Consejo Superior de Investigaciones Científicas (CSIC) (Spain)
Universidad de Sevilla (Spain)
Inventor
Moreno Herrero, Jorge
Haas, Heinrich
Erbar, Stephanie
Stahl, Theo Benjamin
García Fernández, José Manuel
Benito Hernández, Juan Manuel
Lopez Fernandez, Jose
Ortiz Mellet, Maria Del Carmen
De La Cruz Ruiz, Noelia
González Cuesta, Manuel
Jacobus Ambuludi, Egon Jack
Vlatkovic, Irena
Abstract
The present disclosure provides complexes comprising: i) a cationic oligosaccharide comprising one or more cationic moieties bonded to a trehalose, a sucrose, or a gluco-n-oligosaccharide moiety, where n is 2-6; ii) a surfactant; and iii) one or more additives selected from: a sterol, a helper lipid, an immunomodulator, and a targeting molecule; and uses thereof.
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
A61P 31/16 - Antivirals for RNA viruses for influenza or rhinoviruses
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
Consejo Superior de Investigaciones Científicas (CSIC) (Spain)
Inventor
Moreno Herrero, Jorge
Haas, Heinrich
Erbar, Stephanie
García Fernández, José Manuel
Benito Hernández, Juan Manuel
Lopez Fernandez, Jose
Abstract
The present disclosure provides complexes comprising a cationic oligosaccharide and an RNA, wherein the cationic oligosaccharide comprises a plurality of cationic moieties bonded to a trehalose, a sucrose, or a gluco-n-oligosaccharide moiety, where n is 2-6, and a ratio of N/P in the complex is less than 20:1
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA (USA)
Inventor
Sahin, Ugur
Rooney, Michael Steven
Esaulova, Ekaterina
Zuiani, Adam
Addona, Theresa
Poran, Asaf
Goulding, Scott
Velazquez, Ricardo Sanchez
Uebele, Julia
Güler, Alptekin
Lu, Yu-Jung
Friedman, Harvey
Awasthi, Sita
Cohen, Gary H.
Abstract
The present disclosure provides pharmaceutical compositions for delivery of HSV antigens (e.g., an HSV vaccine) and related technologies (e.g., components thereof and/or methods relating thereto).
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
A61P 31/22 - Antivirals for DNA viruses for herpes viruses
4.
PHARMACEUTICAL COMPOSITIONS FOR DELIVERY OF HERPES SIMPLEX VIRUS ANTIGENS AND RELATED METHODS
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA (USA)
Inventor
Güler, Alptekin
Velazquez, Ricardo Sanchez
Uebele, Julia
Vogel, Annette
Pfafenrot, Christina
Ernst, Anna Luise
Hein, Stephanie
Hinz, Sabrina
Sahin, Ugur
Rooney, Michael Steven
Esaulova, Ekaterina
Zuiani, Adam
Addona, Theresa
Poran, Asaf
Goulding, Scott
Walls, Alexandra
Srouji, John
Palowitch, Gavin
Dulberger, Charles Lefco
Dany, Sarah Catharina
Strauss, Stefan Thomas
Cohen, Gary H.
Friedman, Harvey
Awasthi, Sita
Weissman, Drew
Hook, Lauren Michelle
Egan, Kevin
Cairns, Tina M.
Atanasiu, Doina
Saw, Wan Ting
Abstract
The present disclosure provides pharmaceutical compositions for delivery of HSV antigens (e.g., an HSV vaccine) and related technologies (e.g., components thereof and/or methods relating thereto).
The invention relates to agents and methods for targeted delivery of nucleic acids to cells. In some embodiments, the invention involves a particle, said particle comprising a connector compound comprising (i) a moiety incorporating the connector compound into the particle through a charge in the moiety incorporating the connector compound into the particle interacting with an opposite charge in the particle, and (ii) a first interacting moiety, and said particle carrying a nucleic acid payload. A docking compound comprising (i) a second interacting moiety, and (ii) a moiety binding to a cell surface antigen interacts with the connector compound through the first interacting moiety and the second interacting moiety binding to each other, and thus targets the complex to a target cell of interest.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
6.
METHOD OF DETERMINING NUCLEIC ACID CONCENTRATION IN AQUEOUS SOLUTION OR DISPERSION
A method of determining the nucleic acid concentration in an aqueous dispersion, the method comprising the steps a) to c): a) providing a sample of the nucleic acid; b) mixing the sample with a medium containing: i) a zwitterionic surfactant; and optionally ii) a mobile phase selected from the group consisting of an alcohol, acetone, and dimethyl sulfoxide, or a mixture of any thereof; and c) measuring the concentration of the nucleic acid using ultraviolet-visible spectroscopy; is provided. A method of determining the nucleic acid concentration in an aqueous solution, the method comprising the steps a) to c): a) providing a sample of the nucleic acid; b) mixing the sample with a medium containing: i) a zwitterionic surfactant; and/or ii) a solvent selected from the group consisting of an alcohol, acetone, and dimethyl sulfoxide, or a mixture of any thereof; and c) measuring the concentration of the nucleic acid using ultraviolet-visible spectroscopy; is also provided.
C12Q 1/6806 - Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
G01N 21/33 - Investigating relative effect of material at wavelengths characteristic of specific elements or molecules, e.g. atomic absorption spectrometry using ultraviolet light
G01N 33/487 - Physical analysis of biological material of liquid biological material
7.
AGENTS AND METHODS FOR TARGETED DELIVERY OF IMMUNE EFFECTOR CELLS
The invention relates to agents and methods for targeted delivery of immune effector cells to target cells. In one embodiment, the invention involves providing to a subject immune effector cells genetically modified to express a chimeric antigen receptor (CAR) and a compound (docking compound) comprising a binding moiety for an antigen on target cells and a further binding moiety for a CAR. The binding moiety for an antigen on target cells and the binding moiety for a CAR are connected through a linking moiety comprising at least one poly-2-(2-(2- aminoethoxy)ethoxy)acetic acid (pAEEA) moiety or a derivative thereof.
