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3589results about "Active genetic ingredients" patented technology
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Use of Chimeric Antigen Receptor-Modified T-Cells to Treat Cancer
ActiveUS20130287748A1Stimulate immune responseVirusesPeptide/protein ingredientsBinding domainAntigen binding
The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.
Owner:THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
Regulation of endogenous gene expression in cells using zinc finger proteins
InactiveUS7013219B2Fusion with DNA-binding domainAntibody mimetics/scaffoldsZinc fingerGene expression
The present invention provides methods for modulating expression of endogenous cellular genes using engineered zinc finger proteins.
Owner:SANGAMO BIOSCIENCES INC
Pharmaceutical composition containing a stabilised mRNA optimised for translation in its coding regions
ActiveUS20050032730A1Overcome disadvantagesImprove efficiencyAntibacterial agentsVirusesTranslational efficiencyCoding region
The present invention relates to a pharmaceutical composition comprising a modified mRNA that is stabilised by sequence modifications and optimised for translation. The pharmaceutical composition according to the invention is particularly well suited for use as an inoculating agent, as well as a therapeutic agent for tissue regeneration. In addition, a process is described for determining sequence modifications that promote stabilisation and translational efficiency of modified mRNA of the invention.
Owner:CUREVAC SE
Method and compositions using a chimeric antigen receptor for enhanced anti-tumor effector functioning of T cells
Integration of costimulatory signaling domains within a tumor targeting chimeric antigen receptor (CAR), such as the IL13Rα2 specific IL13-zetakine (IL13ζ), enhances T cell-mediated responses against tumors even in the absence of expressed ligands for costimulatory receptors.
Owner:CITY OF HOPE
Modified polynucleotides for the production of secreted proteins
Owner:MODERNATX INC
RNA preparations comprising purified modified RNA for reprogramming cells
ActiveUS20110143397A1Promote growthArtificial cell constructsCell culture active agentsSingle strandSomatic cell
The present invention provides compositions and methods for reprogramming somatic cells using purified RNA preparations comprising single-strand mRNA encoding an iPS cell induction factor. The purified RNA preparations are preferably substantially free of RNA contaminant molecules that: i) would activate an immune response in the somatic cells, ii) would decrease expression of the single-stranded mRNA in the somatic cells, and / or iii) active RNA sensors in the somatic cells. In certain embodiments, the purified RNA preparations are substantially free of partial mRNAs, double-stranded RNAs, un-capped RNA molecules, and / or single-stranded run-on mRNAs.
Owner:THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
Delivery of mRNA for the augmentation of proteins and enzymes in human genetic diseases
InactiveUS20110244026A1Facilitating transfectionReduce deliveryOrganic active ingredientsDigestive systemDiseaseADAMTS Proteins
Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.
Owner:TRANSLATE BIO INC
RNA interference for the treatment of heart failure
InactiveUS8404658B2Reduce expressionDecreasing ventricular arrhythmiasOrganic active ingredientsTissue culturePhospholambanTransfection
The present invention relates to targeted RNAi for the treatment of heart failure by modulating defective cardiac Ca2+ homeostasis via decreasing expression or activity of phospholamban (PLB) using adeno-associated virus (AAV) transfection of cardiomyocytes. Methods for decreasing ventricular arrhythmias, as well as methods for overall improvement of survival from heart failure in subjects are also disclosed. Further, the present invention provides methods which can be used to diagnose susceptibility to treatment by RNAi, and includes pharmaceutical compositions, kits and vectors including an RNAi sequence.
Owner:NANOCOR THERAPEUTICS +1
Liver specific delivery of messenger RNA
Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.
Owner:TRANSLATE BIO INC
Modified nucleoside, nucleotide, and nucleic acid compositions
InactiveUS20130156849A1Therapeutically effectiveRaise hemoglobinOrganic active ingredientsPowder deliveryProtein precursorNucleotide
The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and / or activity.
Owner:MODERNA THERAPEUTICS INC
Treatment regimen for parkinson's disease
InactiveUS20120295960A1Reduce potential side effectsReduce maintenanceNervous disorderNucleic acid vectorSide effectCombination therapy
Provided is an improved treatment for Parkinson's Disease where the efficacy of L-Dopa treatment is increased by including gene therapy in the treatment regimen. The combination therapy results in long-term improvements in response to L-Dopa and diminished side effects caused by L-Dopa.
