Bluebird Bio, Inc.

Olivier Negre

Melissa Bonner

Yves Beuzard

Philippe Louis Leboulch

Christopher Tipper

Emmanuel Payen

The invention provides anti-βglobin antibodies or antigen binding fragments thereof.

Sickled beta globin antibodies

The invention provides improved gene therapy methods and compositions.

VCN enhancer compositions and methods of using the same

The invention generally provides improved anti-sickled-globin antibodies and fragments thereof. In particular embodiments, the antibody or antigen binding fragment thereof recognizes the &#x3b2;s, Glu6Val mutation. In various embodiments, the invention provides a conjugate comprising an anti-&#x3b2;s-globin antibody or antigen binding fragment thereof and a detectable label. In particular embodiments, a hybridoma comprising an anti-&#x3b2;s-globin antibody contemplated herein is provided.

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. In various embodiments, the present invention contemplates a kit comprising an agent that increases prostaglandin EP receptor signaling and staurosporine.

The present invention generally provides improved gene therapy vectors, cell-based compositions, and methods of using the same in methods of gene therapy. The present invention further provides improved gene therapy compositions for expanding hematopoietic cells and related methods for treatment of diseases, disorders, and conditions of the hematopoietic system such as thalassemias and anemias.

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Cambridge, MA, United States of America

Olivier Negre