Abstract: The invention provides improved gene therapy methods and compositions.

Abstract: The invention provides anti-?S globin antibodies or antigen binding fragments thereof.

Abstract: The present invention provides methods and compositions for improving efficacy of viral transduction of cells. More particularly, the present invention provides methods and materials useful for safely and reliably improving the efficiency of methods and materials useful for safely and reliably improving the efficiency of methods for transducing cells, such as human hematopoietic stem cells (HSC), with viruses and/or viral vectors. The compositions and methods are useful for therapeutic indications amenable to treatment with hematopoietic stem cell gene therapies.

Abstract: Provided are improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. Also provided are improved gene therapy methods for treating hematopoietic-related disorders. Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.

Abstract: The invention generally provides improved anti-sickled-globin antibodies and fragments thereof. In particular embodiments, the antibody or antigen binding fragment thereof recognizes the ?s, Glu6Val mutation. In various embodiments, the invention provides a conjugate comprising an anti-?s-globin antibody or antigen binding fragment thereof and a detectable label. In particular embodiments, a hybridoma comprising an anti-?s-globin antibody contemplated herein is provided.

Abstract: The invention provides compositions and methods for treating MPS I.

Abstract: The invention provides, in part, compositions and methods for treating neuronal ceroid lipofuscinoses (NCL). The invention further provides, in part, gene therapy compositions and methods for the treatment, prevention, or amelioration of at least one symptom of NCL. Particular embodiments provide a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a lentiviral vector, or a mammalian cell transduced with a lentiviral vector. Further embodiments provide a method of treating NCL comprising administering a lentiviral vector or a mammalian cell transduced with a lentiviral vector.

Abstract: Disclosed herein are potency assays for a gene therapy treatment for ?-thalassemia. Also disclosed herein are methods for measuring relative potency of a drug product.

Abstract: Disclosed herein are potency assays for a gene therapy treatment for sickle cell disease. Also disclosed herein are methods for measuring relative potency of a drug product used for the treatment of sickle cell disease.

Abstract: The invention provides improved gene therapy methods and compositions. In particular embodiments, gene therapies comprise hematopoietic stem and progenitor cell compositions with increased therapeutic efficacy and methods of making and using the same. In other particular embodiments, the present invention contemplates compositions and methods for increasing transduction efficiency and vector copy number (VCN) of human hematopoietic stem and progenitor cells (HSPCs) to yield improved gene therapy compositions. In various embodiments, the present invention contemplates, in part, a population of HSPCs transduced with a lentiviral vector. In various embodiments, the present invention contemplates a method of treating sickle cell disease in a subject comprising administering the subject an effective amount of the population of hematopoietic cells contemplated herein.

Abstract: The invention provides improved gene therapy vectors, compositions, and methods.

Abstract: The present disclosure provides improved compositions for adoptive T cell therapies for treating, preventing, or ameliorating at least one symptom of a cancer, infectious disease, autoimmune disease, inflammatory disease, and immunodeficiency, or condition associated therewith.

Abstract: The present disclosure provides improved compositions for adoptive T cell therapies targeting CD33 for treating, preventing, or ameliorating at least one symptom of a cancer, infectious disease, autoimmune disease, inflammatory disease, and immunodeficiency, or condition associated therewith. The present disclosure also relates to adoptive T cell therapies targeting CD33 and another target antigen for treating, preventing, or ameliorating at least one symptom of a cancer, infectious disease, autoimmune disease, inflammatory disease, and immunodeficiency, or condition associated therewith.

Abstract: The present disclosure provides improved cell therapy vessels, cell therapy vessel adapters, kits, and methods of using the same.

Abstract: The invention provides improved compositions for adoptive cell therapies for cancers that express CD79A.

Abstract: The invention provides improved methods for detecting anti-BCMA CAR expression on T cells.

Abstract: The invention provides improved compositions and methods for the treatment of solid cancers.

Abstract: The present disclosure provides improved compositions for adoptive T cell therapies targeting NKG2D ligands for treating, preventing, or ameliorating at least one symptom of a cancer, infectious disease, autoimmune disease, inflammatory disease, and immunodeficiency, or condition associated therewith.

Abstract: The present disclosure provides improved genome editing compositions and methods for editing a CBLB gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.

Abstract: The present disclosure provides improved genome editing compositions and methods for editing a CBLB gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.