Owner:OXFORD BIOMEDICA (UK) LTD
Method for vector delivery
InactiveUS20110293571A1Optimize volumeTotal volume of vector distribution in the brain wasBiocideOrganic active ingredientsNeuroscience
Owner:OXFORD BIOMEDICA (UK) LTD
PHARMACEUTICAL COMPOSITION CONTAINING A STABILISED mRNA OPTIMISED FOR TRANSLATION IN ITS CODING REGIONS
InactiveUS20100239608A1Improve efficiencyOvercome disadvantagesAntibacterial agentsOrganic active ingredientsCoding regionBiology
Owner:CUREVAC AG
Compositions and methods for treatment of neoplastic disease
InactiveUS20050112141A1High productFacilitates their targetingFusions for specific cell targetingReceptors for cytokines/lymphoines/interferonsAbnormal tissue growthDisease
The present invention comprises compositions and methods for treating a tumor or neoplastic disease in a host, The methods employ conjugates comprising superantigen polypeptides or nucleic acids with other structures that preferentially bind to tumor cells and are capable of inducing apoptosis. Also provided are superantigen-glycolipid conjugates and vesicles that are loaded onto antigen presenting cells to activate both T cells and NKT cells. Cell-based vaccines comprise tumor cells engineered to express a superantigen along with glycolipids products which, when expressed, render the cells capable of eliciting an effective anti-tumor immune response in a mammal into which these cells are introduced. Included among these compositions are tumor cells, hybrid cells of tumor cells and accessory cells, preferably dendritic cells. Also provided are T cells and NKT cells activated by the above compositions that can be administered for adoptive immunotherapy.
Owner:TERMAN DAVID
Modulation of gene expression by oligomers targeted to chromosomal DNA
Owner:BOARD OF RGT THE UNIV OF TEXAS SYST
Method and compositions for enhanced Anti-tumor effector functioning of t cells
ActiveUS20120148552A1Function increaseBiocidePeptide/protein ingredientsTumor targetChimeric antigen receptor
Integration of costimulatory signaling domains within a tumor targeting chimeric antigen receptor (CAR), such as the IL13Rα2 specific IL13-zetakine (IL13ζ), enhances T cell-mediated responses against tumors even in the absence of expressed ligands for costimulatory receptors.
Owner:CITY OF HOPE
Induction of exon skipping in eukaryotic cells
InactiveUS7973015B2Efficiently maskedPotential for manipulationOrganic active ingredientsSplicing alterationPrecursor mRNARecognition sequence
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, the cell comprising pre-mRNA comprising exons coding for the protein, by inducing so-called exon skipping in the cell. Exon-skipping results in mature MRNA that does not contain the skipped exon, which leads to an altered product of the exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance, an exon recognition sequence, of the exon. The exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of the exon. The nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance, for the treatment of an inherited disease.
Owner:LEIDEN ACADEMISCH ZIEKENHUIS
Switch costimulatory receptors
ActiveUS20140219975A1BiocidePeptide/protein ingredientsChimerin ProteinsReceptor for activated C kinase 1
The present invention relates generally to a fusion protein that when displayed on a cell can convert a negative signal into a positive signal in the cell. The fusion protein is a chimeric protein in that the protein comprises at least two domains, wherein the first domain is a polypeptide that is associated with a negative signal and the second domain is a polypeptide that is associated with a positive signal. Thus, the invention encompasses switch receptors that are able to switch negative signals to positive signals for enhancement of an immune response.
Owner:THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
Split dose administration
The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and / or activity.
