Pipeline

All active studies we track, grouped by phase. Each row links to the study record on ClinicalTrials.gov.

Phase 314

Phase 219

Phase 14

Phase 44

Not applicable6

Phase 3

Large studies that confirm whether a treatment works, usually the last step before filing for approval.

Primary completion	Company	Phase	Status	Condition and study
10 Feb 2026 passed	Intellia Therapeutics	Phase 3?	Active, not recruiting	Hereditary Angioedema HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) · NCT06634420
6 Jun 2027	CRISPR Therapeutics	Phase 3?	Active, not recruiting	Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) · NCT05329649
9 Jun 2027	Vertex Pharmaceuticals	Phase 3?	Recruiting	Beta-Thalassemia, Thalassemia, Hematologic Diseases Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease · NCT05477563
Jul 2027	Intellia Therapeutics	Phase 3?	Recruiting	Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease A Phase 3 Study of NTLA-2001 in ATTRv-PN · NCT06672237
30 Sep 2027	Vertex Pharmaceuticals	Phase 3?	Active, not recruiting	Cystic Fibrosis Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older · NCT06460506
14 Nov 2027	CRISPR Therapeutics	Phase 3?	Active, not recruiting	Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) · NCT05356195
Dec 2027	Intellia Therapeutics	Phase 3?	Recruiting	Transthyretin Amyloidosis (ATTR) With Cardiomyopathy MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) · NCT06128629
31 Dec 2027	Vertex Pharmaceuticals	Phase 3?	Recruiting	Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes · NCT04786262
2 Jun 2028	Vertex Pharmaceuticals	Phase 2/3?	Recruiting	Proteinuric Kidney Disease Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease · NCT05312879
29 Dec 2028	Vertex Pharmaceuticals	Phase 2/3?	Recruiting	Primary Membranous Nephropathy Evaluation of Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN) · NCT07204275
30 Jul 2029	Vertex Pharmaceuticals	Phase 3?	Enrolling by invitation	Cystic Fibrosis Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older · NCT05844449
28 Dec 2029	Vertex Pharmaceuticals	Phase 3?	Enrolling by invitation	Immunoglobulin A Nephropathy A Phase 3 Open-Label Study of Long-term Treatment With Povetacicept in Adults With Immunoglobulin A Nephropathy · NCT07807995
30 Jun 2030	Vertex Pharmaceuticals	Phase 3?	Active, not recruiting	Cystic Fibrosis Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age · NCT05422222
30 Sep 2039	CRISPR Therapeutics	Phase 3?	Enrolling by invitation	Beta-Thalassemia, Thalassemia, Sickle Cell Disease A Long-term Follow-up Study in Participants Who Received CTX001 · NCT04208529

Phase 2

Studies that look for signs that the treatment works and at which dose, in a few hundred patients.

Primary completion	Company	Phase	Status	Condition and study
4 Apr 2024 passed	Intellia Therapeutics	Phase 1/2?	Active, not recruiting	Hereditary Angioedema NTLA-2002 in Adults With Hereditary Angioedema (HAE) · NCT05120830
Dec 2024 passed	Circio Holding ASA	Phase 2?	Active, not recruiting	Pancreas Cancer Tg01 Vaccine / Qs-21 Stimulon™ With Or Without Balstilimab As Maintenance Therapy Following Adjuvant Chemotherapy In Patients With Resected Pancreatic Cancer · NCT05638698
21 Apr 2026 passed	Moderna	Phase 1/2?	Active, not recruiting	Cystic Fibrosis A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) · NCT05668741
Dec 2026	Beam Therapeutics	Phase 1/2?	Active, not recruiting	Lymphoblastic Lymphoma, T-Cell Lymphoblastic Leukemia/Lymphoma, Lymphoblastic Leukemia A Study Evaluating the Safety and Efficacy of BEAM-201 in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL) · NCT05885464
19 May 2027	Circio Holding ASA	Phase 1/2?	Recruiting	Multiple Myeloma, Smoldering Multiple Myeloma The TG01 Study With TG01/QS-21 Vaccine in Patients With High-risk Smouldering Multiple Myeloma and Multiple Myeloma · NCT05841550
Jun 2027	Circio Holding ASA	Phase 2?	Active, not recruiting	Pancreatic Ductal Adenocarcinoma, Refractory Non-Small Cell Lung Cancer Anti-CD38 Antibody With KRAS Vaccine and Anti-PD-1 Antibody in Subjects With Pancreatic Ductal Adenocarcinoma and Refractory Non-Small Cell Lung Cancer · NCT06015724
22 Jul 2027	Vertex Pharmaceuticals	Phase 2?	Active, not recruiting	Autosomal Dominant Polycystic Kidney Disease (ADPKD) Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW) · NCT07161037
30 Dec 2027	Beam Therapeutics	Phase 1/2?	Recruiting	Glycogen Storage Disease Type Ia A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) · NCT06735755
Feb 2028	Beam Therapeutics	Phase 1/2?	Active, not recruiting	Sickle Cell Disease BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease · NCT05456880
May 2028	Beam Therapeutics	Phase 1/2?	Recruiting	Alpha 1-Antitrypsin Deficiency A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) · NCT06389877
1 Jul 2028	CRISPR Therapeutics	Phase 1/2?	Recruiting	Hypertension A Trial Evaluating Safety, Tolerability and Efficacy of CTX340 in Participants With Hypertension · NCT07758634
30 Jan 2029	Vertex Pharmaceuticals	Phase 2?	Enrolling by invitation	Myotonic Dystrophy Type 1 (DM1) A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I · NCT06926621
2 Feb 2029	Cereno Scientific AB	Phase 2?	Not yet recruiting	Pulmonary Arterial Hypertension CS1 Added to Standard of Care Therapy for Pulmonary Arterial Hypertension · NCT07761572
2 Mar 2029	Vertex Pharmaceuticals	Phase 2?	Recruiting	Myasthenia Gravis, Generalized A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis · NCT07501702
30 Dec 2029	CRISPR Therapeutics	Phase 1/2?	Not yet recruiting	Progressive Multiple Sclerosis (PMS), Neuromyelitis Optica, Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease A Safety and Efficacy Study Evaluating CTX112 in Subjects With Refractory Neurologic Autoimmune Disease · NCT07804004
Jan 2030	CRISPR Therapeutics	Phase 1/2?	Recruiting	B-cell Lymphoma, Non-Hodgkin Lymphoma, B-cell Malignancy A Safety and Efficacy Study Evaluating CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies · NCT05643742
30 Jun 2030	CRISPR Therapeutics	Phase 1/2?	Not yet recruiting	Alpha-1 Antitrypsin Deficiency (AATD) A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD) · NCT07850076
Dec 2033	CRISPR Therapeutics	Phase 1/2?	Recruiting	Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease · NCT07549698
14 Aug 2034	Intellia Therapeutics	Phase 1/2?	Recruiting	Hemophilia B A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B · NCT06379789