The present disclosure provides a compound of formula (I): (I), or a pharmaceutically acceptable salt thereof, that is useful for forming particles (e.g., lipid nanoparticles) for delivery of nucleic acids. The present disclosure further provides particle compositions comprising the compound of formula I, as well as uses thereof.
C07D 205/04 - Heterocyclic compounds containing four-membered rings with one nitrogen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
C07D 295/21 - Radicals derived from sulfur analogues of carbonic acid
C07C 311/32 - Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups having the sulfur atoms of the sulfonamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
C07C 317/28 - SulfonesSulfoxides having sulfone or sulfoxide groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton with sulfone or sulfoxide groups bound to acyclic carbon atoms of the carbon skeleton
C07C 323/24 - Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
C07C 323/25 - Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
C07C 335/08 - Derivatives of thiourea having nitrogen atoms of thiourea groups bound to acyclic carbon atoms of a saturated carbon skeleton
C07C 335/40 - Thioureas, i.e. compounds containing any of the groups the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of thiourea or isothiourea groups further bound to other hetero atoms
A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
A61P 37/00 - Drugs for immunological or allergic disorders
A61P 43/00 - Drugs for specific purposes, not provided for in groups
A61K 31/145 - Amines, e.g. amantadine having sulfur atoms, e.g. thiurams (N—C(S)—S—C(S)—N or N—C(S)—S—S—C(S)—N)Sulfinylamines (—N=SO)Sulfonylamines (—N=SO2)
The invention relates to agents and methods for targeted delivery of payloads to cells. The payload includes a variety of molecules such as therapeutic or diagnostic agents and, in particular, toxins, immunomodulators or radiodiagnostics. In some embodiments, the invention involves providing to a subject a compound comprising a payload moiety and at least two tags (tag conjugate) and a compound binding to the tags of the tag conjugate and a target antigen, e.g., a cell surface antigen on a target cell, (docking compound).
A61K 47/55 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of anti-HIV antibody agents and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides encoding an immunoglobulin chain of an anti-HIV antibody agent.
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA (USA)
BIONTECH SE (Germany)
Inventor
Friedman, Harvey
Awasthi, Sita
Cohen, Gary H.
Weissman, Drew
Cairns, Tina
Atanasiu, Doina
Saw, Wan Ting
Güler, Alptekin
Strauss, Stefan Thomas
Abstract
The present disclosure provides a polyribonucleotide encoding a polypeptide that comprises a viral antigen from Herpes Simplex Virus-2 (HSV-2); for example, the viral antigen comprises the ectodomain of HSV-2 glycoprotein B. The polyribonucleotide can be formulated as an RNA composition useful for inducing anti-HSV immune responses in a subject.
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA (USA)
BIONTECH SE (Germany)
Inventor
Friedman, Harvey
Awasthi, Sita
Cohen, Gary H.
Weissman, Drew
Hook, Lauren
Egan, Kevin
Güler, Alptekin
Vogel, Annette
Ernst, Anna Luise
Sanchez Velazquez, Ricardo
Uebele, Julia
Abstract
Disclosed herein are compositions comprising (a) a nucleoside-modified polyribonucleotide encoding a Herpes Simplex Virus-2 (HSV-2) glycoprotein E (gE) antigen or immunogenic fragment thereof, (b) a nucleoside-modified polyribonucleotide encoding an HSV-2 glycoprotein I (gI) antigen or immunogenic fragment thereof, or (c) a combination thereof. Further disclosed are methods for using said compositions for treating an HSV infection.
The present disclosure provides a compound of formula (I) or a pharmaceutically acceptable salt thereof, that is useful for forming particles (e.g., lipid nanoparticles) for delivery of nucleic acids. The present disclosure further provides particle compositions comprising the compound of formula (I), as well as uses thereof.
A61P 37/00 - Drugs for immunological or allergic disorders
A61P 43/00 - Drugs for specific purposes, not provided for in groups
C07C 323/24 - Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
C07D 205/04 - Heterocyclic compounds containing four-membered rings with one nitrogen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
C07D 295/21 - Radicals derived from sulfur analogues of carbonic acid
A61K 31/145 - Amines, e.g. amantadine having sulfur atoms, e.g. thiurams (N—C(S)—S—C(S)—N or N—C(S)—S—S—C(S)—N)Sulfinylamines (—N=SO)Sulfonylamines (—N=SO2)
The disclosure provides RNA therapeutics, e.g. RNA molecules for treating or preventing an infection, and methods for reducing inherent toxicity of antigens or cytotoxicity exhibited by certain microbial antigens or immunogenic variants thereof when expressed intracellularly from RNA, as well as compositions comprising RNA therapeutics produced by such methods. The RNA encoding the antigens, immunogenic variants or fragments thereof is formulated and administered in a way that the antigens, variants or fragments are produced by cells of a subject, in particular after intramuscular or intravenous administration of the RNA and that, at the same time, toxicity or cytotoxicity is avoided.
The invention relates to agents and methods for targeted delivery of nucleic acids to cells. In some embodiments, the invention involves a particle, said particle comprising a connector compound comprising (i) a moiety incorporating the connector compound into the particle through a charge in the moiety incorporating the connector compound into the particle interacting with an opposite charge in the particle, and (ii) a first interacting moiety, and said particle carrying a nucleic acid payload. A docking compound comprising (i) a second interacting moiety, and (ii) a moiety binding to a cell surface antigen interacts with the connector compound through the first interacting moiety and the second interacting moiety binding to each other, and thus targets the complex to a target cell of interest.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
The invention relates to agents and methods for targeted delivery of payloads to cells. The payload includes a variety of molecules such as therapeutic or diagnostic agents and, in particular, toxins, immunomodulators or radiodiagnostics. In some embodiments, the invention involves providing to a subject a compound comprising a payload moiety and a tag (tag conjugate) and a compound binding to the tag of the tag conjugate and a target antigen, e.g., a cell surface antigen on a target cell, (docking compound).
