3 completed, 2 not yet recruiting. 5 Phase 2, 3 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 3, Not yet recruiting 2 |
| Phase 1 | 3 | Not yet recruiting 2, Completed 1 |
| Phase 3 | 2 | Completed 2 |
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Safety and Efficacy Study Evaluating CTX112 in Subjects With Refractory Neurologic Autoimmune Disease CTX112 - Progressive Multiple Sclerosis (PMS), Neuromyelitis Optica | Phase 1 / Phase 2 | Not yet recruiting | CRISPR Therapeutics | 220 | 2026-11-01 | 2026-10-22 |
| A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD) CTX460 - Alpha-1 Antitrypsin Deficiency (AATD) | Phase 1 / Phase 2 | Not yet recruiting | CRISPR Therapeutics | 68 | 2026-10-31 | 2026-09-30 |
| A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed/Refractory Hematologic Malignancies CTX131 - T Cell Lymphoma, B Cell Lymphoma | Phase 1 / Phase 2 | Completed | CRISPR Therapeutics | 12 | 2024-08-13 | 2026-04-03 |
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease Exa-cel - Sickle Cell Disease, Hematological Diseases | Phase 2 / Phase 3 | Completed | Vertex Pharmaceuticals Incorporated | 63 | 2018-11-27 | 2026-09-16 |
| A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia CTX001 - Beta-Thalassemia, Thalassemia | Phase 2 / Phase 3 | Completed | Vertex Pharmaceuticals Incorporated | 59 | 2018-09-14 | 2025-12-17 |
Hematologic Diseases (2) · Hemoglobinopathies (2) · Anemia, Sickle Cell (1) · Autoimmune Diseases of the Nervous System (1) · Genetic Diseases, Inborn (1) · Leukemia (1) · Leukemia, Myeloid, Acute (1) · Lymphoma, B-Cell (1) · Lymphoma, T-Cell (1) · Multiple Sclerosis, Chronic Progressive (1) · Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (1) · Neuromyelitis Optica (1) · Stiff-Person Syndrome (1) · Thalassemia (1) · alpha 1-Antitrypsin Deficiency (1)
United States (3) · Canada (2) · Germany (2) · Italy (2) · United Kingdom (2) · Australia (1) · Belgium (1) · France (1)
PMID 33283989, PMID 34175041, PMID 32873924, PMID 36123234, PMID 36860093, PMID 36975201, PMID 38657265, PMID 38661449, PMID 38866240, PMID 40857358, PMID 40862696, PMID 42252696