45 results about "Enzyme replacement therapy" patented technology

The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

To provide methods of treating a human subject having non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), and in certain embodiments, NPD subtype B.SOLUTION: A method for treating a human subject having an acid sphingomyelinase deficiency comprises: (a) a regimen for debulking an accumulated sphingomyelin substrate in the human subject including (i) administering an initial low non-toxic dose of an acid sphingomyelinase (ASM) to the human subject, and (ii) administering successively higher doses of ASM to the human subject and monitoring the subject for one or more adverse side effects after each successive dose as indicated by elevated bilirubin or a related adverse event; and (b) a maintenance regimen comprising administering a dose equal to or less than the highest dose tolerated by the subject as a maintenance dose for the subject.SELECTED DRAWING: None

The present disclosure relates to compositions in combination with enzyme replacement therapy, such as siRNA molecules and FN3 domains conjugated thereto, as well as methods of making and using these compositions. Also disclosed is a method of treating a glycogen storage disease comprising administering a composition comprising a siRNA molecule and an FN3 domain conjugated thereto, and an enzyme replacement therapy (ERT) for treating the glycogen storage disease.

Disclosed herein are methods for the treatment of Pompe Disease comprising administering a recombinant AAV (rAAV) vector comprising a rAVV genome comprising a heterologous nucleic acid encoding an acid alpha-glucosidase (GAA) polypeptide operatively linked to a liver-specific promoter, wherein the subject is withdrawn or not administered enzyme replacement therapy (ERT).

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.

Disclosed herein is a method for the delivery of prenatal therapeutics, enzyme replacement therapy, or gene therapy to a fetus in need thereof. The method comprises introducing ionizable lipid nanoparticles (LNPs) nanoparticles comprising a therapeutic mRNA composition into the circulation of the fetus in need of treatment such that the ionizable LNPs deliver the therapeutic mRNA composition.

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 provides, among other things, methods of treatment of Metachromatic Leukodystrophy Disease (MLD) and compositions comprising recombinant arylsulfatase A (ASA) protein using enzyme replacement therapy.

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.

The present invention provides, inter alia, methods of treating metachromatic white matter dystrophy disease (MLD) and compositions comprising a recombinant arylsulfatase A (ASA) protein using enzyme replacement therapy.

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 cystathionine beta-synthase for treating homocystinuria. The present invention provides reagents 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 conditions.

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 a high concentration heparan N-sulfatase (HNS) pharmaceutical composition with enhanced stability for CNS delivery and a pharmaceutical formulation comprising the same. The pharmaceutical composition and the pharmaceutical formulation comprising the same according to the present invention have excellent formulation stability, such as reduction of turbidity and significant improvement of purity, which can be used in enzyme replacement therapy (ERT) for the treatment of type III mucopolysaccharide storage disease.

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 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 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 invention provides, among other things, improved methods for purifying arylsulfatase A (ASA) protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant ASA protein can be purified from unprocessed biological materials, such as, ASA-containing cell culture medium, using a process involving as few as four chromatography columns and only one step of post-chromatographic ultrafiltration / diafiltration.

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 invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.

Provided are 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 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 provides, among other things, methods of treatment of Metachromatic Leukodystrophy Disease (MLD) and compositions comprising recombinant arylsulfatase A (ASA) protein using enzyme replacement therapy.

Provided herein are polypeptides comprising one or more non-native cysteine residues that form a disulfide bridge between non-native cysteines within the protein or between non-native cysteines of two monomers of the protein. Such modified human polypeptides are useful in treatment of genetic diseases via enzyme replacement therapy and / or gene therapy.

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 invention relates to a lipid nanoparticle for protein delivery, and more particularly, to a lipid nanoparticle for delivering a plurality of medical proteins, such as a therapeutic protein or an enzyme replacement therapy (ERT) enzyme, into a cell or in a body. According to the present invention, a plurality of proteins such as a therapeutic protein or an enzyme replacement therapy (ERT) enzyme can be stably and efficiently delivered into a cell through the lipid nanoparticles, and thus the lipid nanoparticles can be effectively applied to the treatment and prevention of a plurality of diseases such as lysosomal storage diseases.

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.