| 序号 | 专利名 | 申请号 | 申请日 | 公开（公告）号 | 公开（公告）日 | 发明人 |
|---|---|---|---|---|---|---|
| 181 | Systems and methods for nucleic acid expression in vivo | US15268000 | 2016-09-16 | US10086089B2 | 2018-10-02 | Robert James Debs; Timothy D. Heath; Chakkrapong Handumrongkul |
| The present invention provides compositions, systems, kits, and methods for generating expression of one or more proteins and/or biologically active nucleic acid molecules in a subject (e.g., at therapeutic levels for extended periods required to produce therapeutic effects). In certain embodiments, systems and kits are provided that comprise a first composition comprising a first amount of polycationic structures, and a second composition comprising a therapeutically effective amount of expression vectors (e.g., non-viral expression vectors not associated with liposomes) that are CpG-free or CpG-reduced, where the expression vectors comprise a first nucleic acid sequence encoding: i) a first therapeutic protein or proteins, and/or ii) a first biologically active nucleic acid molecule or molecules. | ||||||
| 182 | Recombinant promoters and vectors for protein expression in liver and use thereof | US15128912 | 2016-04-15 | US10058624B2 | 2018-08-28 | Christopher B. Doering; H. Trent Spencer; Harrison C. Brown |
| Disclosed herein are recombinant viral vectors comprising a liver specific promotor in operable combination with a heterologous nucleic acid sequence encoding a protein, such as a clotting factor. Methods of treating a subject with a clotting disorder, such as hemophilia A or hemophilia B, are also provided. | ||||||
| 183 | Factor IX Polypeptides and Methods of Use Thereof | US15890290 | 2018-02-06 | US20180228879A1 | 2018-08-16 | Glenn PIERCE; SAMANTHA TRUEX; ROBERT T. PETERS; HAIYAN JIANG |
| The present invention provides methods of administering Factor IX; methods of administering chimeric and hybrid polypeptides comprising Factor IX; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells. | ||||||
| 184 | Factor IX Polypeptides and Methods of Use Thereof | US15820080 | 2017-11-21 | US20180207244A1 | 2018-07-26 | Glenn PIERCE; Samantha Truex; Robert T. Peters; Haiyan Jiang |
| The present invention provides methods of administering Factor IX; methods of administering chimeric and hybrid polypeptides comprising Factor IX; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells. | ||||||
| 185 | COMPLEXES OF RNA AND CATIONIC PEPTIDES FOR TRANSFECTION AND FOR IMMUNOSTIMULATION | US15885783 | 2018-01-31 | US20180169267A1 | 2018-06-21 | Mariola FOTIN-MLECZEK; Patrick BAUMHOF |
| The present invention relates to a complexed RNA, comprising at least one RNA complexed with one or more oligopeptides, wherein the oligopeptide, which has the function of cell-penetrating peptide (CPP), has a length of 8 to 15 amino acids and has the empirical formula (Arg)l;(Lys)m;(His)n;(Om)o;(Xaa)x with the majority of residues being selected from Arg, Lys, His, Om. The invention further relates to a method for transfecting a cell or an organism, thereby applying the inventive complexed RNA. Additionally, pharmaceutical compositions and kits comprising the inventive complexed RNA, as well as the use of the inventive complexed RNA for transfecting a cell, tissue or an organism and/or for modulating, preferably inducing or enhancing, an immune response are disclosed herein. | ||||||
| 186 | COMPLEXES OF RNA AND CATIONIC PEPTIDES FOR TRANSFECTION AND FOR IMMUNOSTIMULATION | US15884618 | 2018-01-31 | US20180169266A1 | 2018-06-21 | Mariola FOTIN-MLECZEK; Patrick BAUMHOF |
| The present invention relates to a complexed RNA, comprising at least one RNA complexed with one or more oligopeptides, wherein the oligopeptide, which has the function of cell-penetrating peptide (CPP), has a length of 8 to 15 amino acids and has the empirical formula (Arg)l;(Lys)m;(His)n;(Om)o;(Xaa)x with the majority of residues being selected from Arg, Lys, His, Om. The invention further relates to a method for transfecting a cell or an organism, thereby applying the inventive complexed RNA. Additionally, pharmaceutical compositions and kits comprising the inventive complexed RNA, as well as the use of the inventive complexed RNA for transfecting a cell, tissue or an organism and/or for modulating, preferably inducing or enhancing, an immune response are disclosed herein. | ||||||
| 187 | Purification method for divalent cation binding proteins by anion exchange chromatography | US15615696 | 2017-06-06 | US20180111961A1 | 2018-04-26 | Artur Mitterer; Meinhard Hasslacher; Christian Fiedler |