A61K 47/55 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of anti-HIV antibody agents and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides encoding an immunoglobulin chain of an anti-HIV antibody agent.
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of anti-HIV antibody agents and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides encoding a plurality of immunoglobulin chains of anti-HIV antibody agents.
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of malarial protein antigens and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides encoding malarial protein antigens.
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of malarial protein antigens and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides encoding malarial protein antigens.
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of Plasmodium protein antigens and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides combinations comprising a first pharmaceutical composition comprising a first polyribonucleotide and a second pharmaceutical composition comprising a second polyribonucleotide. In some embodiments, a first polyribonucleotide encodes a first polypeptide that comprises one or more Plasmodium T-cell antigens. In some embodiments, a second polyribonucleotide encodes a second polypeptide that comprises one or more Plasmodium polypeptide (e.g., CSP) or antigenic portions thereof.
TRON - TRANSLATIONALE ONKOLOGIE AN DER UNIVERSITÄTSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITÄT MAINZ GEMEINNÜTZIGE GMBH (Germany)
Inventor
Gangluff, Meike
Sahin, Ugur
Keil, Isabell Sofia
Diken, Mustafa
Abstract
The invention relates to agents and methods for targeted delivery of RNA such as mRNA encoding a polypeptide comprising a cytokine or a functional variant thereof to immune cells for expression of the polypeptide. Delivering RNA encoding a cytokine to immune cells may be useful for immunomodulation of immune cells, in particular for inducing proliferation of immune cells. In some embodiments, the invention involves a particle, and a targeting compound comprising a moiety incorporating into the particle, e.g., a hydrophobic moiety, and having a binding moiety covalently attached thereto. The particle carries an RNA payload, i.e., RNA encoding a polypeptide comprising a cytokine or a functional variant thereof. Targeting of an immune cell may be achieved by the direct or indirect binding of the targeting compound to cell surface antigens on the target immune cell of interest.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
23.
AGENTS AND METHODS FOR TARGETED DELIVERY OF CYTOKINES TO IMMUNE CELLS
TRON - TRANSLATIONALE ONKOLOGIE AN DER UNIVERSITÄTSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITÄT MAINZ GEMEINNÜTZIGE GMBH (Germany)
Inventor
Gangluff, Meike
Sahn, Ugur
Keil, Isabell Sofia
Diken, Mustafa
Abstract
The invention relates to agents and methods for targeted delivery of RNA such as mRNA encoding a polypeptide comprising a cytokine ora functional variant thereof to immune cells for expression of the polypeptide. Delivering RNA encoding a cytokine to immune cells may be useful for immunomodulation of immune cells, in particular for inducing proliferation of immune cells. In some embodiments, the invention involves a particle, and a targeting compound comprising a moiety incorporating into the particle, e.g., a hydrophobic moiety, and having a binding moiety covalently attached thereto. The particle carries an RNA payload, i.e., RNA encoding a polypeptide comprising a cytokine or a functional variant thereof. Targeting of an immune cell may be achieved by the direct or indirect binding of the targeting compound to cell surface antigens on the target immune cell of interest.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
The invention provides agents and methods for treating bacterial infections using RNA. The RNA encoding peptidoglycan hydrolases. e.g., endolysins, is formulated and administered in a way that peptidoglycan hydrolase proteins. e.g., endolysin proteins, can be produced and secreted by cells of a subject to combat bacterial infections.
C12N 9/36 - Hydrolases (3.) acting on glycosyl compounds (3.2) acting on beta-1, 4 bonds between N-acetylmuramic acid and 2-acetylamino 2-deoxy-D-glucose, e.g. lysozyme
A61K 9/00 - Medicinal preparations characterised by special physical form
The present disclosure relates to chimeric antigen receptor (CAR) expressing immune effector cells showing highly specific and sensitive recognition of CLDN6 expressing cancer cells as well as a high potential for treating cancer in humans.
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
The present invention relates to the compound of formula (I) and salts, stereoisomers, tautomers, isotopologues, or N-oxides thereof. The present invention is further concerned with inter alia the use of such a compound or salt, stereoisomer, tautomer, isotopologues, or N-oxide thereof as medicament and a pharmaceutical composition comprising said compound.
TRON-Translationale Onkologie an der Universitätsmedizin der Johannes Gutenberg-Universität Mainz gG (Germany)
Inventor
Eberle, Florian
Sahin, Ugur
Kuhn, Andreas
Vallazza, Britta
Diken, Mustafa
Abstract
The present invention relates to nucleic acid molecules containing poly(dA:dT) regions which are stabilized in E.coli, methods of propagating such nucleic acid molecules in E.coli, methods of obtaining RNA, peptides or proteins using such nucleic acid molecules and to RNA which is obtained from such nucleic acid molecules and its use. In particular, the poly(dA:dT) regions contain at least one disruption by a sequence not encoding a sequence solely composed of A residues.
The present invention generally relates to binding agents that are at least bispecific for the binding to CD3 and CLDN6, i.e., they are capable of binding to at least CD3 and CLDN6. Specifically, the present invention relates to RNA encoding these binding agents which may be used in the treatment or prevention of cancer in a subject.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
29.
MODULAR MULTIPLATFORM SYSTEM FOR MRNA DRUG PRODUCTION
A drug production facility includes: a first module comprising drug substance and drug product manufacturing equipment including equipment for producing RNA-based drug substance, lipid nanoparticle (LNP)-based drug products and lipoplex-based drug products; and a second module that includes fill and finish process equipment.
Consejo Superior de Investigaciones Científicas (Spain)
Universidad de Sevilla (Spain)
Inventor
Moreno Herrero, Jorge
Haas, Heinrich
Erbar, Stephanie
García Fernández, José Manuel
Ortiz Mellet, Maria Del Carmen
Benito Hernández, Juan Manuel
Lopez Fernandez, Jose
Jimenez Blanco, Jose Luis
Abstract
The present disclosure provides a compound of formula I, as well as complexes and compositions comprising a compound of formula I, wherein said compositions and complexes are useful for delivery of certain agents, including, for example, nucleic acids.
