Compositions, devices, and methods for treating cns disorders
AssigneeNational Research Council of Canada
InventorsElina Makino, Erika Pearson, Gregory Hussack, Danica Stanimirovic
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Abstract
Described herein are BBB-transporting 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 various CNS diseases and conditions, e.g., any mucopolysaccharidosis type with CNS manifestations.
Claims50
A fusion protein which comprises an N-terminal to C-terminal structure defined by formula I:
- AB-L1-RB-L2-C or formula II:
- RB-L1-AB-L2-C,
- wherein for each of formula I and II:
- AB comprises a domain that binds to human serum albumin (HSA);
- L1, which may be present or absent, comprises a first linker amino acid sequence;
- RB comprises a domain that binds to the extracellular domain of human IGF1R (hIGF1R);
- L2, which may be present or absent, comprises a second linker amino acid sequence that is the same or different than the first linker amino acid sequence;
- and
- C is a cargo moiety, optionally the cargo moiety comprises, consists essentially of, or consists of, the amino acid sequence of a polypeptide.
Dependencies are inferred from the claim text.