Methods for treating diseases caused by deficiencies of recombinant alpha-L-iduronidase
OwnerEmil D. Kakkisinventor
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Abstract
The present invention provides a formulation comprising a pharmaceutical composition comprising a human recombinant α-L-iduronidase or biologically active or muteins thereof with a purity of greater than 99%, or in combination with a pharmaceutically acceptable carrier. The present invention further provides methods to treat certain genetic disorders including α-L-iduronidase deficiency and mucopolysaccharidosis I (MPS 1) by administering said formulation.
Claims46
A method of treating a human disease caused all or in part by a deficiency in α-L-iduronidase, comprising the steps of:
- (a) administering a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a human recombinant α-L-iduronidase of SEQ ID NO:2, or a biologically active fragment of SEQ ID NO:2 which possesses the same or similar biological activity to SEQ ID NO:2, wherein said human recombinant α-L-iduronidase of SEQ ID NO:2, or the biologically active fragment thereof has a purity of greater than 99%, to a human subject in need thereof;
- (b) optimizing said treatment by assessment of primary efficacy endpoints;
- (c) optimizing said treatment by assessment of secondary efficacy endpoints;
- (d) optimizing said treatment by assessment of tertiary efficacy endpoints;
- and
- (e) optimizing treatment by assessment of safety endpoints.
A method of treating a human disease caused all or in part by a deficiency in α-L-iduronidase, comprising administering to a subject presenting the symptoms of said disease a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a purified human recombinant α-L-iduronidase of SEQ ID NO:2, or a biologically active fragment of SEQ ID NO:2 which possesses the same or similar biological activity of a human recombinant α-L-iduronidase of SEQ ID NO:2, wherein said human recombinant α-L-iduronidase of SEQ ID NO:2, or biologically active fragment thereof in said pharmaceutical composition has a purity of about 99% or greater, in an amount effective to alleviate the symptoms of said deficiency in α-L-iduronidase.
A method of treating a human disease caused all or in part by a deficiency in α-L-iduronidase, comprising administering to a human subject in need thereof a pharmaceutical composition comprising a purified human recombinant α-L-iduronidase of SEQ ID NO:2 and a pharmaceutically acceptable carrier, wherein said human recombinant α-L-iduronidase of SEQ ID NO:2 has a purity of about 99% or greater.
A method of treating human diseases caused all or in part by a deficiency in α-L-iduronidase, comprising the steps of:
- (a) administering a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a purified human recombinant α-L-iduronidase of SEQ ID NO:2, or biologically active fragment of SEQ ID NO:2 which possesses the same or similar biological activity to SEQ ID NO:2, wherein said human recombinant α-L-iduronidase of SEQ ID NO:2, biologically active fragment thereof has a purity of greater than about 99%, 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, or 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 of SEQ ID NO:2, or biologically active fragment of SEQ ID NO:2 which possesses the same or similar biological activity to SEQ ID NO:2 wherein said human recombinant α-L-iduronidase of SEQ ID NO:2, or biologically active fragment has a purity of greater than about 99%.
Dependencies are inferred from the claim text.