Owner:MODERNATX INC
Modified polynucleotides encoding cd28 molecule
Owner:MODERNA THERAPEUTICS INC
Methods for treating cancer using cytokine-expressing polynucleotides
InactiveUS7268120B1Improved in vivo polypeptide expressionMinimizing adverse side effectBiocideOrganic active ingredientsMammalSodium phosphates
The present invention provides a pharmaceutical composition, comprising a non-infectious, non-integrating polynucleotide construct comprising a polynucleotide encoding an interferon ω and one or more cationic compounds. The present invention also provides methods of treating cancer in a mammal, comprising administering into a muscle of the mammal a non-infectious, non-integrating DNA polynucleotide construct comprising a polynucleotide encoding a cytokine. In addition, the present invention also relates to the methodology for selective transfection of malignant cells with polynucleotides expressing therapeutic or prophylactic molecules in intra-cavity tumor bearing mammals. More specifically, the present invention provides a methodology for the suppression of an intra-cavity dissemination of malignant cells, such as intraperitoneal dissemination. Furthermore, the invention relates to compositions and methods to deliver polynucleotides encoding polypeptides to vertebrate cells in vivo, where the composition comprises an aqueous solution of sodium phosphate.
Owner:VICAL INC
Methods for Treatment of Cancer
The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.
Owner:THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
Compositions for Treatment of Cancer
The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.
Owner:THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
RNA containing composition for treatment of tumor diseases
ActiveUS20160331844A1Improve survivalEffectively treating tumor and/or cancer diseasesSsRNA viruses negative-senseOrganic active ingredientsDiseasePharmaceutical drug
Owner:CUREVAC SE
Methods and compositions for treating huntington's disease
ActiveUS20150335708A1Reducing and eliminating Htt aggregateAmeliorating motor deficitPeptide librariesNervous disorderHuntingtons choreaMedicine
Owner:SANGAMO BIOSCIENCES INC +1
Genetic products differentially expressed in tumors and the use thereof
ActiveUS7527933B2Improve cell activityEfficient disseminationCompound screeningApoptosis detectionCancer researchDisease cause
The invention relates to the identification of genetic products expressed in association with tumors and to coding nucleic acids for the expressed products. An embodiment of the invention also relates to the therapy and diagnosis of disease in which the genetic products are aberrantly expressed in association with tumors, proteins, polypeptides and peptides which are expressed in association with tumors, and to the nucleic acids coding for the polypeptides, peptides and proteins.
Owner:GANYMED PHARMA AG
Adeno-Associated Virus Virion for Gene Transfer to Nervous System Cells
ActiveUS20130224836A1Improve efficiencyLower Level RequirementsNervous disorderSpecial deliveryNervous systemAdeno associate virus
The present invention provides a means for transferring a therapeutic gene of interest into a nervous system cell by a highly-efficient and simpler means. More specifically, the present invention provides a recombinant vector that uses an adeno-associated virus (AAV), a method for manufacturing the recombinant vector, and a method for using the recombinant vector. More specifically, recombinant adeno-associated virus virions, which are capable of passing through the brain-brain barrier, for transferring a therapeutic genes of interest into a nervous system cell in a highly-efficient manner, a drug composition containing the recombinant adeno-associated virus virions, a method for manufacturing the recombinant adeno-associated virus virions, and a kit or the like are provided.
Owner:JICHI MEDICAL UNIVERSITY
Oncolytic adenovirus armed with therapeutic genes
InactiveUS20060147420A1Improve efficiencyBiocideOrganic active ingredientsOncolytic adenovirusNucleotide
The present invention involves compositions and methods for treating cancer using a mutant adenovirus comprising a polynucleotide encoding a therapeutic polypeptide that is targeted to cells with a mutant retinoblastoma pathway. The mutant adenovirus is able to kill the tumor cells without harming cells with a wild type retinoblastoma pathway.
Owner:BOARD OF RGT THE UNIV OF TEXAS SYST
Compositions comprising t cell receptors and methods of use thereof
InactiveUS20090053184A1High affinityEfficient killingOrganic active ingredientsBiocideMelanomaBiology
Nucleic acids encoding antitumor TCRs recognizing MART-1, NY-ESO-1, and melanoma gp100 peptides; vectors and cells comprising the same; and methods of using the foregoing.
Owner:GOVERNMENT OF THE US REPRESENTED BY THE SEC
Combination of immuno gene therapy and chemotherapy for treatment of cancer and hyperproliferative diseases
ActiveUS7964571B2Inhibiting growth and metastasisImprove survivalHeavy metal active ingredientsPeptide/protein ingredientsGene deliveryWhole body
Owner:CLSN LAB
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