| The present invention relates to a two-step method for the purification of divalent cation binding proteins with high yield and high purity on anion exchange resin materials, to divalent cation binding proteins obtainable by said method, and to a kit comprising means for carrying out said method. | ||||||
| 188 | Purification of blood coagulation factors | US13522548 | 2011-01-18 | US09896677B2 | 2018-02-20 | Jais Rose Bjelke |
| The present invention relates to the purification of vitamin K-dependent blood coagulation factors, such as Factor IX (FIX). In particular, the invention provides a method for purifying Factor IX having a desired content of gamma-carboxyglutamic acid from a sample comprising a mixture of species of said Factor IX having different contents of gamma-carboxyglutamic acid, said method comprising the steps of: (a) loading said Factor IX sample onto an immunoaffinity chromatography material coupled to a binding moiety for gamma-carboxyglutamic acid; (b) eluting said Factor IX; and (c) selecting a fraction obtained from said elution wherein the polypeptides in the fraction have the desired content of gamma-carboxyglutamic acids; characterized in that the total concentration of Factor IX within said sample exceeds the binding ability of the immunoaffinity chromatography material. | ||||||
| 189 | METHOD OF DETECTING AND/OR IDENTIFYING ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND ISOLATING NOVEL SEQUENCES IDENTIFIED THEREBY | US15782980 | 2017-10-13 | US20180030479A1 | 2018-02-01 | Guangping Gao; James M. Wilson; Mauricio R. Alvira |
| Adeno-associated virus rh.10 sequences, vectors containing same, and methods of use are provided. | ||||||
| 190 | Modified polynucleotides for the production of cosmetic proteins and peptides | US14390103 | 2013-03-09 | US09878056B2 | 2018-01-30 | Stephane Bancel; Eric Yi-Chun Huang |
| The invention relates to cosmetic mRNAs encoding elastin, and methods of using such mRNAs. | ||||||
| 191 | RECOMBINANT PROMOTERS AND VECTORS FOR PROTEIN EXPRESSION IN LIVER AND USE THEREOF | US15128912 | 2016-04-15 | US20170326256A1 | 2017-11-16 | Christopher B. Doering; H. Trent Spencer; Harrison C. Brown |
| Disclosed herein are recombinant viral vectors comprising a liver specific promotor in operable combination with a heterologous nucleic acid sequence encoding a protein, such as a clotting factor. Methods of treating a subject with a clotting disorder, such as hemophilia A or hemophilia B, are also provided. | ||||||
| 192 | Modified polynucleotides for the production of biologics and proteins associated with human disease | US14878400 | 2015-10-08 | US09814760B2 | 2017-11-14 | Stephane Bancel; Tirtha Chakraborty; Antonin de Fougerolles; Sayda M. Elbashir; Matthias John; Atanu Roy; Susan Whoriskey; Kristy M. Wood; Paul Hatala; Jason P. Schrum; Kenechi Ejebe; Jeff Lynn Ellsworth; Justin Guild |
| The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules. | ||||||
| 193 | Modified polynucleotides for the production of proteins associated with human disease | US15060707 | 2016-03-04 | US09782462B2 | 2017-10-10 | Stephane Bancel; Tirtha Chakraborty; Antonin de Fougerolles; Sayda M. Elbashir; Matthias John; Atanu Roy; Susan Whoriskey; Kristy M. Wood; Paul Hatala; Jason P. Schrum; Kenechi Ejebe; Jeff Lynn Ellsworth; Justin Guild |
| The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules. | ||||||
| 194 | Human coagulation factor light chain protein and use of the same | US14894415 | 2014-05-22 | US09782461B2 | 2017-10-10 | Xu Song; Ling Li; Jinwu Chen; Dongsheng Liao; Dengjiao Ma |
| In the invention, the minimum inhibitory concentrations of human coagulation factor light-chain proteins against different Gram-negative bacteria are detected with the in vitro antibacterial activity and the inhibiting effect of the human coagulation factor light-chain proteins against different Gram-negative bacteria is detected with the in vivo antibacterial activity. It has been shown that human coagulation factor light-chain proteins have an obvious inhibitory effect on the Gram-negative bacteria, so as to develop a novel class of medicaments for treating Gram-negative bacteria infection. It has been demonstrated by mass spectrometry and silver staining that human coagulation factor light-chain proteins have the effect on hydrolyzing and eliminating the endotoxin, which facilitates the development of a novel class of medicaments for treating endotoxemia. The human coagulation factor light-chain proteins are light chain proteins of human coagulation factors VII, IX, and X, as well as a protein having homology of more than 50% thereof. | ||||||
