WO2024176112 - LARGE SCALE PRODUCTION OF RECOMBINANT ARYLSULFATASE A AND COMPOSITIONS THEREOF

National phase entry is expected:
Publication Number WO/2024/176112
Publication Date 29.08.2024
International Application No. PCT/IB2024/051618
International Filing Date 20.02.2024
Title **
[English] LARGE SCALE PRODUCTION OF RECOMBINANT ARYLSULFATASE A AND COMPOSITIONS THEREOF
[French] PRODUCTION À GRANDE ÉCHELLE D'ARYLSULFATASE A RECOMBINANTE ET COMPOSITIONS ASSOCIÉES
Applicants **
TAKEDA PHARMACEUTICAL COMPANY LIMITED
Inventors
TCHOUDAKOVA, Anna
KEEFE, Andrew
BARTON, Christopher
GRAESSLE, Evan
LU, Wei
YANG, Lan
FLAHERTY, Brian
YAN, Gang
Priority Data
63/486,166   21.02.2023   US
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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting4883
EPO Filing, Examination, Granting80628
Japan Filing, Examination, Granting4591
South Korea Filing, Examination, Granting6948
USA Filing, Examination, Granting20390
MasterCard Visa
Total: 117,440

The term for entry into the National Phase has expired. This quotation is for informational purposes only

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Abstract[English] The present invention provides, among other things, improved large-scale methods and compositions comprising recombinant arylsulfatase A (ASA) protein for enzyme replacement therapy, for example in treating Metachromatic Leukodystrophy Disease (MLD). In one aspect, the method of the present invention is for large-scale production of highly pure recombinant ASA with high cellular uptake, lysosomal targeting and bioactivity.[French] La présente invention concerne, entre autres, des procédés et des compositions améliorés, à grande échelle, comprenant une protéine d'arylsulfatase A (ASA) recombinante pour une enzymothérapie de substitution, par exemple dans le traitement de la leucodystrophie métachromatique (LDM). Dans un aspect, le procédé selon présente invention sert à la production à grande échelle d'ASA recombinante haute pureté dotée d'une absorption cellulaire, d'un ciblage lysosomal et d'une bioactivité élevés.