Phase 1

First studies in people, mainly about safety and dosing.

Primary completion	Company	Phase	Status	Condition and study
29 Jan 2026 passed	Beam Therapeutics	Phase 1?	Recruiting	Healthy Subjects, Healthy Participant Study This is a Phase 1, Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of BEAM-103 in Healthy Subjects · NCT07304791
Jun 2027	CRISPR Therapeutics	Phase 1?	Recruiting	Cardiovascular, Metabolic Disease, Dyslipidemias A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias · NCT07491172
30 May 2029	Beam Therapeutics	Phase 1?	Recruiting	T-Cell Acute Lymphoblastic Leukemia/Lymphoma Anti-CD7 CAR-T Cells in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia or Lymphoma · NCT06934382
31 Dec 2031	CRISPR Therapeutics	Phase 1?	Recruiting	SLE (Systemic Lupus), Lupus Erythematosus, Systemic, Lupus Nephritis A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease · NCT06925542

Phase 4

Studies after approval.

Primary completion	Company	Phase	Status	Condition and study
1 Oct 2027	Vertex Pharmaceuticals	Phase 4?	Not yet recruiting	Ankle Osteoarthritis, Postoperative Pain, Acute Pain Suzetrigine for Postoperative Pain After Total Ankle Replacement · NCT07795827
28 Dec 2027	Vertex Pharmaceuticals	Phase 4?	Enrolling by invitation	Pain, Acute Postoperative, Breast Disease Suzetrigine for Non-Mastectomy Breast Surgery · NCT07463430
Jan 2028	Vertex Pharmaceuticals	Phase 4?	Not yet recruiting	Postoperative Pain Suzetrigine Added to Multimodal Analgesia for Women Undergoing Minimally Invasive Hysterectomy · NCT07836010
Aug 2028	Vertex Pharmaceuticals	Phase 4?	Not yet recruiting	Knee Arthritis, Knee Osteoarthritis Role of Suzetrigine as a Part of a Multimodal Regimen to Reduce Pain and Opioid Use After Total Knee Arthroplasty · NCT07624526

Not applicable

Observational or other studies where ClinicalTrials.gov does not assign a phase.

Primary completion	Company	Phase	Status	Condition and study
Dec 2026	Beam Therapeutics	N/A	Recruiting	Sickle Cell Trait Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT) · NCT06071377
Dec 2032	Vertex Pharmaceuticals	N/A	Recruiting	DM1, Myotonic Dystrophy, Myotonic Dystrophy 1 Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension · NCT07700225
Mar 2038	Intellia Therapeutics	N/A	Enrolling by invitation	Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001 · NCT05697861
Aug 2038	CRISPR Therapeutics	N/A	Enrolling by invitation	Hematologic Malignancy, Solid Malignancy A Long-term Follow-up Study of Subjects Who Received CRISPR CAR T Cellular Therapies · NCT06208878
Apr 2040	Intellia Therapeutics	N/A	Enrolling by invitation	Hereditary Angioedema Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002 · NCT06262399
12 Feb 2043	Beam Therapeutics	N/A	Recruiting	Hemoglobinopathy, Sickle Cell Disease A Long-term Follow-up Study in Patients Who Received BEAM-101 · NCT07373639