C07H 15/10 - Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of a saccharide radical containing unsaturated carbon-to-carbon bonds
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
C07H 1/00 - Processes for the preparation of sugar derivatives
TRON-TRANSLATIONALE ONKOLOGIE AN DER UNIVERSITÄTSMEDIZIN DER JOHANNES GUTENBE (Germany)
Inventor
Sahin, Ugur
Weber, David
Walter, Carina
Barea Roldán, Diana
Kuner, Ruprecht
Hein, Melanie
Suchan,, Martin
Kissler, Vetena
Kranz, Lena Mareen
Abstract
This disclosure relates to the field of RNA to treat lung cancer, in particular non-small-cell lung carcinoma (NSCLC). Lung cancer is the third most frequent malignancy in women and the second most frequent malignancy in men. NSCLC accounts for about 85% of all lung cancers. Disclosed herein are compositions, uses, and methods for treatment of lung cancers. Administration of therapeutic RNAs to a patient having lung cancer disclosed herein can reduce tumor size, prolong time to progressive disease, and/or protect against metastasis and/or recurrence of the tumor and ultimately extend survival time.
C07H 15/04 - Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of a saccharide radical
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
C07H 1/00 - Processes for the preparation of sugar derivatives
33.
TLR7 AGONIST AND COMBINATIONS FOR CANCER TREATMENT
The present disclosure relates to the specific TLR7 agonist BNT411, such as compositions and kits comprising BNT411, and the use of BNT411 as immunotherapeutic agent in monotherapy and combination therapy to reduce or prevent progression of a tumor, in particular a solid tumor, or treating cancer, in particular a solid cancer.
A61K 31/4745 - QuinolinesIsoquinolines ortho- or peri-condensed with heterocyclic ring systems condensed with ring systems having nitrogen as a ring hetero atom, e.g. phenanthrolines
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/555 - Heterocyclic compounds containing heavy metals, e.g. hemin, hematin, melarsoprol
A61K 31/7048 - Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin
A61K 39/00 - Medicinal preparations containing antigens or antibodies
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
34.
MULTISPECIFIC BINDING AGENTS AGAINST PD-L1 AND CD137 IN COMBINATION WITH ANTI PD-1 ANTIBODIES FOR TREATING CANCERS
The present disclosure relates to combination therapy using a binding agent that binds to human PD-L1 and to human CD 13 7 in combination with pembrolizumab to reduce or prevent progression of a tumor or treating cancer.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
The present disclosure relates to RNA particles comprising phospholipids with a phosphatidylserine head group for delivering RNA to target tissues after administration, in particular after parenteral, intratumoral, or peritumoral administration, and compositions comprising such RNA particles. The present disclosure also relates to methods for preparing RNA particles described herein.
The present invention relates to antibodies capable of binding to human OX40 and to variants thereof comprising a modified Fc region comprising at least one mutation that enhances the Fc-Fc interaction of the antibody and at least one mutation that reduces the Fc effector functions of the antibody. The invention further provides pharmaceutical compositions comprising the antibodies and use of the antibodies for therapeutic and diagnostic procedures, in particular in cancer therapy.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
This disclosure relates to the field of therapeutic RNA, in particular to treat cancer. Disclosed herein are compositions, uses, and methods for reducing an unwanted response or reaction, or both, in a subject, to RNA encoding an amino acid sequence comprising a cytokine protein.
Disclosed herein are RNA polynucleotides comprising a 5′ Cap, a 5′ UTR comprising a cap proximal sequence disclosed herein, and a sequence encoding a payload. Also disclosed herein are compositions and medical preparations comprising the same, and methods of making and using the same.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
B82Y 5/00 - Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/67 - General methods for enhancing the expression
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
This disclosure relates to the field of preventing or treating virus infection, in particular, the disclosure relates to agents for vaccination against virus infection and inducing effective virus antigen-specific immune responses such as antibody and/or T cell responses and methods for generating and using such agents. Administration of agents such as RNA disclosed herein to a subject can protect the subject against virus infection. Specifically, the present disclosure relates to amino acid sequences comprising at least a portion of a virus protein having amino acid modifications found in other variants of the virus protein. Administration of RNA encoding one or more of the amino acid sequences may provide protection against diverse virus variants. Methods and agents described herein are, in particular, useful for the prevention or treatment of coronavirus infection such as SARS-CoV-2 infection.
The present invention relates to antibodies capable of binding to human OX40 and to variants thereof comprising a modified Fc region comprising at least one mutation that enhances the Fc-Fc interaction of the antibody and at least one mutation that reduces the Fc effector functions of the antibody. The invention further provides pharmaceutical compositions comprising the antibodies and use of the antibodies for therapeutic and diagnostic procedures, in particular in cancer therapy.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
41.
ANTIBODIES CAPABLE OF BINDING TO OX40, VARIANTS THEREOF AND USES THEREOF
The present invention relates to antibodies capable of binding to human OX40 and to variants thereof comprising a modified Fc region comprising at least one mutation that enhances the Fc-Fc interaction of the antibody and at least one mutation that reduces the Fc effector functions of the antibody. The invention further provides pharmaceutical compositions comprising the antibodies and use of the antibodies for therapeutic and diagnostic procedures, in particular in cancer therapy.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
An antibody-drug conjugate (ADC) is provided and comprises an antibody which binds to sialyl Tn (STn) or a glycan terminated by an alpha 2,6-linked sialic acid, conjugated to a drug via a linker, wherein the linker comprises a cleavable linker, and the drug comprises a growth inhibitory agent which is exatecan, deruxtecan, or a derivative thereof. The antibody-drug conjugate (ADC) of the invention comprises a linker cleavable by glucuronidase, for examples the linker comprises a β-glucuronide moiety, such as shown in formula I: Formula (I). The antibody-drug conjugate (ADC) comprises a linker which is linked to, or is modified to link to, the drug via a carbamate linkage, or a linker which is linked to, or is modified to link to, the drug via a quaternary ammonium salt linkage. The antibody-drug conjugate (ADC) may comprise a linker which is PEGylated with a group comprising polyethylene glycol (PEG). Novel linkers for use in ADCs are also described.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
43.
