Abstract: The present invention relates to lysosomal enzymes modified by use of cell based methods, a compositions comprising a modified lysosomal enzyme, as well as methods for producing a modified lysosomal enzyme and therapeutic use of such modified lysosomal enzyme. In particular, the present disclosure relates to a modified lysosomal enzyme which has low Man6P and low exposed Mannose and high sialic acid content of alpha2,3 type enabling long circulation time and improved uptake into difficult-to-reach organs like heart, kidney and brain.

Abstract: Provided herein are methods of culturing a mammalian cell in a liquid medium including poloxamer-188 at a concentration of 1.8 g/L or at a greater concentration than 1.8 g/L more or a liquid medium that includes a poloxamer-188 concentration that is selected based on one or more factors selected from the group of: pore size, pore type, gas flow rate, viable cell density in the medium, and markers related to cell stress.

Abstract: The invention provides methods of treating ?-galactosidase A deficiency. Dosage forms, methods of administration, and methods of analyzing human ?-galactosidase A are also included.

Abstract: The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.

Abstract: The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.

Abstract: Disclosed is a method for production of recombinant human alpha-galactosidase A (rh alpha-Gal A) in a large scale, with a high purity. The method comprises the steps of (a) culturing rh alpha-Gal A-producing mammalian cells in a serum-free medium, (b) collecting culture supernatant, (c) subjecting the culture supernatant to anion-exchange column chromatography, (d) to hydrophobic column chromatography, (e) to a column chromatography employing as solid phase a material having affinity for phosphate group, (f) to cation-exchange column chromatography, (g) to dye-affinity column chromatography, and (h) to gel filtration column chromatography, in the order.

Abstract: Multimeric protein structures comprising at least two alpha-galactosidase monomers being covalently linked to one another via a linking moiety are disclosed herein, as well a process for preparing same, and methods of treating Fabry disease via administration of a multimeric protein structure. The disclosed multimeric protein structures exhibit an improved performance, in terms of enhanced activity and/or a longer lasting activity under both lysosomal conditions and in a serum environment.

Abstract: Methods of producing recombinant therapeutic fusion proteins described herein can include culturing Chinese hamster ovary (CHO) cells under conditions suitable for expression of a recombinant therapeutic fusion protein including one or more glycans, where the CHO cells have not been genetically engineered to produce terminal alpha-galactosyl residues on glycans; treating the one or more glycans of the recombinant glycoprotein with one or more exoglycosidases; measuring glycans containing terminal galactose-alpha-1-3-galactose residues on the fusion protein by nuclear magnetic resonance (NMR); and producing a recombinant therapeutic fusion protein in the CHO cells if a target level of terminal galactose-alpha-1-3-galactose residues is measured.

Abstract: The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.

Abstract: Disclosed is a method for production of recombinant human alpha-galactosidase A (rh alpha-Gal A) in a large scale, with a high purity. The method comprises the steps of (a) culturing rh alpha-Gal A-producing mammalian cells in a serum-free medium, (b) collecting culture supernatant, (c) subjecting the culture supernatant to anion-exchange column chromatography, (d) to hydrophobic column chromatography, (e) to a column chromatography employing as solid phase a material having affinity for phosphate group, (f) to cation-exchange column chromatography, and (g) to gel filtration column chromatography, in the order.

Abstract: Methods of treating glycogen storage disease type II, by administering acid ?-glucosidase, are described, as are compositions for use in treatment of glycogen storage disease type II.

Abstract: The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human ?-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.

Abstract: This invention relates to novel ?-galactosidases for the enzymatic removal of the immunodominant monosaccharides on blood products and tissues. Specifically this invention provides a novel family of ?3 glycosidases, used for the enzymatic removal of type B antigens from blood group B and AB reactive blood products, and the Galili antigen from non-human animal tissues, thereby converting these to non-immunogenic cells and tissues suitable for transplantation.

Abstract: The disclosure provides a general method for the production of protein variants with a reduced aggregation propensity without affecting the thermodynamic stability of the variant with respect to the wild-type protein.

Abstract: The disclosure relates to a Gram negative bacterial cell that is transformed with a nucleic acid molecule that encodes a Gram positive twin-arginine translocase and including methods for the production of polypeptides.

