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CD33 CART AML
This phase 1/2 study will determine the safety, feasibility, and efficacy of CD33 CART cells following lymphodepleting chemotherapy in patients with acute myeloid leukemia (AML).
DTX401 Gene Therapy in Glycogen Storage Disease (GSD) Type Ia
Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa)
Gene Therapy Trial for Hemophilia A
This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver.
HGB-212: Phase 3 Beta Thalassemia LentiGlobin BB305 Gene Therapy Trial
Individuals with a certain type of beta thalassemia that is treated with regular transfusions, and who are 50 years old and younger, may be able to participate.
Hodgkin Lymphoma (NHL) (BIANCA)
This purpose of this study is to determine the efficacy and safety of tisagenlecleucel (also referred to as CTL019, or Kymriah™) in children, adolescents and young adults with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL).
Humanized CART19 Phase 2 Trial
This purpose of this study is to determine the efficacy of humanized CD19 CAR T cells (huCART19) in pediatric and young adult patients with high-risk relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).
This study will take the patients white blood cells - T cells - and change them to turn against the cancer.
Phase 2 trial of CART19 for Orphan Indications Of Pediatric B Cell Acute Lymphoblastic Leukemia (B-ALL)
This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19.
RGX-111 Gene Therapy in Patients With Mucopolysaccharidosis Type I (MPS I)
This study will evaluate the safety and tolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with a neurocognitive deficit that are at least 4 months old.
RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)
The purpose of this study is to look at the safety and tolerability of RBX-121, a one-time gene therapy, in subjects with MPS II (Hunter Syndrome).