The first drug Crispr in the world starts slowly

Deshawn “DJ” Chow he waited a year to receive a treatment that could change his life. The 19-year-old was born with sickle cell disease, which makes his red blood cells crescent-shaped and sticky. The misshapen cells build up 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 came more and more frequently for Chow in high school, landing him in the hospital often. 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. It is the first approved medicine to use the Nobel Prize winning technology known as Crispr, a type of gene editing. Chow received Casgevy on December 5 at the City of Hope Cancer Center in Los Angeles. He is among the first patients in the United States 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 the complexity of manufacturing, insurance delays, and the extensive preparation involved for patients, few individuals in the United States have been dosed with Casgevy since it became commercial. The slow rollout highlights the complicated nature of marketing 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 them all 34 US hospitals have approved to administer it from december Of the 26 who responded, only City of Hope National and Children’s Hospital in Washington, DC, said they had administered 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 beta thalassemia patient was treated at Children’s National. Several authorized centers told WIRED that they will begin infusions of Casgevy 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. It is a one-time therapy that involves the collection and editing of a person’s stem cells. For the patient, it means a hard round of chemotherapy before taking the cells, and a month in the hospital afterwards.