Gene and Cell Therapy Clinical Trials

Acute Lymphoblastic Leukemia

• CAR-T Long Term Follow Up (LTFU) Study (PAVO)
• CD22 Redirected Autologous T Cells for ALL
• Pilot Study of Redirected Autologous T Cells Engineered to Contain Humanized Anti-CD19 in Patients With Relapsed or Refractory CD19+ Leukemia and Lymphoma Previously Treated With Cell Therapy
• Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients (CASSIOPEIA)
• Study of huCART19 for Very High-Risk (VHR) Subsets of Pediatric B-ALL
• Study of UCART19 in Pediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukemia (PALL)
• Study Evaluating KTE-X19 in Pediatric and Adolescent Participants With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4)
• Phase 2 Trial of CD19 Redirected Autologous T Cells
• Determine Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell ALL and High Risk B-cell ALL at First Relapse. Determine Feasibility and Safety of CTL019 Therapy in Pediatric Patients With High Risk B-cell ALL That Relapsed < 6 Months Post All-HSCT. (ELIANA)

Acute myeloid leukemia

• Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia

Beta thalassemia

• A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia
• A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype (Bluebird Bio)
• A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Choroideremia

• Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations

Hemophilia A

• A Gene Transfer Study for Hemophilia A
• Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
• Long-Term Safety and Efficacy of SPK-8011 in Males With Hemophilia A

Hemophilia B

• A factor IX (FIX) gene transfer, multi-center evaluation of the LONG term safety and efficacy Study of SPK-9001 (rAAV-Spark100-hFIXPadua) in individuals with hemophilia B

Leber congenital amaurosis

• Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
• Safety Study in Subjects With Leber Congenital Amaurosis
• Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
• Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Methylmalonic acidemia (MMA)

• Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

MPS I

• RGX-111 Gene Therapy in Patients With MPS I

MPS II (Hunter syndrome)

• RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

Non-hodgkin lymphoma

• Phase II Open Label Trial to Determine Safety & Efficacy of Tisagenlecleucel in Pediatric Non-Hodgkin Lymphoma Patients (BIANCA)
• Study Evaluating KTE-X19 in Pediatric and Adolescent Participants With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4)

Phenylketonurias

• Gene Therapy Clinical Study in Adult PKU (pheNIX)

Sickle cell disease

• A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe Sickle Cell Disease

Spinal muscular atrophy

• Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
• Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy (STRONG)