Avigen, Inc.

Genzyme Corporation

Xiaobing Qian

Peter C Colosi

Linda B Couto

Linda Couto

One form of a composition has two types of recombinant adeno-associated virus. The first type encodes a portion of Factor VIII operably linked to an expression control element; and the second type encodes a different portion of Factor VIII operably linked to an expression control element. The first and second nucleotide sequences collectively encode a functional Factor VIII protein. Another form of the composition is a recombinant adeno-associated virus containing a nucleotide sequence encoding functional Factor VIII light or heavy chain operably linked to a tissue-specific promoter.

Adeno-associated vector compositions for expression of Factor VIII

The present invention provides improved viral vectors useful for the expression of genes at high levels in human cells. In particular, the present invention provides adeno-associated vectors (AAV) suitable for gene therapy. These vectors are capable of delivering nucleic acid containing constructs which result in the production of full-length therapeutic levels of biologically active Factor VIII in the recipient individual in vivo. The present invention also provides pharmaceutical compositions comprising such AAV vectors, as well as methods for making and using these constructs.

Adeno-associated vectors for expression of factor VIII by target cells

The present invention provides improved viral vectors useful for the expression of genes at high levels in human cells. In particular, the present invention provides recombinant adeno-associated vectors (AAV) suitable for gene therapy. These vectors are capable of delivering nucleic acid containing constructs which result in the production of full-length therapeutic levels of biologically active Factor VIII in the recipient individual in vivo. The present invention also provides pharmaceutical compositions comprising such AAV vectors, as well as methods for making and using these constructs.

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Alameda, CA, United States of America

Xiaobing Qian