Lysosomal enzymes modified by cell based glycoengineering
AssigneesUniversity of Copenhagen, Glycodisplay Aps
InventorsClaus KRISTENSEN, Weihua TIAN, Henrik CLAUSEN, Zhang YANG, Sergey VAKHRUSHEV
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Abstract
The present invention relates to lysosomal enzymes modified by use of cell based methods, a compositions comprising a modified lysosomal enzyme, as well as methods for producing a modified lysosomal enzyme and therapeutic use of such modified lysosomal enzyme. In particular, the present disclosure relates to a modified lysosomal enzyme which has low Man6P and low exposed Mannose and high sialic acid content of alpha2,3 type enabling long circulation time and improved uptake into difficult-to-reach organs like heart, kidney and brain.
Claims17
A modified recombinant lysosomal enzyme with increased circulation time in plasma as compared to an unmodified version of the same, wherein said enzyme comprises low mannose-6-phosphate (Man6P) and high sialic acid (SA) capping of alpha2,3 type (alpha2,3SA).
2-132. (canceled)
The method of claim 15, wherein the lysosomal storage disease is selected from Aspartylglucoaminouria (AGU), Fabry, Farber, Fucosidosis, Galactosidosialidosis, Gaucher types 1,2, and 3. G-M1 gangliosidosis, Hunter, Hurler-Scheie, Krabbe, Alpha-Mannosidosis (Laman), Beta-Mannosidosis, Maroteaux-Lamy, Metachromatic, Morquio B, Mucolipidosis II/III, and Pompe.
Dependencies are inferred from the claim text.