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Gene Therapy for Fanconi Anemia Complementation Group A

The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the …

Gene Therapy for X Linked Severe Combined Immunodeficiency

A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCIDsevere combined immune deficiency .The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number …

AAV Gene Therapy Study for Subjects With PKU

This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects 2 and 50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) with LentiGlobin BB305 Drug Product for SCD.

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).

Validation of Standardized Test Protocols to Assess the Impact of Visual Pathologies in Daily Life Activities

This tool can be used to measure a therapeutic benefit for new treatments (like gene therapy, retinal implants, intra vitreous injections ) for visual loss patients.

Multi-4SCAR-T Therapy Targeting Breast Cancer

The purpose of this study is to assess the feasibility, safety and efficacy of multiple 4th generation CAR-T cells targeting Her2, GD2, and CD44v6 surface antigen in breast cancer. Another goal of the study is to learn more about the activities of the multi-CAR T cells and their persistency in …

Chimeric Antigen Receptor T-cells for The Treatment of AML Expressing CLL-1 Antigen

Patients eligible for this study have a type of blood cancer Acute Myeloid Leukemia (AML) which has come back or has not gone away after treatment. The body has different ways of fighting disease and infection, and this research study combines two different ways of fighting cancer with antibodies and …

no site and no over all conatct

no site and no over all conatct