The World’s First Crispr Drug Gets a Slow Start

Deshaun “DJ” Chaw 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 different parts of the body and causing excruciating pain episodes. The condition affects about 100,000 people in the United States, most of whom are black.

Pain came more frequently for Chou in high school, landing him in the hospital more often. He missed school, birthday parties and sleepovers with friends. Sometimes, the pain lasts for days. “It was like my body was on fire,” she says.

A year ago, he learned about a new treatment called Casgavy that could end his years-long battle with pain. it is First approved drug Known for using Nobel Prize winning technology Crispr, a type of gene editing. Chow found Casgavy on Dec. 5 at City of Hope Cancer Center in Los Angeles. He was among the first patients to receive the treatment in the United States Since its approval in December 2023. It was approved for beta thalassemia, a related blood disorder, this January.

Because of manufacturing complications, insurance delays, and extensive preparation for patients, few people in the United States have dosed with Casgavi since it became commercially available. The slow rollout underscores the complex nature of commercializing cutting-edge medical treatments and delivering them to patients. Another genetic treatment for sickle cell, Lifgenia, won approval last December and the first patient was treated in September. Developed by Bluebird Bio, it uses an old technology that introduces a new gene to treat a disease.

Vertex Pharmaceuticals and Crispr Therapeutics, which makes Casgavi, have not publicly disclosed how many patients have received the therapy so far. WIRED reaches out to everyone 34 US hospitals are approved to administer it As of December. Of the 26 that responded, only City of Hope and Children’s National Hospital in Washington, DC, said they administered CasGavi. (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. Several approved centers told Wired they will begin infusing casgavir as early as 2025.

“The process of getting this drug is very different from just taking a pill,” says Chow’s hematologist-oncologist Leo Wang at City of Hope. “It’s a one-time therapy that harvests and edits a person’s stem cells. For the patient, that means chemotherapy before receiving the cells.” A tough round and a month in the hospital later.