163results about "Erythropoietin" patented technology

This invention provides purified preparations of an RNA, oligoribonucleotide, or polyribonucleotide comprising a modified nucleoside, and methods of assessing purity of purified preparations of an RNA, oligoribonucleotide, or polyribonucleotide comprising a modified nucleoside.

The invention provides certain specific lipid nanoparticles comprising: (a) one or more nucleic acid molecules; (b) cholesterol; (c) DSPC; (d) PEG-C-DMA; and (e) a cationic lipid; and pharmaceutical compositions comprising the lipid nanoparticles. The lipid nanoparticles and pharmaceutical compositions are particularly useful for delivering a nucleic acid such as siRNA or mRNA to a patient (e.g. a human) or to a cell.

This disclosure provides a method of preventing, alleviating, or treating a condition (i.e., neutropenia) in a patient in need thereof, the condition characterized by compromised white blood cell production in the patient. The method includes administering to the patient a therapeutically effective amount of a protein complex comprising a modified human granulocyte-colony stimulating factor (hG-CSF) covalently linked to an immunoglobulin Fc region via a non-peptidyl polymer. The non-peptidyl polymer is site-specifically linked to an N-terminus of the immunoglobulin Fc region, and the modified hG-CSF comprises substitutions in at least one of Cys17 and Pro65.

The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by inproved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

Processes for providing a PEGylated protein composition comprising mono-PEGylated protein and oligo-PEGylated protein, and processes for providing mono-PEGylated protein compositions with high yield and productivity are provided. The processes are particularly suitable for providing mono-PEGylated erythropoietin composition. The processes comprise reacting a non-PEGylated protein with a PEGylation reagent to produce a mixture comprising non-PEGylated, mono-PEGylated and oligo-PEGylated protein, subjecting the mixture to a step of anion exchange chromatography, and recycling non-PEGylated protein into further PEGylation reactions.

The present invention relates to a mammalian cell comprising a gene encoding a polypeptide of interest, wherein the polypeptide of interest is expressed comprising one or more posttranslational modification patterns. These modifications are useful for example in glycoprotein production where the antibodies with the modifications have an enhanced antibody-dependent cell-mediated cytotoxicity (ADCC). The present invention also relates to methods for producing the glycoproteins and compositions comprising the glycoproteins, and their uses.

To provide: a method for producing a complicated peptide molecule, such as a dimeric peptide, in a simpler manner at lower cost compared with the conventional chemical synthesis methods; a dimeric peptide which comprises a specific short linker and has a satisfactory activity; and others.SOLUTION: A peptide molecule comprises: (I) a first physiologically active peptide portion; (II) a linker portion composed of 49 or less amino acid residues; and (III) a second physiologically active peptide portion located on the opposite side of the linker portion from the first physiologically active peptide portion, wherein the first physiologically active peptide portion and the second physiologically active peptide portion may be the same as or different from each other, and at least 90% of an amino acid sequence of the linker portion is composed of amino acid residues selected from alanine (A), proline (P), and serine (S).SELECTED DRAWING: None

Provided herein are transgenic cells and transgenic non-human animals (e.g., rats and mice) comprising: (i) Rag1 and / or Rag2 gene knockout; (i) knocking out the IL2rg gene; and (iii) a homozygous ineffective mutation in a non-human animal heme oxygenase-1 (Hmox-1) gene, and an optional Fah gene knockout and / or expresses one or more human or humanized polypeptides. Methods and compositions for making and using such transgenic cells and non-human animals are also provided.

Provided are non-plant polypeptides or proteins comprising xylosylated N-linked glycans containing β1,2-xylose, wherein at least 25% of the individual protein species comprise xylosylated N-glycans, compositions comprising the non-plant proteins, cells engineered to produce the polypeptides or proteins, and methods for their production and use.

The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

The present invention relates to bone morphogenetic protein 6 (BMP6) antagonists, such as antibodies and fragments, as well as methods, uses and combinations.

The present invention relates to a method for recombinantly producing a glycoprotein composition comprising at least one glycoprotein comprising the step of cultivating a recombinant host cell which expresses the glycoprotein in a cultivation medium in a bioreactor having a pH measuring device positioned in the bioreactor and configured to be in physical contact with the cultivation medium, characterized in that (a) the cultivating is performed under sterile conditions, (b) the pH value measured with the pH measuring device differs by 0.05 units or less from the pH value of the cultivation medium, and (c) the relative content of at least one glycosylated variant in the glycoprotein composition has reduced batch-to-batch variability, compared to a method where the pH value measured by the pH measuring device differs by more than 0.05 units, preferably by more than 0.03 units, from the pH value in the cultivation medium, and thereby producing the glycoprotein composition. The present invention further relates to glycoprotein compositions produced by the method, and to the use of a method for carbon dioxide based pH calibration for improving batch-to-batch variability.

Polypeptides comprising at least one carboxy-terminal peptide (CTP) of chorionic gonadotrophin attached to the carboxy terminus but not to the amino terminus of a coagulation factor and polynucleotides encoding the same are disclosed. Pharmaceutical compositions comprising the polypeptides and polynucleotides of the invention and methods of using and producing same are also disclosed.

