene therapy for sickle cell, Lyfgenia, was approved in December, and the first patient was treated in September. Made by Bluebird Bio, it uses an older technology and 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 lends a helping hand to everyone 34 hospitals in the United States have been approved to use the drug As of December. Of the 26 hospitals that provided answers, only City of Hope and Children’s National Hospital in Washington, D.C., said they had used Casgevy. (Three hospitals declined to comment, and five others did not respond to multiple inquiries.) Zhou is City of Hope’s first sickle cell patient, and a beta thalassemia patient has been treated at Children’s National. Multiple authorized centers told Wired they will treat their first patients in early 2025.

“The process of getting this drug is very different than just taking a pill,” said Leo Wang, a hematology oncologist at Chow at City of Hope. It’s a one-time treatment that involves collecting and editing a person’s stem cells. For patients, this means a harsh round of chemotherapy before the cells can be harvested, followed by a month in the hospital.