Claims for Patent: 6,093,392

Summary for Patent: 6,093,392

Title:	Methods and compositions for use in gene therapy for treatment of hemophilia
Abstract:	The invention includes a composition comprising a recombinant adeno-associated viral vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5\' and 3\' untranslated regions and a transcription termination signal, and methods of use thereof.
Inventor(s):	High; Katherine A. (Merion, PA), Herzog; Roland W. (Glenolden, PA)
Assignee:	Childrens Hospital of Phildelphia (Philadelphia, PA)
Application Number:	09/038,910
Patent Claims:	1. A method of treating hemophilia in a mammal comprising: (a) providing a recombinant adeno-associated virus vector (rAAV), said rAAV comprising a nucleic acid encoding Factor IX operably linked to an expression control element; and (b) administering an amount of said rAAV to a mammal wherein said Factor IX is expressed at levels having a therapeutic effect on said mammal and wherein said therapeutic effect is an increase in coagulation of blood. 2. The method of claim 1, wherein said recombinant adeno-associated virus vector is administered by injecting said vector into at least two sites in the mammal. 3. The method of claim 1, wherein said recombinant adeno-virus vector is administered at a dose of between about 1.times.10.sup.8 to about 5.times.10.sup.16 viral vector genomes per mammal. 4. The method of claim 1, wherein said mammal is a human and said Factor IX is a human Factor IX. 5. The method of claim 1, wherein said expression control element is selected from the group consisting of a cytomegalovirus immediate early promoter/enhancer, a skeletal muscle actin promoter and a muscle creatine kinase promoter/enhancer. 6. The method of claim 1, wherein said vector further comprises a portion of intron I of a Factor IX gene. 7. The method of claim 1, wherein said nucleic acid encoding Factor IX comprises a mutation which reduces binding of Factor IX encoded thereby to collagen IV as compared to a Factor IX lacking the mutation, wherein the mutation replaces a lysine residue with an alanine residue in the fifth amino acid position from the beginning of mature Factor IX. 8. The method of claim 1, wherein said mammal is a human. 9. The method of claim 1, wherein the administering is to a muscle tissue of the mammal. 10. The method of claim 2, wherein said recombinant adeno-associated virus vector is administered by injecting said vector into at least six sites in the mammal. 11. The method of claim 6, wherein said portion of intron I of a Factor IX gene is from about 0.3 kb to about 1.7 kb.

Details for Patent 6,093,392

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132	06/04/1986		2017-03-14
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132			2017-03-14
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132			2017-03-14