Casgevy / Exa-cel
The first FDA-approved therapy using CRISPR/Cas9, editing autologous hematopoietic stem cells for sickle cell disease and beta thalassemia.
Core metadata
- ID: casgevy_exa_cel
- Era: Modern
- First known date: 2023 (exact)
- Region: United Kingdom and United States
- Review status: source_checked
- Maturity: approved
Prerequisites
- CRISPR Off-Target Profiling (crispr_off_target_profiling)
- Ex Vivo CRISPR Cell Therapy (ex_vivo_crispr_cell_therapy)
- Human Genome Project (DNA Mapping) (human_genome_project_dna_mapping)
Dependents
- None.
Fields
Field lanes
- Genome Editing / CRISPR-Cas: Therapeutics
Node sources
- FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease (U.S. Food and Drug Administration, 2023, official_agency) • Supports: node, maturity
- CASGEVY (U.S. Food and Drug Administration, 2023, official_agency) • Supports: node, maturity
Prerequisite edge evidence
Edge/source evidence summary:
- Prerequisite edges: 3
- Average edge confidence: 68%
- Prerequisite sources: 3
- expert_inference: 3
| Prerequisite | Type | Confidence | Evidence level | Note | Sources |
|---|---|---|---|---|---|
| Ex Vivo CRISPR Cell Therapy (ex_vivo_crispr_cell_therapy) | enabling | 68% | expert_inference | Ex Vivo CRISPR Cell Therapy provides a capability that enables this technology without being the only possible path. |
|
| CRISPR Off-Target Profiling (crispr_off_target_profiling) | enabling | 68% | expert_inference | CRISPR Off-Target Profiling provides a capability that enables this technology without being the only possible path. |
|
| Human Genome Project (DNA Mapping) (human_genome_project_dna_mapping) | enabling | 68% | expert_inference | Human Genome Project (DNA Mapping) provides a capability that enables this technology without being the only possible path. |
|
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