Abstract: This disclosure relates to enhancing growth and/or activity of lactobacilli using a prebiotic formulation which includes iso-malto oligosaccharides and ?-galactosidase; and to enhancing growth and/or activity of bifidobacteria using a prebiotic formulation which includes iso-malto oligosaccharides and ?-glucanase. Other combinations of fibers and enzymes are described below which also stimulate growth and activity of lactobacilli or bifidobacteria. These combinations of enzymes and prebiotics can be taken separately or added to foods, including desserts.

Abstract: A method for the secretory production of a glycoprotein having a human-type sugar chain, comprising a step of introducing a gene of an enzyme capable of performing a transfer reaction of a galactose residue to a non-reducing terminal acetylglucosamine residue, and a gene of heterologous glycoprotein, to obtain a transformed plant cell, a step of culturing the plant cell, and a step of recovering the culture medium of the plant cell.

Abstract: Methods of making ligand-decorated polymer conjugates of therapeutic glycoproteins are described. Improved targeting of glycoproteins to specific tissues is achieved by masking the natural carbohydrate and other surface determinants with high molecular weight polymers, such as, e.g., PEG, polysialic acid, etc., which in turn are decorated with target-specific ligands. In some embodiments, acid-labile linkages in such conjugates or rapidly degradable masking groups allow for the intracellular release of the polymer from the glycoprotein, for example, under conditions found in lysosomes.

Abstract: This disclosure relates to enhancing growth and/or activity of lactobacilli using a prebiotic formulation which includes iso-malto oligosaccharides and ?-galactosidase; and to enhancing growth and/or activity of bifidobacteria using a prebiotic formulation which includes iso-malto oligosaccharides and ?-glucanase. Other combinations of fibers and enzymes are described below which also stimulate growth and activity of lactobacilli or bifidobacteria. These combinations of enzymes and prebiotics can be taken separately or added to foods, including desserts.

Abstract: This invention relates to novel ?-galactosidases for the enzymatic removal of the immunodominant monosaccharides on blood products and tissues. Specifically this invention provides a novel family of ?3 glycosidases, used for the enzymatic removal of type B antigens from blood group B and AB reactive blood products, and the Galili antigen from non-human animal tissues, thereby converting these to non-immunogenic cells and tissues suitable for transplantation.

Abstract: The invention as disclosed herein provides a method for purifying a non-antibody protein from solution, comprising a chromatography step wherein the solution is passed over an affinity construct containing an affinity ligand-coupled solid support, wherein the affinity construct is associated with a bioprocess unit operation, and isolating the non-antibody protein from solution.

Abstract: A thermostable glycosidase enzymes derived from various Thermococcus, Staphylothermus and Pyrococcus organisms is disclosed. The enzymes are produced from native or recombinant host cells and can be utilized in the food processing industry, pharmaceutical industry and in the textile industry, detergent industry and in the baking industry.

Abstract: The present invention relates to methods for degrading or converting a cellulosic material, methods for producing a fermentation product, and methods of fermenting a cellulosic material with an enzyme composition comprising one or more (several) cellulolytic enzymes, a cellobiose dehydrogenase, and a polypeptide having cellulolytic enhancing activity.

Abstract: This invention relates to enzymatic removal of type A and B antigens from blood group A, B, and AB reactive cells in blood products, and thereby converting these to non-A and non-B reactive cells. The invention further relates to using unique ?-N-acetylgalactosaminidases and ?-galactosidases with superior kinetic properties for removing the immunodominant monosaccharides of the blood group A and B antigens and improved performance in enzymatic conversion of red blood cells. The preferred unique ?-N-acetylgalactosaminidases and ?-galactosidases exhibit the following characteristics: (i) exclusive, preferred or no less than 10% substrate specificity for the type A and B branched polysaccharide structures relative to measurable activity with simple mono- and disaccharide structures and aglycon derivatives hereof: (ii) optimal performance at neutral pH with blood group oligosaccharides and in enzymatic conversion of cells: and (iii) a favorable kinetic constant Km with mono- and oligosaccharide substrates.

Abstract: The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having ?-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired ?-galactosidase activity by changing the structure of the active site of wild-type human ?-N-acetylgalactosaminidase.

Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for the provision of proteins lacking or deficient in subjects with a lysosomal storage disease and treatment and/or prevention of lysosomal storage diseases.

