The world’s first CRISPR drug gets a slow start

Deshaun “DJ” Chow He waited a year to receive the treatment that would change his life. The 19-year-old was born with sickle cell disease, 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 bouts of excruciating pain. This condition affects about 100,000 people in the United States, most of whom are black.

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

A year ago, he discovered a new treatment called Casgevy that could end his long battle with pain. that it Drug approved for the first time To use Nobel Prize winning technology known as CRISPR, a type of gene editing. Chow received Casgevy on December 5 at City of Hope Cancer Center in Los Angeles. He is among the first patients in the United States to receive the treatment Since its approval in December 2023. It was also approved to treat beta thalassemia, a related blood disorder, in January this year.

Because of manufacturing complexities, delays in insurance, and extensive patient preparation, few individuals in the United States have been dosed with Casgevy since it became commercially available. The slow rollout highlights the complex nature of marketing cutting-edge medical treatments and getting them to patients. Another gene therapy for sickle cell disease, Livigenia, received approval last December, and the first patient was treated in September. The drug, made by Bluebird Bio, uses an old technology that introduces a new gene to treat the disease.

Vertex Pharmaceuticals and Crispr Therapeutics, which developed Casgevy, have not publicly announced how many patients have received the treatment so far. WIRED has reached everyone 34 US hospitals are approved to administer it As of December. Of the 26 hospitals that provided answers, only City of Hope Hospital and Children’s National Hospital in Washington, D.C., said they had administered Kasgevi. (Three hospitals declined to comment, and five others did not respond to multiple inquiries.) Chow is the first patient with sickle cell anemia at City of Hope, while a patient with beta thalassemia was treated at National Children’s Hospital. Several approved centers told WIRED they will begin infusing Casgevy in early 2025.

“The process of getting this medication is very different from just taking a pill,” says Liu Wang, a hematologist and oncologist who treats Zhao at City of Hope. It is a one-time treatment that involves collecting a person’s stem cells and editing them. For the patient, this means a tough round of chemotherapy before getting the cells, and a month in the hospital afterward.