METHOD OF ANALYSING CONTAMINANTS IN RNA PRODUCTS BY ION-PAIR CHROMATOGRAPHY
A method of analysis of substances which are contaminants in a sample comprising target RNA, typically prepared by in vitro transcription, the method comprising the steps of: a) preparing a sample comprising the target RNA; b) separating the target RNA from the substances by liquid chromatography, wherein the liquid chromatography is ion-pair reversed-phase chromatography, wherein the ion pair comprises a primary (C3-8)alkylamine or a salt thereof; and c) analysis of the substances by one or both of: i) ultraviolet-visible spectroscopy and ii) mass spectroscopy is provided.
C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
C12N 9/36 - Hydrolases (3.) acting on glycosyl compounds (3.2) acting on beta-1, 4 bonds between N-acetylmuramic acid and 2-acetylamino 2-deoxy-D-glucose, e.g. lysozyme
A61K 31/7056 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing five-membered rings with nitrogen as a ring hetero atom
A61K 31/78 - Polymers containing oxygen of acrylic acid or derivatives thereof
This disclosure relates to the field of RNA to prevent or treat coronavirus infection. In particular, the present disclosure relates to methods and agents for vaccination against coronavirus infection and inducing effective coronavirus antigen-specific immune responses such as antibody and/or T cell responses.
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of Plasmodium circumsporozoite protein (CSP) antigens and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides comprising a coding sequence that encodes a full-length Plasmodium CSP polypeptide. In some embodiments, a full-length Plasmodium CSP polypeptide comprises a Plasmodium CSP N-terminal domain, and the coding sequence of a polyribonucleotide encoding the full-length Plasmodium CSP polypeptide has a particular nucleotide content as provided herein.
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
47.
PHARMACEUTICAL COMPOSITIONS FOR DELIVERY OF VIRAL ANTIGENS AND RELATED METHODS
The present disclosure provides pharmaceutical compositions for delivery of viral antigens (e.g., a viral vaccine) and related technologies (e.g., components thereof and/or methods relating thereto).
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of malarial circumsporozoite protein (CSP) antigens and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides comprising a coding sequence that encodes a full length Plasmodium CSP polypeptide. In some embodiments, a full length Plasmodium CSP polypeptide comprises a Plasmodium CSP N-terminal domain, and the coding sequence of a polyribonucleotide encoding the full length Plasmodium CSP polypeptide has a particular nucleotide content as provided herein.
The present disclosure, among other things, provides technologies for identifying, characterizing, and/or monitoring variant sequences of a particular reference infections agent. Among other things, systems, methods, and architectures described herein provide visualization and decision support tools that can, e.g., facilitate decision making processes by local authorities and improve pandemic response in terms of, e.g., resource allocation, policy making, and speed tailored vaccine development. The present disclosure also provides tools for analyzing circulating variants to predict mutations likely to increase immune evasion of infectious agents.
G16B 45/00 - ICT specially adapted for bioinformatics-related data visualisation, e.g. displaying of maps or networks
G16B 50/30 - Data warehousingComputing architectures
G16H 50/80 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for detecting, monitoring or modelling epidemics or pandemics, e.g. flu
The disclosure provides agents and methods for preventing or treating tuberculosis using RNA. The RNA encoding antigens of Mycobacterium tuberculosis, immunogenic variants or fragments thereof is formulated and administered in a way that the antigens, variants or fragments are produced by cells of a subject.
A61K 39/04 - Mycobacterium, e.g. Mycobacterium tuberculosis
A61P 31/06 - Antibacterial agents for tuberculosis
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
C12N 15/67 - General methods for enhancing the expression
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY (USA)
UNIVERSITY OF CAPE TOWN (South Africa)
Inventor
Ates, Louis Simon
Uebele, Julia
Agrawal, Neha
Dulberger, Charles Lefco
Diao, Huitian
Poran, Asaf
Manandhar, Dinesh
Rothenberg, Daniel Abram
Scriba, Thomas
Musvosvi, Munyaradzi
Obermoser, Gerlinde
Davis, Mark
Huang, Huang
Abstract
The disclosure provides agents and methods for preventing or treating tuberculosis using RNA. The RNA encoding chimeric proteins comprising antigens of Mycobacterium tuberculosis, immunogenic variants or fragments thereof is formulated and administered in a way that the antigens, variants or fragments are produced by cells of a subject.
The present invention concerns compositions comprising an RNA molecule, wherein the RNA molecule comprises an expression cassette encoding an immunogenic peptide, which peptide comprises at least two fragments, wherein each fragment comprises at least one epitope, wherein the epitope is derived from an amino acid sequence encoded by the human immunodeficiency virus (HIV) and medical preparations comprising such compositions. Furthermore, the invention concerns methods for preventing or treating HIV.
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY (USA)
UNIVERSITY OF CAPE TOWN (South Africa)
Inventor
Schille, Stefan Albrecht
Scriba, Thomas
Musvosvi, Munyaradzi
Obermoser, Gerlinde
Davis, Mark
Huang, Huang
Abstract
The disclosure provides agents and methods for preventing or treating tuberculosis using RNA. The RNA encoding antigens of Mycobacterium tuberculosis, immunogenic variants or fragments thereof is formulated and administered in a way that the antigens, variants or fragments are produced by cells of a subject.
A method of preparing a combination vaccine includes: (a) obtaining a first amount of a first vaccine in a first syringe, (b) obtaining a second amount of a second vaccine In a second syringe, (c) connecting the first and the second syringe using an adapter, (d) mixing the first vaccine and the second vaccine, and (e) transferring the mixture of the first vaccine and the second vaccine, as needed, to the first syringe or the second syringe, and discarding the syringe that the mixture was not transferred to.