| 195 | GLYCEROL LINKED PEGYLATED SUGARS AND GLYCOPEPTIDES | US15629151 | 2017-06-21 | US20170281785A1 | 2017-10-05 | Shawn DeFrees; Xiao Nong Zeng |
| The present invention provides conjugates between peptides and PEG moieties through glycerol linkers. | ||||||
| 196 | COAGULATION FACTOR IX COMPOSITIONS AND METHODS OF MAKING AND USING SAME | US15427763 | 2017-02-08 | US20170247676A1 | 2017-08-31 | Volker SCHELLENBERGER; Willem P. Stemmer; Nathan C. Geething; Wayne To; Joshua Silverman; Chia-wei Wang; Benjamin Spink |
| The present invention relates to compositions comprising factor IX coagulation factors linked to extended recombinant polypeptide (XTEN), isolated nucleic acids encoding the compositions and vectors and host cells containing the same, and methods of making and using such compositions in treatment of coagulation factor-related diseases, disorders, and conditions. | ||||||
| 197 | IMMUNOGLOBULIN CHIMERIC MONOMER-DIMER HYBRIDS | US15491742 | 2017-04-19 | US20170226189A1 | 2017-08-10 | Robert T. PETERS; Adam R. Mezo; Daniel S. Rivera; Alan J. Bitoni; Susan C. Low |
| The invention relates to a chimeric monomer-dimer hybrid protein wherein the protein comprises a first and a second polypeptide chain, the first polypeptide chain comprising at least a portion of an immunoglobulin constant region and a biologically active molecule, and the second polypeptide chain comprising at least a portion of an immunoglobulin constant region without the biologically active molecule of the first chain. The invention also relates to methods of using and methods of making the chimeric monomer-dimer hybrid protein of the invention. | ||||||
| 198 | Administration of plant expressed oral tolerance agents | US13508754 | 2010-11-09 | US09724400B2 | 2017-08-08 | Henry Daniell; Roland Herzog |
| Protein replacement therapy for patients with hemophilia or other inherited protein deficiencies is often complicated by pathogenic antibody responses, including antibodies that neutralize the therapeutic protein or that predispose to potentially life-threatening anaphylactic reactions by formation of IgE. Using murine hemophilia B as a model, we have developed a prophylactic protocol against such responses that is non-invasive and does not include immune suppression or genetic manipulation of the patient's cells. Oral delivery of coagulation factor IX (F. IX) expressed in chloroplasts, bioencapsulated in plant cells, effectively blocked formation of inhibitory antibodies in protein replacement therapy. Inhibitor titers were mostly undetectable and up to 100-fold lower in treated mice when compared to controls. Moreover, this treatment eliminated fatal anaphylactic reactions that occurred after 4 to 6 exposures to intravenous F. IX protein. While only 20-25% of control animals survived after 6-8 F. IX doses, 90-95% of tolerized mice survived 12 injections without signs of allergy or anaphylaxis. This high-responder strain of hemophilia B mice represents the first hemophilic animal model to study anaphylactic reactions. The plant material was effective over a range of oral antigen doses (equivalent to 5-80 μg recombinant F.IX/kg), and controlled inhibitor formation and anaphylaxis long-term, up to 7 months. Oral antigen administration caused a deviant immune response that suppressed formation of IgE and inhibitory antibodies. This cost-effective and efficient approach to oral delivery of protein antigens to the gut should be applicable to several genetic diseases that are prone to pathogenic antibody responses during treatment. | ||||||
| 199 | Protein purification by anion exchange chromatography | US14351544 | 2012-10-12 | US09701710B2 | 2017-07-11 | Artur Mitterer; Meinhard Hasslacher; Christian Fiedler |
| The present invention relates to a two-step method for the purification of divalent cation binding proteins with high yield and high purity on anion exchange resin materials, to divalent cation binding proteins obtainable by said method, and to a kit comprising means for carrying out said method. | ||||||
| 200 | Method for Improved Isolation of Recombinantly Produced Proteins | US15199585 | 2016-06-30 | US20170183703A1 | 2017-06-29 | Stefan Winge |
| The present invention provides a method for increasing the yield of a protein produced by cultivating eukaryotic cells and adding an ionic substance to the culture medium prior to harvest of the protein. Suitable ionic substances are the salts of the Hofmeister series, amino acids and peptone. | ||||||
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