US2023203465A1PendingUtilityA1

Highly Potent Acid Alpha-Glucosidase With Enhanced Carbohydrates

84
Assignee: AMICUS THERAPEUTICS INCPriority: Sep 30, 2014Filed: Feb 17, 2023Published: Jun 29, 2023
Est. expirySep 30, 2034(~8.2 yrs left)· nominal 20-yr term from priority
Inventors:Russell GotschallHung V. Do
A61K 47/26A61K 9/19A61K 38/47C12N 9/2402C12N 15/09C12N 2510/02A61K 48/005C12Y 302/0102C12N 9/2465A61P 3/08A61P 43/00
84
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Claims

Abstract

Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides. Compositions containing the rhGAA, and methods of use are described.

Claims

exact text as granted — not AI-modified
1 . A composition comprising recombinant human acid alpha-glucosidase (rhGAA), wherein 40%-60% of the N-glycans on the rhGAA are complex type N-glycans, and wherein the rhGAA comprises at least 3.0 mol mannose-6-phosphate (M6P) residues per mol rhGAA. 
     
     
         2 . The composition of  claim 1 , wherein the rhGAA comprises at least 4.0 mol M6P residues per mol rhGAA. 
     
     
         3 . The composition of  claim 1 , wherein the rhGAA comprises at least 5.0 mol M6P residues per mol rhGAA. 
     
     
         4 . The composition of  claim 1 , wherein the rhGAA comprises at least 7.0 mol M6P residues per mol rhGAA. 
     
     
         5 . The composition of  claim 1 , wherein the rhGAA comprises from 3.0 mol to 7.0 mol M6P residues per mol rhGAA. 
     
     
         6 . A composition comprising rhGAA, wherein 40%-60% of the N-glycans on the rhGAA are complex type N-glycans, and wherein at least 2% of the total glycans on the rhGAA are bis-phosphorylated mannose-6-phosphate (bis-M6P) glycans. 
     
     
         7 . The composition of  claim 6 , wherein at least 2.5% of the total glycans on the rhGAA are bis-M6P glycans. 
     
     
         8 . The composition of  claim 6 , wherein at least 3.0% of the total glycans on the rhGAA are bis-M6P glycans. 
     
     
         9 . The composition of  claim 6 , wherein at least 17% of the total glycans on the rhGAA are bis-M6P glycans. 
     
     
         10 . (canceled) 
     
     
         11 . A composition comprising recombinant human acid alpha-glucosidase (rhGAA), wherein the rhGAA comprises at least 6.0 mol M6P residues per mol rhGAA. 
     
     
         12 . The composition of  claim 11 , wherein the rhGAA comprises at least 7.0 mol M6P residues per mol rhGAA.

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