This disclosure relates to the field of therapeutic RNA to treat cancer, in particular advanced solid tumors such as metastatic (Stage IV) or unresectable localized cancer. Disclosed herein are compositions, uses, and methods for treatment of cancers. Administration of therapeutic RNAs to a patient having cancer disclosed herein can reduce tumor size, prolong time to progressive disease, and/or protect against metastasis and/or recurrence of the tumor and ultimately extend survival time.
The invention provides a fluid path system, in particular for aseptic processing of at least a first fluid and a second fluid, the fluid path system comprising at least a first tubing unit and a second tubing unit, wherein the first tubing unit is configured to receive the first fluid from a first reservoir and the second tubing unit is configured to receive the second fluid from a second reservoir, a mixing element configured to combine the first fluid and the second fluid to provide a mixture of the first fluid and the second fluid and to discharge the mixture of the first fluid and the second fluid, where filling and emptying the tubes is obtained by modification of the air or gas pressure inside the tubings by gas-filled syringes which are connected to the tubings through sterile filters, such that only sterile air or gas will come into connection with the fluids in the tubings. Furthermore, a mixing system and a corresponding method for mixing of at least two fluids is provided. The invention allows for an aseptic and precise mixing of fluids or fluid phases, in particular of two or more fluids or fluid phases.
The present disclosure provides technologies for identifying, characterizing, and/or monitoring sequences of a variant of a reference infectious agent (e.g., but not limited to viral variants, for example in some embodiments SARS-CoV-2 variants) for transmissibility factors and/or immune escape potential, and/or for detecting and/or monitoring variants in environmental or biological samples, and/or for designing, preparing, and/or administering vaccines for such variants.
A system for heating and/or cooling a vessel includes: a heating and cooling jacket disposed around the vessel and including (1) a plurality of channels configured to allow a thermal fluid to flow therethrough, (2) at least one fluid inlet, and (3) at least one fluid outlet, each fluidly coupled to the plurality of channels; and a temperature handling unit fluidly coupled to the jacket and configured to selectively heat and cool the thermal fluid.
The present invention relates to therapy using a binding agent that binds to human PD-L1 and to human CD137 to reduce or prevent progression of a tumor or treating cancer.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
60.
MULTISPECIFIC BINDING AGENTS AGAINST CD40 AND CD137 IN COMBINATION THERAPY FOR CANCER
The present invention relates to combination therapy using a binding agent that binds to human CD40 and to human CD137 in combination with a checkpoint inhibitor to reduce or prevent progression of a tumor or treating cancer.
The present invention provides a prognostic marker in human cancer, CLDN18. Methods involving this marker are disclosed for predicting therapeutic efficacy of cancer treatments and the prognosis of cancer. The present invention also provides a method for selecting a subject affected with a cancer for an immunotherapy. Furthermore, the present invention provides a therapy for treating cancer.
The present disclosure, among other things, provides technologies for identifying, characterizing, and/or monitoring variant sequences of a particular reference infections agent. Among other things, systems, methods, and architectures described herein provide visualization and decision support tools that can, e.g., facilitate decision making processes by local authorities and improve pandemic response in terms of, e.g., resource allocation, policy making, and speed tailored vaccine development. The present disclosure also provides tools for analyzing circulating variants to predict mutations likely to increase immune evasion of infectious agents.
The present disclosure, among other things, provides technologies for identifying, characterizing, and/or monitoring variant sequences of a particular reference infections agent. Among other things, systems, methods, and architectures described herein provide visualization and decision support tools that can. e.g., facilitate decision making processes by local authorities and improve pandemic response in terms of, e.g., resource allocation, policy making, and speed tailored vaccine development. The present disclosure also provides tools for analyzing circulating variants to predict mutations likely to increase immune evasion of infectious agents.
The present invention provides a prognostic marker in human cancer, CLDN18. Methods involving this marker are disclosed for predicting therapeutic efficacy of cancer treatments and the prognosis of cancer. The present invention also provides a method for selecting a subject affected with a cancer for an immunotherapy. Furthermore, the present invention provides a therapy for treating cancer.
The present disclosure relates generally to the field of stabilized compositions comprising particles dispersed in an aqueous phase, wherein the aqueous phase comprises a buffer system and has a pH of about 4.0-5.5 and wherein the particles contain (i) nucleic acid (such as DNA or RNA, in particular mRNA or inhibitory RNA, e.g., siRNA); and (ii) a cationic or cationically ionizable lipid, methods for preparing and storing such compositions, and the use of such compositions in therapy.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 47/50 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
A61K 47/16 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen
A61K 47/24 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
A61K 47/28 - Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
A method is provided for identifying a number of variants of concern of a reference disease associated immunogen. The method uses a language model to perform inference on data representing each of a plurality of variants and data representing the reference immunogen. For each of the plurality of variants and the reference immunogen, a characteristic vector is derived from an output feature map of a hidden layer of the language model. For each of the plurality of variants, a measure of distance is generated for the variant that includes calculating a measure of distance between the characteristic vector of the variant and the characteristic vector of the reference immunogen. A semantic change score is calculated for each variant based on the generated measure of distance for that variant. A variant of the reference immunogen is selected based, at least in part, on the generated the semantic change scores.
The present disclosure relates generally to the field of stabilized compositions comprising particles dispersed in an aqueous phase, wherein the aqueous phase comprises a buffer system and has a pH of about 4.0-5.5 and wherein the particles contain (i) nucleic acid (such as DNA or RNA, in particular mRNA or inhibitory RNA, e.g., siRNA); and (ii) a cationic or cationically ionizable lipid, methods for preparing and storing such compositions, and the use of such compositions in therapy.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 47/50 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
A61K 47/16 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen
A61K 47/24 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
A61K 47/28 - Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
An automated drug production process includes: performing in vitro DNA transcription via an automated in vitro transcription (IVT) module, thereby producing an RNA solution to be used for producing drug substance; performing at least one tangential flow filtration (TFF) step on the RNA solution; providing a stream of lipid stock; mixing the stream of lipid stock within the RNA solution in an impingement jet mixer, thereby forming an RNA lipid nanoparticle (LNP) preparation; and formulating the RNA-LNP preparation to produce a drug product.
