Methods and compositions for therapeutic protein delivery
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsAndrew Baik, Katherine Cygnar, Maria Praggastis
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Abstract
Compositions and methods for delivering a therapeutic protein to the central nervous system (CNS), in order to treat diseases and disorders that impair the CNS, such as treating lysosomal storage diseases are disclosed. Therapeutic proteins delivered via a therapeutically effective amount of a nucleotide composition encoding the therapeutic protein conjugated to a cell surface receptor-binding protein that crosses the blood brain barrier (BBB) are provided.
Claims48
A method of delivering a therapeutic protein to the central nervous system (CNS) of a patient, comprising delivering a nucleotide composition encoding a multidomain therapeutic protein to the liver in the patient to form a liver depot for the production and secretion of the multidomain therapeutic at a consistent serum level of at least 1 μg/mL over consecutive days, weeks or months following delivery to provide a therapeutically effective amount of the multidomain therapeutic protein in the CNS,
- wherein the multidomain therapeutic protein comprises an antibody or antigen-binding portion thereof that binds CD63 or ITGA7 and an enzyme domain.
A multidomain therapeutic protein comprising two or more delivery domains and an enzyme domain, wherein one of the two or more delivery domains binds human transferrin receptor (hTfR), and wherein an other of the two or more delivery domains comprises an antibody or antigen-binding portion thereof that binds CD63 or ITGA7.
A multidomain therapeutic protein comprising at least two delivery domains and at least one enzyme domain, wherein each of the at least two delivery domains is independently selected from the group consisting of an antibody, a half-body, and an scFv, and wherein one or more of the delivery domains is associated the at least one enzyme domain, preferably wherein the one or more delivery domains is covalently linked to the at least one enzyme domain.
46-65. (canceled)
Dependencies are inferred from the claim text.