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US 6,585,971 B1Granted
Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
AssigneeHarbor-UCLA Research and Education Institute
InventorEmil D. Kakkis
Life of this patent
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Abstract
The present invention provides a recombinant human α-L-iduronidase and biologically active fragments and mutants thereof, large scale methods to produce and purify commercial grade recombinant human α-L-iduronidase enzyme as well as methods to treat certain genetic disorders including α-L-iduronidase deficiency and mucopolysaccharidosis I (MPS 1).
Claims30
A method of treating diseases caused all or in part by a deficiency in α-L-iduronidase, comprising the steps of:
- (a) administering a pharmaceutical composition comprising a purified human recombinant α-L-iduronidase having a purity of greater than about 99%, wherein said purified human recombinant α-L-iduronidase comprises the amino acid of residue 26 to 653 of SEQ ID NO:2, to a human subject in need thereof, and
- (b) optimizing said treatment by evaluating biochemical and clinical symptoms of said subject through routine assessment of history, physical examination, echocardiography, electrocardiography, magnetic resonance imaging, polysomnography, skeletal survey, range of motion measurements, corneal photographs, and skin biopsy.
A method of treating diseases caused all or in part by a deficiency in α-L-iduronidase, comprising the steps of:
- administering a pharmaceutical composition to a human subject in need thereof;
- wherein said pharmaceutical composition comprises a purified human recombinant α-L-iduronidase having a purity of greater than about 99%, wherein said purified human recombinant α-L-iduronidase comprises the amino acid of residue 26 to 653 of SEQ ID NO:2.
Dependencies are inferred from the claim text.