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US 2025/0136671 A1Published application
Therapies for mucopolysaccharidosis type 6
AssigneesSigilon Therapeutics, Inc., National Research Council of Canada
InventorsElina Makino, Erika Pearson, Gregory Hussack
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Abstract
Described herein are ARSB fusion proteins, polynucleotides encoding the fusion proteins and mammalian cells genetically modified to express and secrete the fusion proteins, as well as compositions, implantable devices and device preparations comprising the fusion proteins or genetically modified cells secreting the fusion proteins, and methods of making and using the same for treating MPS-6.
Claims55
A fusion protein which comprises an N-terminal to C-terminal structure defined by formula (F):
- AB-L-ARSB, wherein:
- AB comprises a domain that binds to human serum albumin (HSA);
- L, which may be present or absent, comprises a linker amino acid sequence;
- and
- ARSB comprises an amino acid sequence for a mature mammalian ARSB protein.
2 more elements
Dependencies are inferred from the claim text.