19 results about "Enzyme replacement therapy" patented technology

The present disclosure features methods for treating neutralizing antibodies that reduce the efficacy of soluble alkaline phosphatase therapy (e.g., asfoenzyme alpha), which is an enzyme replacement therapy, such as for the treatment of bone mineralization disorders, e.g., hypophosphoesterase (HPP). The methods include diagnosing the subject for the presence of a neutralizing antibody that affects the efficacy of the treatment, administering a treatment suitable for reducing the deleterious effects of the neutralizing antibody, and continuing an alkaline phosphatase treatment.

The invention provides application of a flavin monooxygenase 3 inhibitor in preparation of a medicine for preventing and / or treating acute pancreatitis, and belongs to the technical field of medicines. The flavin monooxygenase 3 inhibitor is methimazole or a pharmaceutically acceptable salt, a prodrug or a derivative of methimazole. Experimental results show that after the methimazole is orally taken, the nidus area of animal pancreatitis is obviously reduced, the pathological score is obviously improved, the in-vivo inflammation level is obviously inhibited, and the blood amylase and lipase levels are obviously reduced; the levels of pancreatitis factors and chemotactic factors are reduced, and the oxidative stress index is reduced. In addition, by combining methimazole with the traditional Chinese medicine monomer for inhibiting the trimethylamine oxide level, the curative effect is better. Compared with the prior art, the application has the advantages that the core regulation effect of the trimethylamine oxide in the pathological process of the acute pancreatitis is disclosed for the first time, the limitation of the traditional anti-inflammatory or enzyme replacement therapy is broken through, and the acute pancreatitis is treated by inhibiting the trimethylamine oxide level by inhibiting the flavin monooxygenase 3.

The present invention relates to a pharmaceutical composition for CNS delivery of high-concentration heparan N-sulfatase (HNS) with improved stability, and a pharmaceutical dosage form containing the same. The pharmaceutical composition and pharmaceutical dosage form containing the same according to the present invention have excellent dosage form stability, such as reduced turbidity and significantly improved purity, and are therefore useful in enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis type III.

The present invention relates to a pharmaceutical composition for preventing or treating Sanfilippo syndrome type A (MPS IIIA) comprising heparan N-sulfatase (HNS), and more particularly, to an optimal dose and period of administration of heparan N-sulfatase, which can effectively reduce the accumulation of heparan sulfate (HS) while improving the cognition of patients. According to the present invention, it can be used as enzyme replacement therapy (ERT) for treating Sanfilippo syndrome type A.

Provided herein are improved compositions and methods for enzyme replacement therapy using modified human cystathionine beta synthase (CBS) in the treatment of homocystinuria and related diseases and disorders.

The present invention relates to fusion proteins comprising an enzyme replacement therapy enzyme, and provides fusion proteins comprising an enzyme replacement therapy enzyme and an Fc region, and methods of using such proteins to treat lysosomal storage disorders. Also provided herein are methods of delivering an agent across the blood-brain barrier.

The present invention relates to cystathionine beta-synthases for use in the treatment of homocystinuria. The present invention provides agents and methods for enzyme replacement therapy using human cystathionine beta-synthase (CBS), homologs, variants, or mutants thereof to treat homocystinuria and other related diseases and disorders.

The present disclosure provides less immunogenic mutant α-galactosidase A protein (α-gal), methods of making, and methods of use. The less immunogenic mutant α-gal of the present invention provides a reduced immune response when administered to a subject. Methods of treating Fabry in a subject are also provided, the methods comprising administering the disclosed less immunogenic mutant α-gal.

The present invention relates to lipid nanoparticles for the delivery of proteins and, specifically, to lipid nanoparticles for the intracellular or in vivo delivery of various medicinal proteins such as therapeutic proteins or enzymes for enzyme replacement therapy (ERT). The present invention can be advantageously used for the treatment and prevention of various diseases, including lysosomal storage disorders, by stably and effectively delivering various proteins such as therapeutic proteins or ERT enzymes into cells through lipid nanoparticles.

This disclosure relates to systems and methods for modeling the pharmacokinetics and pharmacodynamics of enzyme replacement therapies (ERT) in various systems and tissues.

The present disclosure provides methods of screening, diagnosing, monitoring and / or treating acid sphingomyelinase (ASM) disorders such as Niemann-Pick disease. In particular, the methods encompass techniques for improved diagnosis and / or treatment of an ASM disorder, for example using enzyme replacement therapy.

The present invention relates to silica particles comprising nucleic acids encapsulated inside the same. Furthermore, the present invention relates to a method for producing said particles and the uses thereof in gene transfer or cell marking and as a medicinal product, specifically as a medicinal product for protein / enzyme replacement therapy.

The present invention provides, inter alia, improved methods for purifying arylsulfatase A (ASA) proteins that are recombinantly produced for enzyme replacement therapy. The present invention is based, in part, on the surprising discovery that recombinant ASA proteins can be purified from untreated biological materials (e.g., ASA-containing cell culture media) using processes that include only four chromatographic columns and only one step of post-chromatographic column ultrafiltration / diafiltration.

The present invention relates to the medical field, in particular to nanotechnology-based enzyme replacement therapy, preferably for Parkinson's disease, based on the restoration of lysosomal glucocerebrosidase activity by enzyme-polymer nanoconjugation of GBA, GBA-polymer conjugates for such use, and methods for their preparation.

The present invention provides fusion proteins comprising an enzyme for enzyme replacement therapy and an Fc region, and methods of using such proteins to treat lysosomal storage disorders. The present invention also provides methods for delivering drugs across the blood-brain barrier.

The present disclosure provides less immunogenic mutant α-galactosidase A protein (α-gal), methods of making and methods of use. The less immunogenic mutant α-gal of the present invention provides a reduced immune response when administered to a subject.

The present invention relates to the field of medicine, in particular to: a nanotechnology-based enzyme replacement therapy based on recovery of lysosomal glucocerebrosidase activity by enzyme-polymer nanoconjugation of GBA, preferably for Parkinson's disease; a GBA polymer conjugate for use in such a use; and a method for manufacturing the same.

Methods are provided for treating Fabry disease in patients. Certain methods relate to administering a treatment to Fabry disease patients who are already receiving ERT or are ERT-naive. Certain methods comprise administering to the patient about 100 mg to about 150 mg of migalastat free base equivalent to enhance and / or stabilize cardiac function.