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Methods and compositions for increasing α-L-iduronidase activity in the CNS

  • US 9,567,400 B2
  • Filed: 01/27/2015
  • Issued: 02/14/2017
  • Est. Priority Date: 07/27/2007
  • Status: Active Grant
First Claim
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1. A method of treating a central nervous system α

  • -L-iduronidase deficiency in a subject in need thereof, comprising systemically administering to the subject in need thereof a therapeutically effective dose of a fusion antibody having α

    -L-iduronidase activity, wherein;

    (a) at least about 25,000 units of α

    -L-iduronidase activity are delivered to a brain tissue of the subject in need thereof;

    (b) the therapeutically effective dose of a fusion antibody having α

    -L-iduronidase activity comprises at least about 200,000 units of α

    -L-iduronidase activity/Kg of body weight;

    (c) the fusion antibody having α

    -L-iduronidase activity comprises a fusion protein comprising an amino acid sequence of an immunoglobulin heavy chain and an α

    -L-iduronidase;

    (d) the fusion antibody having α

    -L-iduronidase activity binds to an endogenous receptor of a blood brain barrier (BBB) transport system; and

    (e) the amino acid sequence of the α

    -L-iduronidase is covalently linked at its amino terminus to the carboxy terminus of the amino acid sequence of the immunoglobulin heavy chain,wherein the α

    -L-iduronidase retains at least 30% of its enzymatic activity compared to an unfused α

    -L-iduronidase.

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