Abstract: The present disclosure provides improved conditioning methods for use in hematopoietic stem cell transplantation and hematopoietic stem cell-based gene therapies.

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 methods and compositions.

Abstract: A retroviral construct harboring nucleic acids encoding a high mobility group AT-hook 2 (HMGA2) protein and lacking let-7 binding sites is described as are methods of using the retroviral vector to increase the efficacy and in vivo expansion of transduced cells in gene therapy applications.

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.