Granted Patent
B2
US 12,565,643 · App. 17/640,246 · Granted Mar 3, 2026
Alpha-galactosidase protein for enzyme replacement therapy (ERT) and methods of use
Assignee:
The Medical College of Wisconsin, Inc.
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Abstract
The present disclosure provides less immunogenic mutant α-galactosidase A protein (α-gal), methods of making and methods of use. The less immunogenic mutant α-gal of the present invention provides a reduced immune response when administered to a subject.
Claims (5)
1 . A virus comprising an expression construct comprising a nucleic acid sequence encoding a mutant α-galactosidase A protein (α-gal), wherein the mutant α-gal comprises a S62N amino acid change, wherein the amino acid change is relative to SEQ ID NO: 1, wherein the mutant a-gal has at least 98% identity to SEQ ID NO: 1, and wherein the virus is a retrovirus, adenovirus, or adeno-associated virus.
2 . A host cell comprising the virus of claim 1 .
3 . The virus of claim 1 , wherein the mutant α-gal consists of a S62N amino acid change, relative to SEQ ID NO: 1.
4 . The host cell of claim 2 , wherein the cell is a CD34+ stem cell or a T cell.
5 . The virus of claim 1 , wherein the mutant α-gal further comprises one or more amino acid change selected from the group consisting of E58N, E59N, D61N, E66P, M70A, M70R, M70N, M70D, M70G, M70H, M70Q, M701, M70L, M70K, M70P, S78D, S78E, S78V, E79A, E79N, E79D, E79G, D83E, A84I, A84V, Y86L, R100F, D101N, D101E, A309N, A318G, and P323R.
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Mar 4, 2022
From: MEDIN, JEFFREY A.; SCALIA, SIMONE
To: THE MEDICAL COLLEGE OF WISCONSIN, INC.
Reel/Frame 059176/0458 →
Continuity (2)
Provisional Application
62902211
· Sep 18, 2019
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