Claims for Patent: 8,236,300

Summary for Patent: 8,236,300

Title:	In vivo and ex vivo gene transfer into renal tissue using gutless adenovirus vectors
Abstract:	A method for treating a renal disease in a subject is disclosed. The method includes administering into a kidney of the subject with an effective amount of a gutless adenoviral vector containing a polynucleotide encoding a therapeutic agent. The gutless adenoviral vector contains the nucleotide sequence of SEQ ID NO:13 or SEQ ID NO:15 and expresses the therapeutic agent in a kidney tissue of the subject.
Inventor(s):	Sehgal; Lakshman R. (Monarch Beach, CA), Wong; Jonathan (Palot Alto, CA)
Assignee:	Biovec, LLC (Chicago, IL)
Application Number:	13/023,322
Patent Claims:	1. A gutless adenovirus vector, comprising a polynucleotide encoding a therapeutic agent; a renal-specific regulatory element operably linked to said polynucleotide sequence; and a stuffer comprising the nucleotide sequence of SEQ ID NO:13 or SEQ ID NO:15, wherein said therapeutic agent is thrombomodulin. 2. The gutless adenovirus vector of claim 1, wherein said renal-specific regulatory element is selected from the group consisting of high-capacity (type 2) Na.sup.+/glucose cotransporter gene (Sglt2) promoter, Ksp-cadherin promoter, ClC-K1 chloride channel gene promoter, uromodulin promoter, Nkcc2/Slc12a1 gene promoter, and the p1 promoter of the parathyroid hormone (PTH)/PTH-related peptide receptor gene. 3. The gutless adenovirus vector of claim 1, wherein said stuffer comprises the nucleotide sequences of SEQ ID NO:13 and SEQ ID NO:15. 4. The gutless adenovirus vector of claim 1, wherein said thrombomodulin is human thrombomodulin or a variant thereof. 5. The gutless adenovirus vector of claim 4, wherein said thrombomodulin is human thrombomodulin. 6. The gutless adenovirus vector of claim 2, wherein said renal-specific regulatory element is high-capacity (type 2) Na.sup.+/glucose cotransporter gene (Sglt2) promoter. 7. The gutless adenovirus vector of claim 2, wherein said renal-specific regulatory element is Ksp-cadherin promoter. 8. The gutless adenovirus vector of claim 2, wherein said renal-specific regulatory element is ClC-K1 chloride channel gene promoter. 9. The gutless adenovirus vector of claim 2, wherein said renal-specific regulatory element is uromodulin promoter. 10. The gutless adenovirus vector of claim 2, wherein said renal-specific regulatory element is Nkcc2/Slc12a1 gene promoter. 11. The gutless adenovirus vector of claim 2, wherein said renal-specific regulatory element is the p1 promoter of the parathyroid hormone (PTH)/PTH-related peptide receptor gene.

Details for Patent 8,236,300

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Nps Pharmaceuticals, Inc.	NATPARA	parathyroid hormone	For Injection	125511	01/23/2015		2022-12-02