Cell Genesys, Inc.

Gbp Ip, LLC

Thomas L Dull

Deborah A Farson

Luigi Naldini

Rochelle Witt

Mitchell H Finer

Ryan McGuinness

Krisztina M Zsebo

James G McArthur

Andrew Simmons

Dale John Talbot

Michael Kelly

Lisa V Tsui

Margo R Roberts

Lu Qin

Anatoly Bukovsky

Richard O Snyder

Keegan Cooke

Lentiviral vectors modified at the 5' LTR or both the 5&#x2032; and 3&#x2032; LTR's are useful in the production of recombinant lentivirus vectors. Such vectors can be produced in the absence of a functional tat gene. Multiple transformation of the host cell with the vector carrying the transgene enhances virus production.

Method and means for producing high titer, safe recombinant lentivirus vectors

Lentiviral vectors modified at the 5' LTR or both the 5&#x2032; and 3&#x2032; LTR are useful in the production of recombinant lentivirus vectors (See the Figure). Such vectors can be produced in the absence of a functional tat gene. Multiple transformation of the host cell with the vector carrying the transgene enhances virus production. The vectors can contain inducible or conditional promoters.

Method and means for producing high titer, safe, recombinant lentivirus vectors

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

The invention provides a novel retroviral packaging system, in which retroviral packaging plasmids and packagable vector transcripts are produced from high expression plasmids after stable or transient transfection in mammalian cells. High titers of recombinant retrovirus are produced in these transfected mammalian cells and can then transduce a mammalian target cell by cocultivation or supernatant infection. The methods of the invention include the use of the novel retroviral packaging plasmids and vectors to transduce primary human cells, including T cells and human hematopoietic stem cells, with foreign genes by cocultivation or supernatant infection at high efficiencies. The invention is useful for the rapid production of high titer viral supernatants, and to transduce with high efficiency cells that are refractory to transduction by conventional means.

Method for production of high titer virus and high efficiency retroviral mediated transduction of mammalian cells

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

The invention provides a novel retroviral packaging system, in which retroviral packaging plasmids and packagable vector transcripts are produced from high expression plasmids after stable or transient transfection in mammalian cells. High titers of recombinant retrovirus are produced in these transfected mammalian cells and can then transduce a mammalian target cell by cocultivation or supernatant infection. The methods of the invention include the use of the novel retroviral packaging plasmids and vectors to transduce primary human cells, including T cells and, human hematopoietic stem cells, with foreign genes by cocultivation or supernatant infection at high efficiencies. The invention is is useful for the rapid production of high titer viral supernatants, and to transduce with high efficiency cells that are refractory to transduction by conventional means.

Lentiviral vectors modified at the 5&prime; LTR or both the 5&prime; and 3&prime; LTR's are useful in the production of recombinant lentivirus vectors. Such vectors can be produced in the absence of a functional tat gene. Multiple transformation of the host cell with the vector carrying the transgene enhances virus production.

Lentiviral vectors modified at the 5' LTR or both the 5' and 3' LTR's are useful in the production of recombinant lentivirus vectors. Such vectors can be produced in the absence of a functional tat gene. Multiple transformation of the host cell with the vector carrying the transgene enhances virus production.

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San Francisco, CA, United States of America

Thomas L Dull