The present invention relates to in vitro sialylated human factor H protein or biologically active sialylated fragments or biologically active sialylated variants thereof, wherein the protein does not comprise trisia lylated N-glycans of the structure A3G3S3 (NaNaNa), for use in a method of treatment, wherein the method of treatment involves administering subcutaneously said in vitro sialylated human factor H protein or biologically active sialylated fragments or biologically active sialylated variants thereof subcutaneously. The methods of treatment comprise treating complement-mediated diseases such as C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), age-related macular degeneration (AMD) or paroxysmal nocturnal hemoglobinuria.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 9/00 - Medicinal preparations characterised by special physical form
A61P 13/00 - Drugs for disorders of the urinary system
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
The present invention relates to the field of expression of recombinant glycoproteins in plants. In particular, the present invention relates to a method of producing a genetically modified plant capable of producing glycoproteins harbouring paucimannosidic N-glycans, to a method of producing a glycoprotein of interest harbouring paucimannosidic N-glycans and to genetically modified plants, plant cells, protoplasts, protonemas, gametophytes, sporophytes or spores produced with or used in such methods.
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
07 - Machines and machine tools
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Recombinant proteins; proteins for use in manufacture;
proteins for use in science. Biopharmaceuticals; moss for pharmaceutical purposes;
recombinant protein preparations for pharmaceutical
purposes. Bioreactors for use in manufacturing biopharmaceuticals. Custom manufacture of pharmaceuticals; special production of
proteins for scientific purposes. Development of biopharmaceutical products and processes;
research and development in the pharmaceutical and
biotechnology fields.
5.
SIALYLATED HUMAN FACTOR H PROTEIN FOR TREATMENT OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
The present invention relates to in vitro sialylated human factor H protein or biologically active sialylated fragments or biologically active sialylated variants thereof for use in treating paroxysmal nocturnal hemoglobinuria, for treating thromboinflammation, for treating pathological platelet aggregate formation for treating microangiopathy, and/or for treating Long COVID. Combination with a C5 inhibitor, e.g. eculizumab, is also envisaged.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
in vitroin vitro sialylated human factor H protein or biologically active sialylated fragments or biologically active sialylated variants thereof for use in treating paroxysmal nocturnal hemoglobinuria, for treating thromboinflammation, for treating pathological platelet aggregate formation for treating microangiopathy, and/or for treating Long COVID. Combination with a C5 inhibitor, e.g. eculizumab, is also envisaged.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
01 - Chemical and biological materials for industrial, scientific and agricultural use
07 - Machines and machine tools
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Recombinant proteins for industrial use; proteins for use in manufacture; proteins for use in science, namely, protein in raw material form for scientific research Bioreactors for use in manufacturing biopharmaceuticals Custom manufacture of pharmaceuticals; special production in the nature of custom manufacture of proteins for scientific purposes Development of biopharmaceutical products and processes; research and development in the pharmaceutical and biotechnology fields.
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
07 - Machines and machine tools
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Recombinant proteins; proteins for use in manufacture; proteins for use in science.
(2) Biopharmaceuticals; moss for pharmaceutical purposes; recombinant protein preparations for pharmaceutical purposes.
(3) Bioreactors for use in manufacturing biopharmaceuticals. (1) Custom manufacture of pharmaceuticals; special production of proteins for scientific purposes.
(2) Development of biopharmaceutical products and processes; research and development in the pharmaceutical and biotechnology fields.
01 - Chemical and biological materials for industrial, scientific and agricultural use
07 - Machines and machine tools
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Recombinant proteins; Proteins for use in manufacture; Proteins for use in science. Bioreactors for use in manufacturing biopharmaceuticals. Custom manufacture of pharmaceuticals; Special production of proteins for scientific purposes. Development of (bio)pharmaceutical products and processes; Research and development in the pharmaceutical and biotechnology fields.
10.
SIALYLATED HUMAN FACTOR H PROTEIN AND THERAPEUTIC USE THEREOF
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
11.
SIALYLATED HUMAN FACTOR H PROTEIN AND THERAPEUTIC USE THEREOF
in vitroin vitroin vitro methods of producing such proteins and using such proteins in methods of treating complement-mediated diseases such as C3 glomerulopathy, atypical hemolytic uremic syndrome, or age-related macular degeneration.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
12.
METHOD FOR OBTAINING MATERIAL FROM PLANT CELL SURFACES
The invention concerns a method for detaching expressed material from the surface or from the apoplast of plant cells, wherein the plant cells are treated with a rotor-stator in a liquid medium, wherein the specific heat from the rotor-stator introduced by rotation of the rotor is a maximum of 3 kJ per kg of the liquid medium and per g/L dry weight of the plant cells and the specific heat capacity introduced into the medium is a maximum of 1.5 kJ per kg of the liquid medium per minute and per g/L dry weight of the plant cells.
