On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy™ (exagamglogene autotemcel), the first-ever CRISPR-Cas9 gene-editing therapy for human use. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy treats severe sickle cell disease (SCD) in patients aged 12+.
Key Details: How it works: Patients’ stem cells are edited outside the body using CRISPR-Cas9 to reactivate fetal hemoglobin (which counteracts sickle-shaped red blood cells). Edited cells are then infused back.
Efficacy: In clinical trials, 97% of patients (29/30) avoided severe pain crises for ≥12 months post-treatment.
Significance: A functional cure for a genetic disorder affecting 100,000+ Americans (mostly of African descent).
Cost: $2.2 million per patient (though insurers may cover it due to long-term savings from avoided hospitalizations).
Challenges: Requires intensive chemotherapy and weeks of hospitalization.
Limited to specialized medical centers initially.
Scientists Editing Cells
(Credit: Science Magazine)
Sources & Further Reading:
FDA Official Announcement (Dec 8, 2023):
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
Clinical Trial Data (New England Journal of Medicine):
CRISPR-Cas9 Editing for Sickle Cell Disease
Global Impact Analysis (Nature, Dec 2023):
CRISPR Gene Editing Earns First Medical Approval
Patient Experience (NPR):
"I Feel Cured": Sickle Cell Patient’s Journey
Key Quote
"This is a landmark moment. CRISPR has moved from a lab tool to a life-saving treatment."
— Dr. Alexis Thompson, Hematologist, Children’s Hospital of Philadelphia
Current Status (2024): UK/EU Approval: Casgevy was also approved in the UK (Nov 2023) and EU (Jan 2024).
Expanding Access: Trials underway for beta-thalassemia and other genetic disorders.
This therapy represents the first real-world application of CRISPR to cure genetic disease, ending decades of suffering for sickle cell patients. All sources cited are from major scientific journals, regulatory bodies, and reputable news outlets
Key Details: How it works: Patients’ stem cells are edited outside the body using CRISPR-Cas9 to reactivate fetal hemoglobin (which counteracts sickle-shaped red blood cells). Edited cells are then infused back.
Efficacy: In clinical trials, 97% of patients (29/30) avoided severe pain crises for ≥12 months post-treatment.
Significance: A functional cure for a genetic disorder affecting 100,000+ Americans (mostly of African descent).
Cost: $2.2 million per patient (though insurers may cover it due to long-term savings from avoided hospitalizations).
Challenges: Requires intensive chemotherapy and weeks of hospitalization.
Limited to specialized medical centers initially.
Scientists Editing Cells
(Credit: Science Magazine)
Sources & Further Reading:
FDA Official Announcement (Dec 8, 2023):
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
Clinical Trial Data (New England Journal of Medicine):
CRISPR-Cas9 Editing for Sickle Cell Disease
Global Impact Analysis (Nature, Dec 2023):
CRISPR Gene Editing Earns First Medical Approval
Patient Experience (NPR):
"I Feel Cured": Sickle Cell Patient’s Journey
Key Quote
"This is a landmark moment. CRISPR has moved from a lab tool to a life-saving treatment."
— Dr. Alexis Thompson, Hematologist, Children’s Hospital of Philadelphia
Current Status (2024): UK/EU Approval: Casgevy was also approved in the UK (Nov 2023) and EU (Jan 2024).
Expanding Access: Trials underway for beta-thalassemia and other genetic disorders.
This therapy represents the first real-world application of CRISPR to cure genetic disease, ending decades of suffering for sickle cell patients. All sources cited are from major scientific journals, regulatory bodies, and reputable news outlets