Clinical Pipeline Overview
36 active trials across 5 companies, by phase
Trials by phase
Phase 4 (2)
| Company | Status | Indication | Expected |
|---|---|---|---|
| VRTX | ENROLLING_BY_INVITATION | Pain, Acute Postoperative, Breast Disease | 2027-12-28 |
| VRTX | NOT_YET_RECRUITING | Knee Arthritis, Knee Osteoarthritis | 2028-08 |
Phase 3 (11)
| Company | Status | Indication | Expected |
|---|---|---|---|
| NTLA | ACTIVE_NOT_RECRUITING | Hereditary Angioedema | 2026-02-10 |
| CRSP | ACTIVE_NOT_RECRUITING | Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance | 2027-06-06 |
| VRTX | RECRUITING | Beta-Thalassemia, Thalassemia, Hematologic Diseases | 2027-06-09 |
| VRTX | RECRUITING | Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia | 2027-06-30 |
| NTLA | RECRUITING | Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease | 2027-07 |
| VRTX | ACTIVE_NOT_RECRUITING | Cystic Fibrosis | 2027-09-30 |
| CRSP | ACTIVE_NOT_RECRUITING | Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn | 2027-11-14 |
| NTLA | RECRUITING | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 2027-12 |
| VRTX | ENROLLING_BY_INVITATION | Cystic Fibrosis | 2029-07-30 |
| VRTX | ACTIVE_NOT_RECRUITING | Cystic Fibrosis | 2030-06-30 |
| CRSP | ENROLLING_BY_INVITATION | Beta-Thalassemia, Thalassemia, Sickle Cell Disease | 2039-09-30 |
Phase 2/3 (2)
| Company | Status | Indication | Expected |
|---|---|---|---|
| VRTX | RECRUITING | Proteinuric Kidney Disease | 2028-06-02 |
| VRTX | RECRUITING | Primary Membranous Nephropathy | 2028-12-29 |
Phase 2 (3)
| Company | Status | Indication | Expected |
|---|---|---|---|
| VRTX | RECRUITING | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 2027-07-22 |
| VRTX | ENROLLING_BY_INVITATION | Myotonic Dystrophy Type 1 (DM1) | 2029-01-30 |
| VRTX | RECRUITING | Myasthenia Gravis, Generalized | 2029-03-02 |
Phase 1/2 (9)
| Company | Status | Indication | Expected |
|---|---|---|---|
| NTLA | ACTIVE_NOT_RECRUITING | Hereditary Angioedema | 2024-04-04 |
| MRNA | ACTIVE_NOT_RECRUITING | Cystic Fibrosis | 2026-04-21 |
| BEAM | ACTIVE_NOT_RECRUITING | Lymphoblastic Lymphoma, T-Cell Lymphoblastic Leukemia/Lymphoma, Lymphoblastic Leukemia | 2026-12 |
| BEAM | RECRUITING | Glycogen Storage Disease Type Ia | 2027-12-30 |
| BEAM | ACTIVE_NOT_RECRUITING | Sickle Cell Disease | 2028-02 |
| BEAM | RECRUITING | Alpha 1-Antitrypsin Deficiency | 2028-05 |
| CRSP | RECRUITING | B-cell Lymphoma, Non-Hodgkin Lymphoma, B-cell Malignancy | 2030-01 |
| CRSP | RECRUITING | Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia | 2033-12 |
| NTLA | RECRUITING | Hemophilia B | 2034-08-14 |
Phase 1 (4)
| Company | Status | Indication | Expected |
|---|---|---|---|
| BEAM | RECRUITING | Healthy Subjects, Healthy Participant Study | 2026-01-29 |
| CRSP | RECRUITING | Cardiovascular, Metabolic Disease, Dyslipidemias | 2027-06 |
| BEAM | RECRUITING | T-Cell Acute Lymphoblastic Leukemia/Lymphoma | 2029-05-30 |
| CRSP | RECRUITING | SLE (Systemic Lupus), Lupus Erythematosus, Systemic, Lupus Nephritis | 2031-12-31 |
Unspecified (5)
| Company | Status | Indication | Expected |
|---|---|---|---|
| BEAM | RECRUITING | Sickle Cell Trait | 2026-12 |
| NTLA | RECRUITING | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis | 2038-03 |
| CRSP | ENROLLING_BY_INVITATION | Hematologic Malignancy, Solid Malignancy | 2038-08 |
| NTLA | ENROLLING_BY_INVITATION | Hereditary Angioedema | 2040-04 |
| BEAM | RECRUITING | Hemoglobinopathy, Sickle Cell Disease | 2043-02-12 |