What Cambridge is waiting on.

FDA target action date (PDUFA)

FDA target action date (PDUFA)

Study to Assess the Safety and Tolerability of CFT7455 in Relapsed/Refractory Non-Hodgkin's Lymphoma or Multiple Myeloma

Multiple Myeloma · Lymphoma, Non-Hodgkin's

FDA target action date (PDUFA)

A Study to Evaluate Efficacy and Safety of Intravenous Sabirnetug in Participants With Early Alzheimer's Disease (ALTITUDE-AD)

Alzheimer Disease

Study of DISC-0974-201 in Participants With IBD and Anemia

Inflammatory Bowel Disease (IBD) · Anemia · Inflammatory Bowel Disease (IBD); Anemia

A Study of an Epstein-Barr Virus (EBV) Candidate Vaccine, mRNA-1189, in 10- to 30-Year-Old Healthy Adolescents and Adults

Epstein-Barr Virus Infection

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Duchenne Muscular Dystrophy

Study of RP2 Monotherapy and RP2 in Combination With Nivolumab in Patients With Solid Tumors

Cancer

A Study of Setmelanotide in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome · Obesity · Hyperphagia

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

Muscular Dystrophy, Duchenne

A Study of SPY072 in Rheumatic Disease

Rheumatoid Arthritis · Psoriatic Arthritis · Axial Spondyloarthritis

A Study of TX000045 in Patients With Pulmonary Hypertension Secondary to Heart Failure With Preserved Ejection Fraction (the APEX Study)

Pulmonary Hypertension · Heart Failure With Preserved Ejection Fraction

Suprachoroidal Administration in Subjects With Metastases to the Choroid

Eye Cancer · Primary Cancer

FDA target action date (PDUFA)

A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

Glycogen Storage Disease

Study of RP3 Monotherapy and RP3 in Combination With Nivolumab in Patients With Solid Tumours

Advanced Solid Tumor

FDA target action date (PDUFA)

A Phase 1/2 Trial of ADI-270 in ccRCC

Clear Cell Renal Cell Carcinoma

A Phase 1 Study of Prulacabtagene Leucel (Prula-cel, Formerly ADI-001) in Autoimmune Disease

Lupus Nephritis · Autoimmune Diseases · Systemic Sclerosis (SSc)

A Clinical Trial Evaluating Patients With Primary Vitreoretinal Lymphoma

Primary Vitreoretinal Lymphoma

C-Forward: Efficacy and Safety of BEM/RZR vs SOF/VEL in Subjects With Chronic HCV

HEPATITIS C VIRUS CHRONIC INFECTION · Hepatitis C, Chronic · Hepatitis C Virus Infection

AURA-IPF: A Randomized Phase 2 Study to Evaluate the Safety and Efficacy of AP02 (Nintedanib Solution) in IPF

Idiopathic Pulmonary Fibrosis (IPF)

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)

Lymphoblastic Lymphoma · T-Cell Lymphoblastic Leukemia/Lymphoma · Lymphoblastic Leukemia

A Phase 1 of CTX-8371 in Patients With Advanced Malignancies

Non Small Cell Lung Cancer · Triple Negative Breast Cancer · Hodgkin Lymphoma

A Phase 2 Study to Investigate the Efficacy and Safety of Zelicapavir in Participants Aged ≥28 Days to ≤36 Months of Age Infected With Respiratory Syncytial Virus

RSV Infection · RSV · Respiratory Syncytial Virus (RSV)

A Study of Rituximab-Gemcitabine-Dexamethasone-Platinum (R-GDP) With or Without Selinexor in Patients With Relapsed/Refractory Diffuse Large B-cell Lymphoma

Relapsed/Refractory Diffuse Large B-cell Lymphoma

Selinexor (KPT-330) in Patients With Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL)