The present invention is generally in the fields of pharmaceuticals, in particular antibacterials, and protein engineering. In particular, the present invention relates to peptidoglycan hydrolases such as endolysins and nucleic acids, e.g., RNAs, encoding the peptidoglycan hydrolases of the invention, as well as medical uses thereof, for example, for treating diseases caused by and/or associated with a Staphylococcus (e.g., S. aureus) infection. Furthermore, the present invention relates to solidified yeast culture media for screening yeast cells for the secretion of peptidoglycan hydrolases with bactericidal activity and corresponding screening methods.
Compounds disclosed herein have been prepared for use in the treatment of diseases, disorders or conditions treatable by activation of the stimulator of interferon genes (STING) adaptor protein, such as in the treatment or prevention of cancers.
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
C07D 417/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
The invention provides potency assays for simultaneously analyzing at least two different nucleic acid sequences such as RNA and/or DNA sequences encoding a functional sequence such as an antigen or epitope. The potency assays of the present invention may be performed with nucleic acid sequences encoding at least two different functional sequences, including at least two different antigens or epitopes.
Compounds of formula (IA") disclosed herein have been prepared for use in the treatment of diseases, disorders or conditions treatable by activation of the stimulator of interferon genes (STING) adaptor protein, such as in the treatment or prevention of cancers.
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
C07D 471/02 - Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups in which the condensed system contains two hetero rings
The invention provides potency assays for simultaneously analyzing at least two different nucleic acid sequences such as RNA and/or DNA sequences encoding a functional sequence such as an antigen or epitope. The potency assays of the present invention may be performed with nucleic acid sequences encoding at least two different functional sequences, including at least two different antigens or epitopes.
in-silicoin-silico design of custom, engineered, antigens. In particular, in certain embodiments, methods and system of the present disclosure provide for engineering antigens to reduce their activation of a memory immune response, such as B cell and/or T cell based response, when introduced into a subject. Designing antigens in this manner, may, for example, improve their performance as immunogenic compositions for purposes of vaccination. For example, without wishing to be bound to any particular theory, it is believed that, among other things, reducing the extent to which a memory immune response is triggered can lead to improved production of new antibodies that are selectively tailored by the subject's immune system to neutralize particular (e.g., arisen) epitopes of a reference antigen.
Disclosed herein are compositions comprising protein antigens and RNA encoding the same (e.g., compositions comprising protein antigens and RNA encoding antigens) for use in treating and/or preventing various infectious agents. Also disclosed herein are immunogenic compositions and medical preparations comprising the same, and methods of making and using the same. In some embodiments, the technologies provided herein can be used to address and/or overcome immune imprinting in various infectious diseases.
A fluid handling system (100), comprising a fluid path system, the fluid path system comprising a first fluid path (202) for guiding a first fluid substance towards a mixing region (300) of the fluid path system, a second fluid path (204) for guiding a second fluid substance towards the mixing region, wherein the mixing region is arranged to be in fluid communication with the first fluid path and the second fluid path such that the first fluid substance and the second fluid substance can be mixed in the mixing region to form a fluid substance mixture, wherein the fluid handling system comprises an unguided flow region (400), wherein the unguided flow region is configured such that the fluid substance mixture can be or is prevented from contacting an inner wall (402, 404) suitable for guiding a flow of the fluid substance mixture along a flow direction (AR4) of the fluid substance mixture while the fluid substance mixture travels through the unguided flow region, and wherein the unguided flow region extends for a predetermined distance, e.g. extends only for the predetermined distance (d1), downstream of the mixing region.
B01F 23/45 - Mixing liquids with liquidsEmulsifying using flow mixing
B01F 25/433 - Mixing tubes wherein the shape of the tube influences the mixing, e.g. mixing tubes with varying cross-section or provided with inwardly extending profiles
A fluid handling system, comprising a fluid path system, the fluid path system comprising a first fluid path for guiding a first fluid substance towards a mixing region of the fluid path system, a second fluid path for guiding a second fluid substance towards the mixing region, wherein the mixing region is arranged to be in fluid communication with the first fluid path and the second fluid path such that the first fluid substance and the second fluid substance can be mixed in the mixing region to form a fluid substance mixture, wherein the fluid handling system comprises an unguided flow region, wherein the unguided flow region is configured such that the fluid substance mixture can be or is prevented from contacting an inner wall suitable for guiding a flow of the fluid substance mixture along a flow direction of the fluid substance mixture while the fluid substance mixture travels through the unguided flow region, and wherein the unguided flow region extends for a predetermined distance, e.g. extends only for the predetermined distance, downstream of the mixing region.
B01F 23/45 - Mixing liquids with liquidsEmulsifying using flow mixing
B01F 25/433 - Mixing tubes wherein the shape of the tube influences the mixing, e.g. mixing tubes with varying cross-section or provided with inwardly extending profiles
79.
PREPARATION AND STORAGE OF LIPOSOMAL RNA FORMULATIONS SUITABLE FOR THERAPY
The present disclosure relates to methods for preparing RNA lipoplex particles for delivery of RNA to target tissues after parenteral administration, in particular after intravenous administration, and compositions comprising such RNA lipoplex particles. The present disclosure also relates to methods which allow preparing RNA lipoplex particles in an industrial GMP-compliant manner. Furthermore, the present disclosure relates to methods and compositions for storing RNA lipoplex particles without substantial loss of the product quality and, in particular, without substantial loss of RNA activity.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharidesDerivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
80.