To provide pharmaceuticals for treating or preventing anemia, such as moderate to severe anemia.SOLUTION: The invention relates to bone morphogenetic protein 6 (BMP6) antagonists, such as antibodies and fragments, as well as methods, uses and combinations.SELECTED DRAWING: None

Therapeutic mRNA molecules are provided that are expressible to provide a target polypeptide or protein.SOLUTION: The RNA molecule can contain one or more 5-methoxyuridines and 5-methylcytidines. Further provided is a DNA template that can be transcribed to provide a target mRNA and that can have altered nucleotides such as reduced deoxyadenosine. The present invention also provides a process for producing a therapeutic mRNA molecule. An RNA molecule can be translated in vitro or in vivo to provide an active polypeptide or protein.SELECTED DRAWING: Figure 1

The present invention relates to RNA therapy and, in particular, decreasing immunogenicity of RNA. Specifically, the present invention provides methods for decreasing immunogenicity of RNA, said methods comprising modifying the nucleotide sequence of the RNA by reducing the uridine (U) content, wherein said reduction of the U content comprises an elimination of U nucleosides from the nucleotide sequence of the RNA and / or a substitution of U nucleosides by nucleosides other than U in the nucleotide sequence of the RNA. Using RNA having decreased immunogenicity allows administration of RNA as a drug to a subject, e.g. in order to obtain expression of a pharmaceutically active peptide or protein, without eliciting an immune response which would interfere with therapeutic effectiveness of the RNA or induce adverse effects in the subject.

This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5' UTRs, where a 5' UTR may be expressed by a gene of a plant. In some embodiments, a 5 UTR may be expressed by a gene of a member of Arabidopsis genus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.

The peptides according to aspects of the present invention, as well as pharmaceutical compositions and external skin preparations containing the peptides, promote the growth of individual hair follicle cells and increase the amount of hair growth-related cytokines, thereby having the effects of hair care, hair growth, or hair growth promotion, and thus effectively treating, improving or preventing hair loss.

The present invention provides, in part, asymmetric piperazine-based lipid compounds of Formula (I'), and sub-formulas thereof or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

The application discloses a preparation method of a pig blood bioreactor for simultaneously expressing human albumin and erythropoietin, and belongs to the technical field of genetic engineering and animal bioreactor. The method is characterized in that a specific sgRNA is designed for a +91 position of a pig ALB gene by using a CRISPR / Cas9 RNP complex-mediated gene editing technology, a human ALB, EPO and FC fusion gene is knocked into a pig ALB gene site, after electric transfection and positive monoclonal cell screening, a positive monoclonal cell is used as a donor cell to perform somatic cell nuclear transfer, a reconstructed embryo is obtained and transplanted into a recipient sow, and finally, a gene editing cloned pig is obtained. The cloned pig can stably express human albumin and human erythropoietin in blood at the same time. The application has the characteristics of high editing efficiency, low off-target effect and high pregnancy rate of the recipient, and can be used for large-scale production of human albumin and erythropoietin biological products.

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.

To provide an agent for reducing iron overload in patients having conditions such as beta-thalassaemia and myelodysplastic syndrome (MDS).SOLUTION: Antibodies to enzyme matriptase-2 (MTP-2) are presented. Inhibiting MTP-2 reduces uptake of dietary iron and reduces release of iron from cellular stores in the body. Inhibitors of MTP-2 (such as antibodies to a serine protease domain) can be used to treat iron overload, which is a feature of diseases such as beta-thalassaemia and which otherwise leads to toxic accumulation of iron. Combination of an MTP-2 inhibitor with an activin receptor ligand trap, or with erythropoeitin, provides additional therapeutic effects.SELECTED DRAWING: Figure 1

The present invention relates to a nucleic acid sequence, comprising or coding for a coding region, encoding at least one peptide or protein comprising a therapeutic protein or a fragment, variant or derivative thereof, at least one histone stem-loop and a poly(A) sequence or a polyadenylation signal. Furthermore the present invention provides the use of the nucleic acid for increasing the expression of said encoded peptide or protein, particularly for the use in gene therapy. It also discloses its use for the preparation of a pharmaceutical composition, e.g. for use in gene therapy, particularly in the treatment of diseases which are in need of a treatment with a therapeutic peptide or protein, preferably as defined herein. The present invention further describes a method for increasing the expression of a peptide or protein comprising a therapeutic protein or a fragment, variant or derivative thereof, using the nucleic acid comprising or coding for a histone stem-loop and a poly(A) sequence or a polyadenylation signal.

The present invention relates to a solid oral pharmaceutical composition comprising (i) a core comprising a peptide or protein drug, and (ii) a first coating, wherein the first coating comprises a copolymer (A) in combination with a copolymer (B) and / or a copolymer (C) and / or a copolymer (D).

The invention provides a preparation method and application of a human erythropoietin transgenic pig, and belongs to the field of gene engineering. According to the invention, a human EPO gene is knocked into a fibroblast line of a pig to construct a humanized EPO transgenic pig, and genotype DNA identification and protein expression analysis of a cloned pig are further carried out to obtain a transgenic human EPO gene editing xenogenic organ transplantation donor pig. According to the invention, the problems of renal anemia and the like in the process of xenogeneic organ kidney transplantation are improved by modifying the pig transferred humanized gene, and the method has an important application value for promoting pre-clinical research of xenogeneic organ transplantation of pig-non-human primates and clinical application of xenogeneic organ transplantation of pig-human.

(i) Rag1 and / or Rag2 gene knockout; (ii) IL2rg gene knockout; and (iii) a homozygous null mutation in the non-human animal heme oxygenase-1 (Hmox-1) gene, and optionally a Fah gene knockout, and / or expressing one or more human or humanized polypeptides, genetically modified cells and genetically modified non-human animals (e.g., rats and mice) are provided herein. Methods and compositions for making and using such genetically modified cells and non-human animals are also provided.

The present invention relates to a solid oral pharmaceutical composition comprising (i) a core comprising a peptide or protein drug, and (ii) a first coating, wherein the first coating comprises a copolymer (A) in combination with a copolymer (B) and / or a copolymer (C) and / or a copolymer (D).

The present invention provides methods, agents, compounds, and compositions useful for administering to subjects having or at risk of having anemias. In certain embodiments, methods herein comprise administering two agents to a subject, which are useful for the treatment of an anemia.