Diffuse Large B-cell Lymphoma

A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

Epileptic Encephalopathy · SCN2A Encephalopathy

A Clinical Trial for Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine

Developmental and Epileptic Encephalopathy 1

FDA target action date (PDUFA)

FDA target action date (PDUFA)

A Basket Study of Customized Autologous TCR-T Cell Therapies in Patients With Locally Advanced (Unresectable) or Metastatic Solid Tumors

Head and Neck Cancer · Cervical Cancer · Non-small Cell Carcinoma

Study of Safety and Tolerability of BCA101 Monotherapy and in Combination Therapy in Patients With EGFR-driven Advanced Solid Tumors

Head and Neck Squamous Cell Carcinoma · Squamous Cell Carcinoma of Anal Canal · Colorectal Cancer

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

Myotonic Dystrophy 1

A Study of NVL-330 in Patients With Advanced or Metastatic HER2-altered NSCLC (HEROEX-1)

Locally Advanced Solid Tumor · Metastatic Solid Tumor

Randomized Clinical Trial Evaluating the Efficacy and Safety of Vormatrigine in Adults With Focal Seizures

Focal Epilepsy

Study to Evaluate the Efficacy and Safety of Intravitreal OTX-TKI (Axitinib Implant) in Subjects With Neovascular Age- Related Macular Degeneration

Neovascular Age-related Macular Degeneration

FDA target action date (PDUFA)

FDA target action date (PDUFA)

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

ALS

A Biomarker Study in Men With Localized Prostate Cancer Treated With Aglatimagene Besadenovec

Prostate Cancer Patients Treated by Radiotherapy · Prostate Cancer (Adenocarcinoma)

A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)

Polycythemia Vera (PV)

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Myotonic Dystrophy Type 1 (DM1)

A Study to Investigate the Safety and Efficacy of mRNA-1403 in Participants ≥18 Years of Age for the Prevention of Acute Gastroenteritis

Acute Gastroenteritis · Norovirus Acute Gastroenteritis

FDA target action date (PDUFA)

A Phase 1, Open-label Trial of Belzupacap Sarotalocan (AU-011) in Bladder Cancer

Non-muscle-invasive Bladder Cancer · NMIBC · Non-Muscle Invasive Bladder Carcinoma

A Study of an FGFR2/3 Inhibitor (CGT4859) in Patients With Cholangiocarcinoma and Other Advanced Solid Tumors

Intrahepatic Cholangiocarcinoma (Icc) · Cholangiocarcinoma · Other Solid Tumors, Adult

A Study to Evaluate the Efficacy and Safety of OTX-TKI (Axitinib Implant) in Participants With Non-Proliferative Diabetic Retinopathy

Non-Proliferative Diabetic Retinopathy

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

Myotonic Dystrophy 1

A Study to Evaluate the Efficacy and Safety of SPT-300 (GlyphAllo) in Participants With Major Depressive Disorder, With or Without Anxious Distress (BUOY-1 Study)

Major Depressive Disorder (MDD) · Major Depressive Disorder With Anxious Distress

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

Dravet Syndrome

A Study to Evaluate the Efficacy, Safety, and Immunogenicity of mRNA-1647 Cytomegalovirus (CMV) Vaccine in Allogenic Hematopoietic Cell Transplantation (HCT) Participants.

Cytomegalovirus Infection

FDA target action date (PDUFA)

A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

Hypothalamic Obesity · Multiple Pituitary Hormone Deficiency Genetic Form · Septo-Optic Dysplasia

Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy

Diabetic Peripheral Neuropathic Pain

A Phase 1 Study of ADI-001 in Rheumatoid Arthritis

Rheumatoid Arthritis (RA)

Safety and Tolerability of TNG456 Alone and in Combination With Abemaciclib in Patients With Solid Tumors With MTAP Loss

