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Compositions and methods for the treatment of hemophilia a

a technology of hemophilia and composition, applied in the field of medicine and gene therapy, can solve problems such as compromised vital organs

Inactive Publication Date: 2007-07-19
THE CHILDRENS HOSPITAL OF PHILADELPHIA
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The present invention provides variant Factor VIII (FVIII) molecules with higher specific activity than native molecules. These molecules can be used in gene therapy for the treatment of Hemophilia A. The variant polypeptides are expressed in a recombinant system, isolated and purified, and formulated into a pharmaceutical composition for administration to patients in need. The methods described in the patent text allow for the treatment of Hemophilia A using the variant FVIII molecules.

Problems solved by technology

Severely affected patients are prone to serious hemorrhages that may dissect through tissue planes, ultimately resulting in death due to compromised vital organs.

Method used

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  • Compositions and methods for the treatment of hemophilia a
  • Compositions and methods for the treatment of hemophilia a
  • Compositions and methods for the treatment of hemophilia a

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example i

Novel Minigene Encoding Improved Factor VIII

[0084] In accordance with the present invention, new recombinant Factor VIII minigenes are provided which stimulate production of Factor VIII in cells comprising the minigene with higher activity than prior art recombinant constructs.

[0085] Native FVIII is synthesized as a single chain polypeptide (2332 amino acids) preceded by a 19-residue signal sequence and has a molecular weight of Mr=330,000. The signal sequence is removed upon translocation of FVIII into the ER and the native FVIII is then cleaved in the B-domain in connection with its secretion. This results in the release of a heterodimer comprised of a Mr=200,000 heavy chain and a Mr=80,000 light chain, the association of which is metal-ion dependent. See FIG. 1.

Creation of the FVIII Minigenes:

[0086] The FVIII cDNA in the expression plasmid pSP64 was purchased from the ATCC. Following a Sal I restriction digest, the FVIII cDNA was subcloned into the mammalian expression plasm...

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Abstract

Improved materials and methods for the treatment of Hemophilia A are provided.

Description

CROSS REFERENCE TO RELATED APPLICATIONS [0001] This application claims priority under 35 U.S.C. §119(e) to U.S. Provisional Patent Application No. 60 / 382,486 filed on May 22, 2002, the entire disclosure of which is incorporated by reference herein.GOVERNMENT RIGHTS [0002] Pursuant to 35 U.S.C. §202(c), it is acknowledged that the U.S. Government has certain rights in the invention described herein, which was made in part with funds from the National Institutes of Health, Grant Numbers: HL48322 and T32HL07439.FIELD OF THE INVENTION [0003] This invention relates to the fields of medicine and gene therapy. More specifically, the invention provides materials and methods for the restoring factor VIII activity in patients in need thereof. BACKGROUND OF THE INVENTION [0004] Several publications and patent documents are referenced in this application in order to more fully describe the state of the art to which this invention pertains. Full citations for these references are found at the en...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): A61K48/00C07H21/04C07K14/755A61K38/00
CPCA61K38/00C07K14/755A61K48/00
Inventor HIGH, KATHERINECAMIRE, RODNEY
Owner THE CHILDRENS HOSPITAL OF PHILADELPHIA
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