Treatment of alpha-galactosidase a deficiency
AssigneeShire Human Genetic Therapies, Inc.
InventorsDouglas A. Treco, Kenneth Loveday, Marianne Borowski
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Abstract
The invention provides methods of treating α-galactosidase A deficiency. Dosage forms, methods of administration, and methods of analyzing human α-galactosidase A are also included.
Claims20
A kit for the treatment of α-Gal A deficiency, comprising:
- (a) a human α-Gal A glycoprotein preparation;
- and (b) instructions to administer the preparation to a subject in need thereof, wherein the serum clearance of the α-Gal A preparation from subject's circulation is less than 4 mL/min/kg on the linear portion of the AUC vs. dose curve, wherein the α-Gal A glycoprotein preparation has the following four characteristics:
- (1) has less than 35% tri- and tetra-sialylated glycans combined;
- (2) has greater than 50% complex glycans;
- (3) has less than 45% phosphorylated glycans;
- and
- (4) has a ratio of sialylated glycans to phosphorylated glycans greater than 1.
A kit for the treatment of α-Gal A deficiency, comprising:
- a human α-Gal A glycoprotein preparation and instructions to administer the preparation to a subject in need thereof at a unit dose of less than 1.0 mg per kilogram of body weight of the subject, wherein the α-Gal A glycoprotein preparation has the following four characteristics:
- (1) has less than 35% tri- and tetra-sialylated glycans combined;
- (2) has greater than 50% complex glycans;
- (3) has less than 45% phosphorylated glycans;
- and
- (4) has a ratio of sialylated glycans to phosphorylated glycans greater than 1.
A kit for the treatment of α-Gal A deficiency, comprising a human α-Gal A glycoprotein preparation and instructions to administer the preparation to a subject in need thereof no more than once every 8 weeks, wherein the α-Gal A glycoprotein preparation has the following four characteristics:
- (1) has less than 35% tri- and tetra-sialylated glycans combined;
- (2) has greater than 50% complex glycans;
- (3) has less than 45% phosphorylated glycans;
- and
- (4) has a ratio of sialylated glycans to phosphorylated glycans greater than 1.
Dependencies are inferred from the claim text.