Abstract: A Product is described and which contains at least one type of ligand bound to a separation material and which allows selective binding or cleavage of a biomolecule for example in human blood.

Abstract: The present invention relates to polypeptides having xylanase activity and nucleic acid sequences encoding such polypeptides. The invention also relates to nucleic acid constructs, vectors, and host cells comprising the nucleic acid constructs as well as methods for producing and using the polypeptides. One specific application of the xylanase is the selective hydrolysis of pentose sugar components of hemicellulose-containing plant biomass. The nucleotide sequence may be used for the production of the xylanase or optimized mutants thereof.

Abstract: The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having ?-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired ?-galactosidase activity by changing the structure of the active site of wild-type human ?-N-acetylgalactosaminidase.

Abstract: The present invention relates to production of fermentable sugars from lignocellulosic material by enzymatic conversion. The fermentable sugars are useful e.g. in the production of bioethanol. Novel polypeptides having endoglucanase activity, polynucleotides encoding them and vectors and host cells containing the polynucleotides are disclosed. A method for treating cellulosic material with the novel endoglucanase as well as uses of the enzymes and enzyme preparations and a method of preparing them are described.

Abstract: The present invention provides a pharmaceutical composition comprising a protein having ?-galactosidase activity for treating Fabry disease, which causes no allergic side effect, which is highly stable in blood (plasma) and which can readily be taken up by a cell of an affected organ. The pharmaceutical composition for treating Fabry disease of the invention comprises, for example, a protein which acquires an ?-galactosidase activity through alteration of the structure of the active site of wild-type human ?-N-acetylgalactosaminidase.

Abstract: The present invention discloses novel polypeptides and enzyme preparations containing them, which enhance the efficiency of the cellulosic degradation even at elevated temperatures. The polypeptides are produced by recombinant technology, and means for their production are described. The novel polypeptides are useful in processing biomass, and in biofuel, starch, textile, detergent, pulp and paper, food, feed or beverage industries. They may also be used e.g. in cleaning the interior of a dishwashing machine or for biofinishing or biostoning. The novel polypeptides are also useful in animal feed.

Abstract: A method of enhancing the activity of lysosomal ?-Galactosidase A (?-Gal A) in mammalian cells and for treatment of Fabry disease by administration of 1-deoxy-galactonojirimycin and related compounds.

Abstract: Isolated and/or purified polypeptides and nucleic acid sequences encoding polypeptides from Alicyclobacillus acidocaldarius are provided. Further provided are methods of at least partially degrading, cleaving, or removing polysaccharides, lignocellulose, cellulose, hemicellulose, lignin, starch, chitin, polyhydroxybutyrate, heteroxylans, glycosides, xylan-, glucan-, galactan-, or mannan-decorating groups using isolated and/or purified polypeptides and nucleic acid sequences encoding polypeptides from Alicyclobacillus acidocaldarius.

Abstract: The present invention provides methods of evaluating CHO cells.

Abstract: Nucleic acid expression constructs are provided and, more particularly, nucleic acid constructs for expression of human alpha-galactosidase in plant cells, cells expressing the nucleic acid construct, producing the human alpha-galactosidase and uses thereof.

Abstract: The present invention provides a pharmaceutical composition comprising a protein having ?-galactosidase activity for treating Fabry disease, which causes no allergic side effect, which is highly stable in blood (plasma) and which can readily be taken up by a cell of an affected organ. The pharmaceutical composition for treating Fabry disease of the invention comprises, for example, a protein which acquires an ?-galactosidase activity through alteration of the structure of the active site of wild-type human ?-N-acetylgalactosaminidase.

Abstract: The invention relates to strains of S. cerevisiae capable of secreting ?-galactosidase into culture medium, as well as to methods for obtaining ?-galactosidase using said strains and methods for producing biomass and bioethanol by means of culturing said strains in media rich in galactose. In another aspect, the invention provides methods for expressing recombinant proteins using a composition rich in galactose as a culture medium for the microorganisms producing said protein.

