32 results about "Iduronate-2-sulfatase" patented technology

Provided herein are methods and compositions for treating a subject suffering from a deficiency in iduronate 2-sulfatase in the CNS. The methods include systemic administration of a bifunctional fusion antibody comprising an antibody that crosses the blood brain barrier (BBB) and an iduronate 2-sulfatase.

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.

Disclosed is a modified iduronate-2-sulfatase (IDS) gene constructed by inserting the nucleotide of SEQ ID NO: 2 into a wild-type IDS gene. In addition to being negatively charged, the improved IDS enzyme encoded by the modified gene exhibits a sufficient retention time in blood to target the bone, so that it is more effective for treating Hunter syndrome.

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.

The present invention provides, among other things, compositions, kits and methods for subcutaneous delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention provides methods for treating Hunter syndrome by subcutaneous administration of a replacement iduronate-2-sulfatase (I2S) protein. In some embodiments, the present invention provides a kit comprising an arrangement of components for subcutaneously administering iduronate-2-sulfatase (I2S) protein.

Disclosed is a composition comprising recombinant iduronate-2-sulfatase (IDS). The glycosylation pattern and formylglycine content of the IDS composition are different from those of Elaprase and have superior pharmaceutical efficacy and are safer than the conventional agent and thus can be effectively used for the therapy of Hunter syndrome.

The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant I2S protein can be purified from unprocessed biological materials, such as, I2S-containing cell culture medium, using a process involving as few as four chromatography columns.

The invention discloses a production method of escherichia coli expressed human iduronate-2-sulfatase. The method employs a 5L fermentation tank of escherichia coli recombinant bacteria for high-density culture and induction expression, the fermentation broth subjected to induction expression is centrifuged to collect thalli, high-pressure homogenization technique is employed to crush the recombinant bacteria to obtain a recombinant protein inclusion body, the recombinant protein inclusion body is dissolved and is then loaded to a Ni column for affinity chromatography purification, and after desalination and freeze-drying, the recombinant human iduronate-2-sulfatase can be obtained. The method provided by the invention simplifies the tedious washing steps of the inclusion body, explores appropriate ultrasonic conditions to transform insoluble inclusion body protein into soluble protein, avoids the denaturation and renaturation operations on the inclusion body brought about by use of 8Murea, 6M guanidine hydrochloride or other strong denaturing agents, and can achieve a protein purity of 95% or more and a recovery efficiency of 70% or above by Ni column affinity chromatography, thus reaching the effect of simple, effective and rapid purification.

The present invention is related to a compound that includes a lysosomal enzyme and a targeting moiety, for example, a compound that includes iduronate-2-sulfatase conjugated to Angiopep-2 through a linker formed by specific click chemistry reactions. In certain embodiments, these compounds, owing to the presence of the targeting moiety, can cross the blood-brain barrier or accumulate in the lysosome more effectively than the enzyme alone. The invention also features pharmaceutical compositions containing such compounds and methods for treating lysosomal storage disorders (e.g., mucopolysaccharidosis Type II) using such compounds.

The present invention relates to a transgenic mouse model for developing enzyme replacement therapy for iduronate-2-sulfatase deficiency syndrome, for example, Hunter syndrome. More specifically, the present invention relates to a transgenic mouse to be used for developing enzyme replacement therapy for iduronate-2-sulfatase, wherein the immune response against injected enzyme, such as, recombinant iduronate-2-sulfatase has been minimized in transgenic mouse model in the course of treating in vivo iduronate-2-sulfatase replacement.

Provided is a modified iduronate-2-sulfatase (IDS) gene constructed by inserting the nucleotide of SEQ ID NO: 2 into a wild-type IDS gene. In addition to being negatively charged, the improved IDS enzyme encoded by the modified gene exhibits a sufficient retention time in blood to target the bone, so that it is more effective for treating Hunter syndrome.