Transgene S.a.

The United States of America As Represented by the Department of Health

Andrea Pavirani

Michel Perricaudet

Majid Mehtali

Leslie Stratford-Perricaudet

Jean-Luc Imler

Pierre Meulien

Wilfried Dalemans

Ronald G Crystal

Sophie Jallat

Frederic Perraud

The present invention relates to a replication deficient recombinant adenovirus vector in the genome of which is inserted an expression cassette comprising the DNA fragment coding for the human CFTR protein, said DNA fragment being placed under the control of the elements for the expression thereof. The present invention also concerns a vector according to claim 1 wherein in the expression cassette the human CFTR gene is under the control of the endogenous human CFTR promoter.

Replication deficient recombinant adenovirus vector

Novel defective adenoviruses for the transfer and expression of an exogenous nucleotide sequence in a host cell or organism. The invention also relates to novel complementation lines and to the process for the preparation of these novel defective adenoviruses and their use in therapy and to a pharmaceutical composition containing same.

Defective adenoviruses and corresponding complementation lines

The present invention relates, in general, to a adenovirus mediated transfer of genes to the lung. In particular, the present invention relates to a method of recombinant, replication-deficient adenovirus mediated transfer of desired genes to the lung whereby desired proteins of interest are produced for local and/or systemic use.

Adenovirus comprising deletions on the E1A, E1B and E3 regions for

Cell lines have been prepared from transgenic mice comprised of a first exogenous DNA sequence encoding human factor IX which is controlled by a promoter region specific for hepatic cells and a second exogenous DNA sequence encoding an oncogene, such as the SV40 virus gene encoding the T-antigen or the mouse c-myc gene, which is controlled by a promoter region specific for hepatic cells. Promoter regions specific for hepatic cells included the promoter region for the factor IX gene and the promoter region for the .alpha.-1 antitrypsin gene. The cultured cells produce biologically active human factor IX which is then isolated from the culture media.

Cell lines from a transgenic mouse which express biologically active IX

Factor VIII analog which has undergone deletion of amino acids 771 to 1666, prepared from eukaryotic cells transformed by an expression vector carrying the cDNA of the factor VIII which has undergone deletion.

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Strasbourg, France

Andrea Pavirani