5-year long-term follow-up data for CASGEVY in sickle cell disease at the ASH 2026 annual meeting. CASGEVY is the first FDA-approved CRISPR-edited therapy. Watch: durability of VOC-free response, fetal hemoglobin levels, late safety events.
What’s at stake
CASGEVY (exagamglogene autotemcel) is an ex vivo gene therapy co-developed by CRISPR Therapeutics and Vertex Pharmaceuticals that uses CRISPR/Cas9 to edit patients' own blood stem cells, providing a functional cure for sickle cell disease and transfusion-dependent beta thalassemia. The therapy harvests a patient's stem cells, disables the BCL11A enhancer — a genetic switch that keeps protective fetal hemoglobin silenced after birth — then reinfuses the edited cells after conditioning chemotherapy, reactivating fetal hemoglobin to compensate for defective adult hemoglobin. CASGEVY is the first FDA-approved CRISPR-based medicine and the first curative option for these hemoglobin disorders outside of allogeneic stem cell transplant.
No primer in glossary yet.
The major heme/onc meeting (~30,000 attendees). Early-December; key venue for blood cancer and rare hematology readouts.
- ASHconferenceAmerican Society of Hematology Annual Meeting
The major heme/onc meeting (~30,000 attendees). Early-December; key venue for blood cancer and rare hematology readouts.
Competitive landscape
| Drug | Company | Modality | Mechanism | Phase | Next catalyst |
|---|---|---|---|---|---|
| reni-cel | EDIT | cell therapy | CRISPR/Cas12a HBG1/2 promoter edit | CONFERENCE · May 26 | |
| BEAM-101 | BEAM | cell therapy | base editing of HBG1/2 to reactivate fetal hemoglobin | READOUT · Dec 26 | |
| CB-010 | CRBU | cell therapy | allogeneic CRISPR-edited anti-CD19 CAR-T | CONFERENCE · May 26 | |
| JAKAFIruxolitinib | INCY | small molecule | JAK1/JAK2 inhibitor | READOUT · Sep 26 |
Disclosure trail
- Apr 1, 2026·29d agopinned · highest confidenceHIGH confconferenceWINDOWtop claimDEC–DEC2026
“CRISPR Therapeutics will present 5-year follow-up data from the CLIMB-SCD long-term study of CASGEVY at the 68th ASH Annual Meeting (December 5–8, 2026, Atlanta).”
conf 92%via llm