Granted Patent
B2
US 12,188,058 · App. 17/188,994 · Granted Jan 7, 2025
Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
Inventors:
Erno Pungor (Novato, CA); Charles Hague (Novato, CA); Zhi Chen (Novato, CA); Melita Dvorak-Ewell (Berkeley, CA); Michel Claude Vellard (Novato, CA); Vish Koppaka (San Rafael, CA)
Assignee:
BIOMARIN PHARMACEUTICAL INC.
C12N9/16C12Q1/34C12Q1/44G01N33/6893A01K2217/075A01K2267/035A61K38/00C12Y301/06014G01N2333/924G01N2400/40G01N2500/04G01N2800/04
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Abstract
This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.
Claims (1)
1. A method of treating a human subject suffering from Metachromatic Leukodystrophy (MLD) comprising administering to the subject in need of said lysosomal sulfatase enzyme an effective amount of said lysosomal sulfatase enzyme, wherein said lysosomal sulfatase enzyme is a recombinant human arylsulfatase A (ARSA) enzyme comprising at least 0.5 bis-phosphorylated oligomannose chains per protein chain and is produced by a CHO-derived END3 complementation group cell.
Assignments (2)
SECURITY INTEREST
Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Aug 19, 2021
From: DVORAK-EWELL, MELITA; VELLARD, MICHEL CLAUDE; KOPPAKA, VISH
To: BIOMARIN PHARMACEUTICAL INC.
Reel/Frame 057223/0892 →
Continuity (10)
Provisional Application
61110246
· Oct 31, 2008
Provisional Application
61099373
· Sep 23, 2008
Provisional Application
61022179
· Jan 18, 2008
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