Csl Behring Gene Therapy, Inc.

Csl Gene Therapy Pty Ltd.

Calimmune, Inc.

Chi-Lin Lee

Jeffrey S Bartlett

Geoffrey Phillip Symonds

Ming Yan

Jeffrey Ahlers

Gene-Errol Eugenio Ringpis

The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or &#x3b2;-thalassemia or to reduce symptoms thereof.

Compositions and methods for treating beta-hemoglobinopathies

The present disclosure provides a method of generating a stable producer cell line. The generation of stable producer cell lines, such as those provided in accordance with the present invention, increases the reproducibility and ease of creating high titer lentiviral stocks while easing biosafety concerns and the variation in expressed envelope proteins defines the tropism of the generated virus. The present disclosure also provides for a novel lentiviral transfer vector plasmid.

Bio-production of lentiviral vectors

Growing community of inventors

Arcadia, CA, United States of America

Chi-Lin Lee