Claims for Patent: 9,198,595

Summary for Patent: 9,198,595

Title:	Reagents and methods for modulating cone photoreceptor activity
Abstract:	The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.
Inventor(s):	Neitz; Jay (Seattle, WA), Neitz; Maureen (Seattle, WA), Kuchenbecker; James A. (Seattle, WA), Hauswirth; William W. (Gainesville, FL)
Assignee:	University of Washington Through its Center for Commercialization (Seattle, WA) University of Florida Research Foundation, Incorporated (Gainesville, FL) Medical College of Wisconsin (Milwaukee, WI)
Application Number:	14/075,415
Patent Claims:	1. A method for cone cell gene therapy in a primate, comprising (I) administering to the eye of a primate having color blindness a recombinant gene delivery vector comprising (a) a promoter region, wherein the promoter region is specific for retinal cone cells; and (b) a gene encoding a therapeutic selected from the group consisting of S-Opsin, M-Opsin and L-Opsin, wherein the gene is operatively linked to the promoter region; and (II) testing the eye of the primate for discrimination of blue-green colors against a red-violet background; wherein in vivo expression of the therapeutic in cone cells of the primate results in the primate being able to visualize and to discriminate between red and green colors. 2. The method of claim 1 wherein the primate is of the Parvorder Catarrhini. 3. The method of claim 1 wherein the promoter comprises a sequence selected from the group consisting of the L opsin promoter (SEQ ID NO: 1), the M opsin promoter (SEQ ID NO: 2), and the S opsin promoter (SEQ ID NO: 3). 4. The method of claim 1, wherein the gene delivery vector further comprises an enhancer element upstream of the promoter, wherein the gene is operatively linked to the enhancer element. 5. The method of claim 4, the enhancer element is specific for primate retinal cone cells. 6. The method of claim 5, wherein the enhancer element comprises the nucleic acid sequence of SEQ ID NO: 51. 7. The method of claim 1, wherein the gene delivery vector further comprises an intron comprising a splice donor/acceptor region, wherein the intron is located downstream of the promoter region and is located upstream of the gene. 8. The method of claim 1, wherein the gene delivery vector comprises a recombinant adeno-associated virus (AAV) gene delivery vector. 9. The method of claim 8, wherein the AAV gene vector is pseudotyped for AAV2. 10. The method of claim 1, wherein the gene encodes one or more therapeutic polypeptides comprising a sequence selected from the group consisting of SEQ ID NO: 7, SEQ ID NO: 9, SEQ ID NO: 11; and a polymorph of SEQ ID NO: 11 selected from the group consisting of (i) Thr65Ile (ii) Ile111Val (iii) Ser116Tyr (iv) Leu153Met (v) Ile171Val (vi) Ala174Val (vii) Ile178Val (viii) Ser180Ala (ix) Ile230Thr (x) Ala233Ser (xi) Val236Met (xii) Ile274Val (xiii) Phe275Leu (xiv) Tyr277Phe (xv) Val279Phe (xvi) Thr285Ala (xvii) Pro298Ala; and (xviii) Tyr309Phe. 11. The method of claim 1, wherein the photoreceptors of the primate are healthy. 12. The method of claim 1, wherein the primate is an adult primate.

Details for Patent 9,198,595

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		2029-09-15
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132			2029-09-15
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132			2029-09-15