The present invention relates to a lysosomal protein composition comprising a plurality of lysosomal proteins that are potentially diversely glycosylated according to a glycosylation pattern, wherein said glycosylation pattern has at least 45% paucimannosidic N-glycans; a method of manufacturing the lysosomal protein composition in a bryophyte plant or cell, and medical and non-medical uses of the lysosomal protein composition. E.g. the lysosomal protein can be α-Galactosidase for the treatment of Fabry Disease or β-Glucoceramidase for the treatment of Gaucher's Disease. The unique glycosylation results in improved therapeutic efficacy—surprisingly even without mannose-6-phosphate that is common for CHO cell produced lysosomal proteins.
The present invention relates to a method of treating a patient with Pompe disease comprising providing a composition of a recombinant acid alpha-glucosidase, which comprises a glycosylation structure of formula 1, wherein a "G" represents N-acetylglucosamine, a "M" represents mannose, and a "G/M" represents a terminal N-acetylglucosamine or terminal mannose, wherein one but not both of the "G/M" subunits may be absent in which case the present "G/M" subunit may represent two terminal mannose subunits, wherein one or more of the "G" or "M" subunits are not α1,3-fucosylated, not α1,6-fucosylated and not β1,2-xylosylated, and wherein any one of "G", "M" or "G/M" may be methylated; and said glycosylation structure of formula 1 is present in at least 20% of all glycosylation structures of the recombinant acid alpha-glucosidases of the composition; and administering the composition to the patient.
The invention concerns a method for detaching expressed material from the surface or from the apoplast of plant cells, wherein the plant cells are treated with a rotor-stator in a liquid medium, wherein the specific heat from the rotor-stator introduced by rotation of the rotor is a maximum of 3 kJ per kg of the liquid medium and per g dry weight of the plant cells in 1 L of the liquid medium, and the specific heat introduced into the medium is a maximum of 1.5 kJ per kg of the liquid medium per minute and per g dry weight of the plant cells in 1 L of the liquid medium.
The invention relates to a method for detaching expressed material from the surface or the apoplast of plant cells, the plant cells being treated in a liquid medium using a rotor-stator. The specific heat of the rotor-stator introduced by the rotation of the stator is at most 3 kJ per kg of the liquid medium and per g/l dry mass of the plant cells and the specific heat output introduced into the medium is at most 1.5 kJ per kg of the liquid medium per minute and per g/l dry mass of the plant cells.
The invention relates to a polypeptide comprising a C3 convertase effector domain, a C5 convertase effector domain and optionally a terminal complex inhibitory effector domain which is resistant to deregulation by physiologic FHR-Proteins and has a dimerization motif, and to its therapeutic use.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
18.
Glycosylated lysosomal proteins, method of production and uses
The present invention relates to a lysosomal protein composition comprising a plurality of lysosomal proteins that are potentially diversely glycosylated according to a glycosylation pattern, wherein said glycosylation pattern has at least 45% paucimannosidic N-glycans; a method of manufacturing the lysosomal protein composition in a bryophyte plant or cell, and medical and non-medical uses of the lysosomal protein composition. E.g. the lysosomal protein can be a-Galactosidase for the treatment of Fabry Disease or β-Glucoceramidase for the treatment of Gaucher's Disease. The unique glycosylation results in improved therapeutic efficacy—surprisingly even without mannose-6-phosphate that is common for CHO cell produced lysosomal proteins.
The invention relates to a polypeptide comprising a C3 convertase effector domain, a C5 convertase effector domain and optionally a terminal complex inhibitory effector domain which is resistant to deregulation by physiologic FHR-Proteins und has a dimerization motif, and to its therapeutic use.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
20.
GLYCOSYLATED LYSOSOMAL PROTEINS, METHOD OF PRODUCTION AND USES
The present invention relates to a lysosomal protein composition comprising a plurality of lysosomal proteins that are potentially diversely glycosylated according to a glycosylation pattern, wherein said glycosylation pattern has at least 45 % paucimannosidic N-glycans; a method of manufacturing the lysosomal protein composition in a bryophyte plant or cell, and medical and non-medical uses of the lysosomal protein composition. E.g. the lysosomal protein can be a-Galactosidase for the treatment of Fabry Disease or ß-Glucoceramidase for the treatment of Gaucher 's Disease. The unique glycosylation results in improved therapeutic efficacy - surprisingly even without mannose- 6-phosphate that is common for CHO cell produced lysosomal proteins.
The invention relates to a method for separating off a liquid supernatant from cells, comprising the steps: providing a mixture of the cells with a liquid, charging a first filter housing with the mixture, wherein in the filter housing a filter having a pore size of between 4 µm and 50 µm is provided on a flat base surface pierced in a sieve manner and the walls of the filter housing are connected so as to seal with the flat base surface that is pierced in a sieve manner, applying a differential pressure of at least 0.5 bar on the mixture, as a result of which the liquid portion of the mixture is forced through the filter and a filter cake containing cells remains in the filter housing, and removal of the filtered liquid.
The invention relates to a method for producing a recombinant protein in cells with a cell wall, comprising the step of increasing the secretion of the recombinant protein through the cell wall by expression of the protein in the cells in a culture medium containing a combination of a surface-active polymer and monovalent metal ions and with an osmolarity at least 0.32 osmol/L, said invention further relating to culture media and nutrient mixtures for the method.