Non Small Cell Lung Cancer · Glioma Glioblastoma Multiforme · Glioma, Malignant

Selinexor and Backbone Treatments of Multiple Myeloma Patients

Multiple Myeloma

FOG-001 in Locally Advanced or Metastatic Solid Tumors

Cancer · Colorectal Cancer · Solid Tumor

Evaluation of Efficacy and Safety of Suzetrigine (SUZ) for Pain Associated With Diabetic Peripheral Neuropathy

Diabetic Peripheral Neuropathic Pain

A Study of PYX-201 in Advanced Solid Tumors

Solid Tumor · Advanced Solid Tumor

A Study to Evaluate ALN-2232 in Participants With Obesity

Obesity

First-in-Human Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, as a Single Agent in Patients With Advanced Solid Tumors and in Combination With Endocrine Therapy +/- a CDK4/6 or CDK4 Inhibitor in Patients With Advanced Solid Tumors or Advanced Breast Cancer

PIK3CA Mutation · Solid Tumor, Adult · HER2-negative Breast Cancer

A Study to Investigate GB-0895 in Adults With Mild to Moderate Asthma or COPD

Asthma · COPD (Chronic Obstructive Pulmonary Disease)

Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia

Myelofibrosis; Anemia · Anemia · Myelofibrosis

Efficacy and Safety of Ribupatide (KAI-9531) Administered Once Weekly in Participants Living With Obesity Who Do Not Have Diabetes

Obesity

A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG)

Generalized Myasthenia Gravis

VY7523-102: Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Dose Study in Participants With Early Alzheimer's Disease

Alzheimer's Disease (AD)

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Duchenne Muscular Dystrophy

A Study to Evaluate the Safety, Tolerability, and Efficacy of BIIB017 (Peginterferon Beta-1a) in Pediatric Participants for the Treatment of Relapsing-Remitting Multiple Sclerosis

Multiple Sclerosis, Relapsing-Remitting

FDA target action date (PDUFA)

A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus

Type 2 Diabetes Mellitus (T2DM)

A Phase 2 Study of ACR-368 in Endometrial Adenocarcinoma

Endometrial Adenocarcinoma

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

Evaluation of Efficacy and Safety of Suzetrigine for Pain Associated With Diabetic Peripheral Neuropathy

Diabetic Peripheral Neuropathic Pain

A Study of Praliciguat in Participants With Focal Segmental Glomerulosclerosis (FSGS)

Focal Segmental Glomerulosclerosis

A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis

Pulmonary Fibrosis · Progressive Pulmonary Fibrosis · Pulmonary Fibrosis Secondary to Systemic Sclerosis

Study of GB-4362 With Enfortumab Vedotin and Pembrolizumab for Advanced Urothelial Cancer

Advanced Urothelial Cancer · Metastatic Urothelial Carcinoma

A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

Sickle Cell Disease

A Study of Selinexor Monotherapy in Subjects With JAK Inhibitor-naïve Myelofibrosis and Moderate Thrombocytopenia

Myelofibrosis · Moderate Thrombocytopenia · Mild Thrombocytopenia

A Study of KT-621 Administered Orally to Participants With Moderate to Severe Atopic Dermatitis

Atopic Dermatitis

[18F]PI-2620 Phase 3 Histopathological Study

Alzheimer Disease

A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

Catecholaminergic Polymorphic Ventricular Tachycardia

A Study of Long-acting Antibodies Alone and in Combinations for Moderate to Severe Ulcerative Colitis

Ulcerative Colitis · Inflammatory Bowel Diseases · Colitis

Study to Evaluate the Safety, Tolerability & Efficacy of TNG462 in Combination in PDAC & NSCLC Patients

PDAC · PDAC - Pancreatic Ductal Adenocarcinoma · NSCLC

Safety and Tolerability of TNG462 in Patients With MTAP-deleted Solid Tumors

Locally Advanced Solid Tumor

A Phase 2 Study of VS-7375 in Patients With KRAS G12D-Mutated Colorectal Cancer

Colorectal Cancer

A Phase 2 Study of VS-7375 in Patients With KRAS G12D-Mutated Non-Small Cell Lung Cancer

