Cell Genesys, Inc.

Gbp Ip, LLC

Ryan McGuinness

Thomas L Dull

James G McArthur

Andrew Simmons

Dale John Talbot

Michael Kelly

Lisa V Tsui

Mitchell H Finer

Luigi Naldini

Richard O Snyder

Recombinant lentiviruses and transfer vectors for transgene delivery as well as methods for gene therapy using such vectors are disclosed. The invention provides a third generation lentiviral packaging system and a set of vectors for producing recombinant lentiviruses, as well as novel tissue specific enhancer and promoter elements useful for optimizing liver specific transgene delivery. The transgene is preferably a blood clotting factor such as human factor IX (hFIX) or human factor VIII (hFVIII) and can be used for treatment of hemophilia.

Lentiviral vectors featuring liver specific transcriptional enhancer and methods of using same

Liver specific transcriptional enhancer

Lentiviral vectors encoding clotting factors for gene therapy

The instant invention provides methods and materials for expressing a polypeptide with factor VIII activity comprising administering an rAAV vector encoding a truncated version of human factor VIII, containing, for example, a 90 kD heavy chain of factor VIII fused to a light chain of factor VIII.

Recombinant AAV vectors for gene therapy of hemophilia A

A method for selecting packaging cells that express high levels of gag/pol is provided.

Selection system for generating efficient packaging cells for lentiviral vectors

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Oakland, CA, United States of America

Ryan McGuinness