Pipeline
The 24 drug candidates we track, grouped by their most advanced phase. Each programme is derived from the company's registered studies on ClinicalTrials.gov and, where the page could be read, checked against the company's own pipeline page. Last checked against the sources: 27 October 2026.
Phase 39
Phase 212
Phase 12
Phase 41
Phase 3
| Programme | Company | Most advanced | Conditions | Partner | Sources | Checked |
|---|---|---|---|---|---|---|
| CTX001 Exagamglogene autotemcel, Exa-cel |
CRISPR Therapeutics | Phase 3? | Beta-Thalassemia; Thalassemia; Sickle Cell Disease and 3 more | Vertex Pharmaceuticals Incorporated | NCT04208529, NCT05329649, NCT05356195 Company pipeline page |
27 October 2026 |
| NTLA-2001 nexiguran ziclumeran |
Intellia Therapeutics | Phase 3? | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy; Neuromuscular Disease; Neuromuscular Diseases (NMD) and 3 more | Regeneron Pharmaceuticals | NCT06128629, NCT06672237 Company pipeline page |
27 October 2026 |
| NTLA-2002 Biological NTLA-2002 |
Intellia Therapeutics | Phase 3? | Hereditary Angioedema | – | NCT05120830, NCT06634420 Company pipeline page |
27 October 2026 |
| CTX001 Exagamglogene autotemcel, Exa-cel |
Vertex Pharmaceuticals | Phase 3? | Beta-Thalassemia; Thalassemia; Hematologic Diseases and 3 more | – | NCT05477563 | 27 October 2026 |
| VX-121 VX-121/TEZ/D-IVA, VX-121/VX-661/VX-561, VX-121/VX-661/CTP-656, VX-121/tezacaftor/deutivacaftor, VNZ/TEZ/D-IVA, vanzacaftor/tezacaftor/deutivacaftor |
Vertex Pharmaceuticals | Phase 3? | Cystic Fibrosis | – | NCT05422222, NCT05844449 | 27 October 2026 |
| VX-445 ELX/TEZ/IVA, VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor |
Vertex Pharmaceuticals | Phase 3? | Cystic Fibrosis | – | NCT06460506 | 27 October 2026 |
| VX-880 Zimislecel, Formerly known as STx-02 |
Vertex Pharmaceuticals | Phase 3? | Diabetes Mellitus, Type 1; Impaired Hypoglycemic Awareness; Severe Hypoglycemia | – | NCT04786262 | 27 October 2026 |
| Povetacicept ALPN-303 |
Vertex Pharmaceuticals | Phase 2/3? | Primary Membranous Nephropathy; Myasthenia Gravis, Generalized; Immunoglobulin A Nephropathy | – | NCT07204275, NCT07501702, NCT07807995 | 27 October 2026 |
| VX-147 | Vertex Pharmaceuticals | Phase 2/3? | Proteinuric Kidney Disease | – | NCT05312879 | 27 October 2026 |
Phase 2
| Programme | Company | Most advanced | Conditions | Partner | Sources | Checked |
|---|---|---|---|---|---|---|
| CS1 | Cereno Scientific AB | Phase 2? | Pulmonary Arterial Hypertension | – | NCT07761572 Company pipeline page |
27 October 2026 |
| TG01 TG01 Vaccine |
Circio Holding ASA | Phase 2? | Pancreas Cancer; Multiple Myeloma; Smoldering Multiple Myeloma | – | NCT05638698, NCT05841550 | 27 October 2026 |
| VX-407 | Vertex Pharmaceuticals | Phase 2? | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | – | NCT07161037 | 27 October 2026 |
| VX-670 | Vertex Pharmaceuticals | Phase 2? | Myotonic Dystrophy Type 1 (DM1) | – | NCT06926621 | 27 October 2026 |
| BEAM-101 | Beam Therapeutics | Phase 1/2? | Sickle Cell Disease | – | NCT05456880 Company pipeline page |
27 October 2026 |
| BEAM-201 Allogeneic anti-CD7 CAR-T cells (BEAM-201) |
Beam Therapeutics | Phase 1/2? | Lymphoblastic Lymphoma; T-Cell Lymphoblastic Leukemia/Lymphoma; Lymphoblastic Leukemia and 1 more | – | NCT05885464, NCT06934382 | 27 October 2026 |
| BEAM-301 BEAM-301: Single dose of BEAM-301 administered by IV |
Beam Therapeutics | Phase 1/2? | Glycogen Storage Disease Type Ia | – | NCT06735755 Company pipeline page |
27 October 2026 |
| BEAM-302 | Beam Therapeutics | Phase 1/2? | Alpha 1-Antitrypsin Deficiency | – | NCT06389877 Company pipeline page |
27 October 2026 |
| CTX112 Zugocabtagene geleucel, zugo-cel |
CRISPR Therapeutics | Phase 1/2? | B-cell Lymphoma; Non-Hodgkin Lymphoma; B-cell Malignancy and 3 more | – | NCT05643742, NCT06925542, NCT07549698, NCT07804004 Company pipeline page |
27 October 2026 |
| CTX340 | CRISPR Therapeutics | Phase 1/2? | Hypertension | – | NCT07758634 Company pipeline page |
27 October 2026 |
| CTX460 | CRISPR Therapeutics | Phase 1/2? | Alpha-1 Antitrypsin Deficiency (AATD) | – | NCT07850076 Company pipeline page |
27 October 2026 |
| VX-522 VX-522 mRNA therapy |
Moderna | Phase 1/2? | Cystic Fibrosis | Vertex Pharmaceuticals Incorporated | NCT05668741 | 27 October 2026 |
Phase 1
| Programme | Company | Most advanced | Conditions | Partner | Sources | Checked |
|---|---|---|---|---|---|---|
| BEAM-103 | Beam Therapeutics | Phase 1? | Healthy Subjects; Healthy Participant Study | – | NCT07304791 Company pipeline page |
27 October 2026 |
| CTX310 | CRISPR Therapeutics | Phase 1? | Cardiovascular; Metabolic Disease; Dyslipidemias and 3 more | – | NCT07491172 Company pipeline page |
27 October 2026 |
Phase 4
| Programme | Company | Most advanced | Conditions | Partner | Sources | Checked |
|---|---|---|---|---|---|---|
| Suzetrigine suzetrigine, Journavx, JOURNAVX® |
Vertex Pharmaceuticals | Phase 4? | Pain, Acute Postoperative; Breast Disease; Knee Arthritis and 3 more | – | NCT07463430, NCT07624526, NCT07795827, NCT07836010 | 27 October 2026 |
Partners are the industry sponsors and collaborators named in the study records. Phase 4 means studies after approval.
All registered studies
Every active study we track, by phase. Each row links to the study record on ClinicalTrials.gov.
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 |