COMBINATION DOSAGE REGIME OF CD137 AND PD-L1 BINDING AGENTS
The present invention relates to a method for reducing or preventing progression of a tumor or treating cancer in a subject, comprising administering to the subject a binding agent comprising a first antigen-binding region binding to human CD137, and a second antigen-binding region binding to human PD-L1. The invention further provides a binding agent for use in reducing or preventing progression of a tumor or for use in treatment of cancer.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
An aqueous dispersion having an aqueous mobile phase and a dispersed phase, wherein: the dispersed phase comprises a lipid mixture including a cationically ionizable lipid; and the aqueous mobile phase comprises a buffer solution having a pH from about 6.5 to about 8; wherein the aqueous dispersion is substantially free of inorganic cations, organic solvents and nucleic acids, is described. Methods of preparing the aqueous dispersion, nucleic acid-lipid particles and methods of preparing them using the aqueous dispersion, and their use in medicine are disclosed.
An aqueous dispersion having an aqueous mobile phase and a dispersed phase; wherein the dispersed phase comprises a lipid mixture including a cationically ionisable lipid; and the aqueous mobile phase comprises an anion of an aqueous acid; wherein the aqueous dispersion is substantially free of inorganic cations, organic solvents and RNA, is described. Methods of preparing the aqueous dispersion, nucleic acid-lipid particles and methods of preparing them using the aqueous dispersion, and their use in medicine are disclosed.
This invention relates to methods for screening for a genotype for loss of antigen presentation via MHC class I in a subject and/or respectively detecting a subject's increased risk of resistance against immunotherapy such as against vaccination.
A method of forming an aqueous dispersion, the method comprising mixing: (i) an organic phase comprising a lipid mixture comprising a cationic or cationically ionisable lipid dissolved in a water-soluble organic solvent; and (ii) an aqueous phase, the aqueous phase comprising an aqueous amino acid, or a mixture thereof; to produce the aqueous dispersion, is disclosed. Aqueous dispersions prepared by the method, nucleic acid-lipid particles and methods of preparing them using the aqueous dispersion, and their use in medicine, are also disclosed.
The present disclosure provides pharmaceutical compositions for delivery of HSV antigens (e.g., an HSV vaccine) and related technologies (e.g., components thereof and/or methods relating thereto).
The present invention relates to binding agents binding to receptors of the TNF superfamily, in particular binding agents binding to at least two different receptors of the TNF superfamily, as well as to their use in medicine. The present invention further relates to nucleic acid molecules encoding such binding agents, to cells comprising such nucleic acid molecules and to pharmaceutical compositions and kits.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
The present disclosure relates to RNA comprising one or more miRNA binding sequences, wherein the one or more miRNA binding sequences bind to miRNA that is present in cells in which expression of the RNA is not desired. Delivering the RNA to cells after administration, in particular after intramuscular or intravenous administration, allows expression of a polypeptide encoded by the RNA in certain cells while expression in other cells is repressed. In some embodiments, such cells comprise endothelial cells. RNA compositions described herein allow expression of a pharmaceutically active peptide or polypeptide by the RNA in a subject while reducing or avoiding the risks of undesired effects resulting from expression of the pharmaceutically active peptide or polypeptide in certain cells or tissues.
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
The present disclosure relates to RNA comprising one or more miRNA binding sequences, wherein the one or more miRNA binding sequences bind to miRNA that is present in cells in which expression of the RNA is not desired. Delivering the RNA to cells after administration, in particular after intramuscular or intravenous administration, allows expression of a polypeptide encoded by the RNA in certain cells while expression in other cells is repressed. In some embodiments, such cells comprise endothelial cells. RNA compositions described herein allow expression of a pharmaceutically active peptide or polypeptide by the RNA in a subject while reducing or avoiding the risks of undesired effects resulting from expression of the pharmaceutically active peptide or polypeptide in certain cells or tissues.
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
89.
RNA COMPOSITIONS COMPRISING A BUFFER SUBSTANCE AND METHODS FOR PREPARING, STORING AND USING THE SAME
The present disclosure relates generally to the field of RNA compositions comprising a buffer substance, methods for preparing and storing such compositions, and the use of such compositions in therapy.
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
The present disclosure provides compositions (e.g., pharmaceutical compositions) for delivery of anti-HIV antibody agents and related technologies (e.g., components thereof and/or methods relating thereto). Among other things, the present disclosure provides polyribonucleotides encoding an immunoglobulin chain of an anti-HIV antibody agent.
A composition comprising: (a) an active ingredient; and (b) a lipid mixture comprising: (i) a cationically ionisable lipid capable of forming a lipid nanoparticle; (ii) a steroid; and (iii) a negatively charged amphiphile having a hydrophilic portion and a lipophilic portion; wherein the composition is a lipid nanoparticle composition and is substantially free of a polyethylene glycol-conjugated lipid, wherein the polyethylene glycol (PEG) moiety of the PEG-conjugated lipid has at least 5 consecutive ethylene glycol repeating units, is provided.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
92.
METHODS OF TREATING PANCREATIC CANCER WITH A PD-1 AXIS BINDING ANTAGONIST AND AN RNA VACCINE
The present disclosure provides methods for treating an individual with pancreatic cancer with an individualized cancer vaccine and a PD-1 axis antagonist.
The present invention provides a combination therapy for effectively treating and/or preventing diseases associated with cells expressing CAI 9-9, including cancer diseases such as pancreatic cancer and metastases thereof.
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
A61K 31/4745 - QuinolinesIsoquinolines ortho- or peri-condensed with heterocyclic ring systems condensed with ring systems having nitrogen as a ring hetero atom, e.g. phenanthrolines
A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 31/555 - Heterocyclic compounds containing heavy metals, e.g. hemin, hematin, melarsoprol
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
The present invention relates to combination therapy using a binding agent that binds to human CD40 and to human CD137 in combination with a checkpoint inhibitor which is an inhibitor of the PD-1/PD- L1 axis (in particular pembrolizumab) and chemotherapy to reduce or prevent progression of head and neck squamous cell carcinoma (HNSCC) or treat HNSCC.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
The present disclosure relates generally to the field of multispecific binding agents for use in therapy, in particular for use in treating cancer, wherein the binding agents bind to human CD40 and to human CD137.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 39/00 - Medicinal preparations containing antigens or antibodies