Abstract: Provided herein are methods and compositions for expressing a modified polypeptide in a host cell, wherein the modified polypeptide comprises a terminal mannose at an N-linked glycosylation site of the polypeptide. The methods and compositions used herein involve the use of RNA effector molecules (e.g., siRNA, dsRNA etc) administered to a host cell to modify the expression of target genes involved in protein glycosylation (e.g., Mgat1, Mgat4, SLC35A1, SLC35A2 or GNE).

Abstract: Described herein are methods and genetically engineered cells useful for producing an altered N-glycosylation form of a target molecule. Also described are methods and molecules with altered N-glycosylation useful for treating a variety of disorders such as metabolic disorders.

Abstract: The present invention provides an aqueous composition comprising a protein with enzymatic activity of alpha-galactosidase. The present invention further provides a method of stabilizing an aqueous composition comprising a protein with enzymatic activity of alpha-galactosidase, and a method of preparing a purified aqueous composition comprising the protein with enzymatic activity of alpha-galactosidase.

Abstract: A method for the secretory production of a glycoprotein having a human-type sugar chain, comprising a step of introducing a gene of an enzyme capable of performing a transfer reaction of a galactose residue to a non-reducing terminal acetylglucosamine residue, and a gene of heterologous glycoprotein, to obtain a transformed plant cell, a step of culturing the plant cell, and a step of recovering the culture medium of the plant cell.

Abstract: The present invention relates to an isolated mutant eubacterium comprising at least one mutation resulting in a substitution of at least one amino acid in the beta-subunit of the RNA-polymerase encoded for by the rpoB-gene providing an altered production of a product of interest when said production of a product of interest is compared to the production of the same product in an isogenic wild type strain grown at identical conditions, wherein the substitution of at least one amino acid occurs at any of positions 469, 478, 482, 485, or 487 of SEQ ID NO:2, or at the equivalent positions in any eubacterial RNA-polymererase beta-subunit family member. Another aspect of the invention relates to a process for producing at least one product of interest in a mutant eubacterium and to a use of the mutant eubacterium according to the invention for producing at least one product of interest.

Abstract: The invention relates to a polypeptide which comprises the amino acid sequence set out in SEQ ID NO: 2 or an amino acid sequence encoded by the nucleotide sequence of SEQ ID NO: 1, or a variant polypeptide or variant polynucleotide thereof, wherein the variant polypeptide has at least 76% sequence identity with the sequence set out in SEQ ID NO: 2 or the variant polynucleotide encodes a polypeptide that has at least 76% sequence identity with the sequence set out in SEQ ID NO: 2. The invention features the full length coding sequence of the novel gene as well as the amino acid sequence of the full-length functional polypeptide and functional equivalents of the gene or the amino acid sequence. The invention also relates to methods for using the polypeptide in industrial processes. Also included in the invention are cells transformed with a polynucleotide according to the invention suitable for producing these proteins.

Abstract: Promoter regions associated with the Yarrowia lipolytica n-alkane-hydroxylating cytochrome P450 (ALK2) gene are disclosed and have been found to be particularly effective for the expression of heterologous genes in yeast. These promoter regions will be useful for driving high-level expression of genes involved in the production of omega-3 and omega-6 fatty acids.

Abstract: Promoter regions associated with the Yarrowia lipolytica peroxisomal 2,4-dienoyl-CoA reductase (SPS19) gene are disclosed and have been found to be particularly effective for the expression of heterologous genes in yeast. These promoter regions will be useful for driving high-level expression of genes involved in the production of omega-3 and omega-6 fatty acids.

Abstract: The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having ?-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired ?-galactosidase activity by changing the structure of the active site of wild-type human ?-N-acetylgalactosaminidase.

Abstract: The present invention relates to a method for producing tagatose using soy oligosaccharide or soluble sugar solution containing the same, more precisely, a method for producing tagatose comprising the following steps; hydrolyzing soy oligosaccharide by using ?-galactosidase selectively; producing tagatose continuously by enzymatic isomerization of galactose obtained from the hydrolysate; separating the produced tagatose by chromatography; and recycling the non-reacted materials.

Abstract: The present invention provides methods and materials by which glycosylation of glycoproteins can be regulated. Methods include the monitoring and regulation of parameters such that a glycoprotein having a desired product quality is obtained.

Abstract: The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having ?-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired ?-galactosidase activity by changing the structure of the active site of wild-type human ?-N-acetylgalactosaminidase.