Non-Small Cell Lung Cancer

A Phase 2 Study of VS-7375 in Patients With KRAS G12D-Mutated Pancreatic Cancer

Pancreatic Ductal Adenocarcinoma (PDAC) · G12D Mutated KRAS

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Sickle Cell Disease · Hydroxyurea Failure · Hydroxyurea Intolerance

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Beta-Thalassemia · Thalassemia · Hematologic Diseases

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

Von Willebrand Disease (VWD) · Heavy Menstrual Bleeding (HMB)

A Study Assessing HMB-002 in Participants With Von Willebrand Disease

Von Willebrand Disease (VWD) · Von Willebrand Disease (VWD), Type 1 · Von Willebrand Disease (VWD), Type 2

A Phase 3 Study of NTLA-2001 in ATTRv-PN

Neuromuscular Disease · Neuromuscular Diseases (NMD) · Neurodegenerative Disease

Efficacy and Safety of Oral KAI-7535 in Adult Participants Living With Obesity or Overweight With at Least 1 Weight-Related Comorbidity

Obesity · Overweight

Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia

Glanzmann Thrombasthenia

Lu-TARGO (177Lu-TARGeted Osteosarcoma Therapy)

Relapsed / Refractory Osteosarcoma

A Study of mRNA-4106 or mRNA-4200 Administered Alone or in Combination With Anti-Cancer Agents in Participants With Solid Tumors

Advanced Solid Tumors

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy

Cerebral Amyloid Angiopathy

A Study of CTX-009 in Combination With Paclitaxel in Adult Patients With Unresectable Advanced, Metastatic or Recurrent Biliary Tract Cancers (COMPANION-002)

Biliary Tract Cancer · Cholangiocarcinoma · Gall Bladder Cancer

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

Propionic Acidemia

A Study of Botensilimab and Balstilimab for the Treatment of Colorectal Cancer

Metastatic Colorectal Cancer

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

Neutropenia

A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma

Advanced Hepatocellular Carcinoma · Metastatic Hepatocellular Carcinoma

Study of Oral MRT-2359 With Apalutamide in Prostate Cancer

Castration-Resistant Prostate Cancer (CRPC) · Castration-Resistant Prostate Cancer · Prostate Cancer

A Phase 1 Study of JADE201 in Participants With Rheumatoid Arthritis

Rhematoid Arthritis

A Study of SPY072 in Adults With Moderate to Severe Hidradenitis Suppurativa (The SKYLIGHT Trial)

Hidradenitis Suppurativa (HS)

A Study to Assess the Efficacy and Safety of Verekitug in Participants With COPD

Chronic Obstructive Pulmonary Disease

A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency

Congenital Factor VII Deficiency

A Study of Emavusertib + An Approved Bruton Tyrosine Kinase Inhibitor (BTKi) in Participants With Chronic Lymphocytic Leukemia (CLL) and Other B-cell Malignancies

Chronic Lymphocytic Leukemia · B-cell Malignancies

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Beta-Thalassemia · Thalassemia · Genetic Diseases, Inborn

A Phase 3 Randomized, Masked, Controlled Trial to Evaluate Efficacy and Safety of Belzupacap Sarotalocan (AU-011) Treatment Compared to Sham Control in Subjects With Primary Indeterminate Lesions or Small Choroidal Melanoma

Choroidal Melanoma · Indeterminate Lesions · Uveal Melanoma

Safety, Tolerability, and Immunogenicity of mRNA-4157 Alone and in Combination in Participants With Solid Tumors

Solid Tumors

A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease

Huntington's Disease

A Study of [225Ac]Ac-AKY-1189 in Patients With Solid Tumors

Urothelial Carcinoma Bladder · Triple Negative Breast Cancer (TNBC) · Hormone Receptor Positive Breast Adenocarcinoma

