Claims for Patent: 8,367,056

Summary for Patent: 8,367,056

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:	13/023,314
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 vector backbone comprising the nucleotide sequence of SEQ ID NO:23, except that a thrombomodulin expression cassette containing a Fse-SbfI fragment has been removed from SEQ ID NO:23, and wherein said therapeutic agent is an iRNA agent or an antisense RNA. 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. A pharmaceutical composition, comprising: a gutless adenovirus vector comprising: a polynucleotide encoding an iRNA agent or an antisense; a renal-specific regulatory element operably linked to said polynucleotide sequence; a vector backbone comprising the nucleotide sequence of SEQ ID NO:23, except that a thrombomodulin expression cassette containing a Fse-SbfI fragment has been removed from SEQ ID NO:23; and a pharmaceutically acceptable carrier. 4. The pharmaceutical composition of claim 3, 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. 5. The pharmaceutical composition of claim 3, wherein said iRNA agent or an antisense RNA inhibits TGF-.beta.1 expression. 6. The pharmaceutical composition of claim 3, wherein said shRNA inhibits platelet-derived growth factor expression. 7. A gutless adenovirus vector, comprising: a polynucleotide encoding a therapeutic agent; a renal-specific regulatory element operably linked to said polynucleotide sequence, wherein said renal-specific regulatory element is selected from the group consisting of high-capacity (type 2) Na.sup.+/glucose cotransporter gene (Sglt2) promoter, and Nkcc2/Slc12a1 gene promoter; and a vector backbone comprising the nucleotide sequence of SEQ ID NO:23, except that a thrombomodulin expression cassette containing a Fse-SbfI fragment has been removed from SEQ ID NO:23, wherein said therapeutic agent is an iRNA agent or an antisense RNA. 8. A pharmaceutical composition comprising: the gutless adenovirus vector of claim 7, and a pharmaceutically acceptable carrier.

Details for Patent 8,367,056

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