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US 10,300,113 B2Granted
Targeted therapeutic proteins
AssigneeBiomarin Pharmaceutical Inc.
InventorsJonathan LeBowitz, Stephen M. Beverley
Life of this patent
Estimating the term…
Abstract
Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
Claims17
A formulation for parenteral administration comprising:
- a target therapeutic comprising a therapeutic agent that is therapeutically active in a mammalian lysosome and a variant of human IGF-II having an amino acid sequence at least 70% identical to mature human IGF-II (SEQ ID NO:8) that binds an extracellular domain of human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner;
- and a pharmaceutically acceptable carrier.
A formulation for parenteral administration comprising:
- a therapeutic fusion protein comprising a lysosomal enzyme and a lysosomal targeting domain comprising a mutein of human IGF-II having an amino acid sequence at least 70% identical to mature human IGF-II (SEQ ID NO:8) that binds human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
Dependencies are inferred from the claim text.