A Study of [225Ac]Ac-AKY-2519 in Patients With Metastatic Castration-Resistant Prostate Cancer

Castration Resistant Metastatic Prostate Cancer · Prostate Cancer · mCRPC (Metastatic Castration-resistant Prostate Cancer)

A Study of KT-621 Administered Orally to Adult Participants With Moderate to Severe Eosinophilic Asthma

Eosinophilic Asthma

Study to Evaluate Efficacy and Safety of Roluperidone in Adult Subjects With Negative Symptoms and Stable Positive Symptoms of Schizophrenia and to Evaluate the Relapse Rate of Roluperidone and Antipsychotic Medications

Negative Symptoms of Schizophrenia

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

A Study of Neladalkib (NVL-655) in Patients With Advanced NSCLC and Other Solid Tumors Harboring ALK Rearrangement or Activating ALK Mutation (ALKOVE-1)

Locally Advanced Solid Tumor · Metastatic Solid Tumor

Study of RP2 in Combination With Second-line Therapy in Patients With Locally Advanced or Metastatic HCC

Hepatocellular Carcinoma · Biliary Tract Cancer

Phase 1/2 Study of PYX-201 in Combination With Pembrolizumab in Advanced Solid Tumors

Advanced Solid Tumors

A Study to Evaluate ALN-6222 in Participants With Obesity

Obesity

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy · hATTR-PN

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)

Glycogen Storage Disease Type Ia

A First-in-human Study of RLY-8161 in Advanced NRAS-Mutant Solid Tumors

Advanced NRAS-Mutant Melanoma · Advanced NRAS-Mutant Solid Tumors · NRAS Mutation

A Study of Zidesamtinib (NVL-520) in Patients With Advanced NSCLC and Other Solid Tumors Harboring ROS1 Rearrangement (ARROS-1)

Locally Advanced Solid Tumor · Metastatic Solid Tumor

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

Duchenne Muscular Dystrophy

A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes

Diabetes Mellitus, Type 1 · Impaired Hypoglycemic Awareness · Severe Hypoglycemia

A Study of Selinexor (Seli) + Low-dose Dexamethasone (LDD) in Penta-refractory Multiple Myeloma (MM), Seli and Bortezomib + LDD in Triple-class Refractory MM.

Multiple Myeloma, Refractory

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

Phenylketonuria

A Study to Learn About The Effects of Felzartamab on Thyroid Function and Its Safety in Adults With Graves' Disease Ages 18 to 75 Years Old

Graves' Disease

A Study of Botensilimab (AGEN1181) for the Treatment of Advanced Melanoma

Advanced Melanoma

A Phase 2 Study to Evaluate the Safety, PD, PK, and Clinical Activity of ADX-097 in Participants With IgAN, LN or C3G

IgA Nephropathy · Lupus Nephritis (LN) · C3 (Complement Component 3) Glomerulopathy

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

Sickle Cell Disease

Efficacy and Safety of Ribupatide in Participants Living With Obesity or Overweight With Weight-Related Comorbidities Who Do Not Have Diabetes

Obesity · Overweight

A Study to Evaluate the Impact of Renal Impairment on the Pharmacokinetics (PK) of Ribupatide

End Stage Renal Disease · Renal Impairment · Obesity

A Study to Learn About the Effects of Felzartamab Infusions in Adults With Kidney Transplants Who Have Late Isolated Microvascular Inflammation

Microvascular Inflammation

A Study Evaluating Ficerafusp Alfa (BCA101) QW in Combination With Pembrolizumab vs Alternative Ficerafusp Alfa Dosing in 1L R or M HNSCC

Metastatic Head and Neck Squamous Cell Carcinoma · Recurrent Head and Neck Squamous Cell Carcinoma

Efficacy and Safety of Ribupatide Administered Once Weekly Compared With Semaglutide and Placebo in Participants Living With Obesity Who Do Not Have Diabetes

Obesity