Cell and Gene 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
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
Hemophilia A
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)
MPS I
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)