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987results about "Non-active genetic ingredients" patented technology
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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
Methods and compounds for controlled release of recombinant parvovirus vectors
InactiveUS7201898B2Prevent relapseImproved pulmonary mechanicsSuture equipmentsAntibacterial agentsControlled releaseSupport matrix
The invention uses recombinant parvoviruses, and particularly recombinant adeno-associated virus (rAAV) to deliver genes and DNA sequences for gene therapy following manipulation of the therapeutic virus for packaging and transport. The invention delivers therapeutic viral vectors via rAAV affixed to support matrixes (i.e., sutures, surgically implantable materials, grafts, and the like).
Owner:NORTH CAROLINA AT CHAPEL HILL THE UNIV OF
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
Lipid formulation
The invention features an improved lipid formulation comprising a cationic lipid of formula (A), a neutral lipid, a sterol and a PEG or PEG-modified lipid, where R1 and R2 are independently alkyl, alkenyl or alkynyl, each can be optionally substituted, and R3 and R4 are independently lower alkyl or R3 and R4 can be taken together to form an optionally substituted heterocyclic ring. In one embodiment, R1 and R2 are independently selected from oleoyl, pamitoyl, steroyl, linoleyl and R3 and R4 are methyl. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.
Owner:ARBUTUS BIOPHARMA CORPORAT ION
Compositions and methods for delivering messenger RNA
InactiveUS20160151284A1Symptoms improvedLower Level RequirementsPowder deliveryOrganic active ingredientsDiseaseLipid particle
The present invention provides compositions comprising mRNA molecules encapsulated within lipid particles. The lipid particles comprise a cationic lipid, a non-cationic lipid, and an mRNA molecule that is encapsulated within the lipid particle. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease. The invention also provides cationic lipids that are useful for preparing the compositions of the invention.
Owner:ARBUTUS BIOPHARMA CORPORAT ION
Peptide having cell membrane penetrating activity
InactiveUS20130129726A1Prominent penetrating efficiencyTargeting of drugs is required high efficientlyPeptide/protein ingredientsDrug compositionsHistamine releasing factorProtein transduction domain
Provided are transmembrane complexes that contain a protein transduction domain (PTD) from the N-terminus of IgE-dependent histamine-releasing factor (HRF) and a target substance that is to be delivered into a cell. Also provided are nucleic acid molecules encoding the transmembrane complex, and methods of delivering the target substance into a cell interior by contacting the transmembrane complex with a cell. Also provided are transfection kits containing the PTD and the target substance.
Owner:EWHA UNIV IND COLLABORATION FOUND
Adeno-associated virus vectors
InactiveUS6897045B2Improve stabilityIncreased episomal stabilityOrganic active ingredientsBiocideVirusAdeno-associated virus
The invention provides an isolated and purified DNA molecule comprising at least one DNA segment, a biologically active subunit or variant thereof, of a circular intermediate of adeno-associated virus, which DNA segment confers increased episomal stability, persistence or abundance of the isolated DNA molecule in a host cell. The invention also provides a composition comprising at least two adeno-associated virus vectors.
Owner:UNIV OF IOWA RES FOUND
Composition comprising gas-filled microcapsules for ultrasound mediated delivery
InactiveUS20090041833A1Efficient deliveryPowder deliveryDrug compositionsLipid formationActive agent
Composition comprising gas filled microcapsules and a bioactive agent, useful for an ultrasound-mediated delivery of said bioactive agent. The microcapsules comprise a relatively stiff shell of polymeric or lipid material and have in particular a resistance to a mechanical index of at least 0.15, while the bioactive agent is substantially unbound to the shell of the microcapsules. The composition of the invention is particularly suitable for effectively delivering a genetic material into a cell, upon exposure of the composition to a level of acoustic pressure capable of destroying a portion of the microcapsules and releasing the gas contained therein.
Owner:BRACCO RES USA
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
Isolation Of Novel AAV'S And Uses Thereof
ActiveUS20100186103A1Determine effectAvoid time-consume and costly processVectorsSpecial deliveryAdeno associate virusBinding site
The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.
Owner:UNIV OF MASSACHUSETTS
Compositions and methods for delivering nucleic acid to a cell
InactiveUS20140065204A1Efficient transfectionSpecial deliveryGenetic therapy composition manufactureCell biologyNucleic acid
Owner:MERRIMACK PHARMACEUTICALS INC
Localized delivery of nucleic acid by polyelectrolyte assemblies
ActiveUS20060251701A1Strong therapeutic activityReduce inflammationSurgeryNon-active genetic ingredientsPolyelectrolyteMedical device
The present invention provides implantable medical devices coated with polyelectrolyte assemblies that are fabricated by layer-by-layer deposition of nucleic acid and polycation. Such devices facilitate the local delivery of a nucleic acid contained in the polyelectrolyte assembly into a cell or tissue at an implantation site. Also provided are methods of fabricating and using implantable medical devices according to the invention.
Owner:WISCONSIN ALUMNI RES FOUND
Amine-containing transfection reagents and methods for making and using same
There are provided for herein novel amine-containing transfection compounds and methods for making and using same. The compounds are generally obtained by reacting a primary amine with an unsaturated compound. Transfection complexes made using the amine-containing transfection compounds in combination with additional compounds to encapsulate biologically active agents such as nucleic acids are also provided for herein. Methods of using the transfection complexes for the in vivo or in vitro delivery of biologically active agents are also described. The transfection complexes of the present invention are highly potent, thereby allowing effective modulation of a biological activity at relatively low doses compared to analogous transfection compounds known in the art.
Owner:LIFE TECH CORP
Gel-based delivery of recombinant adeno-associated virus vectors
InactiveUS20060078542A1High transduction efficiencyIncrease exposure timeBiocidePowder deliveryMuscle tissueHeart disease
Disclosed are water-soluble gel-based compositions for the delivery of recombinant adeno-associated virus (rAAV) vectors that express nucleic acid segments encoding therapeutic constructs including peptides, polypeptides, ribozymes, and catalytic RNA molecules, to selected cells and tissues of vertebrate animals. Also disclosed are gel-based rAAV compositions are useful in the treatment of mammalian, and in particular, human diseases, including for example, cardiac disease or dysfunction, and musculoskeletal disorders and congenital myopathies, including, for example, muscular dystrophy, acid maltase deficiency (Pompe's disease), and the like. In illustrative embodiments, the invention provides rAAV vectors comprised within a biocompatible gel composition for enhanced viral delivery / transfection to mammalian tissues, and in particular to vertebrate muscle tissues such as a human heart or diaphragm tissue.
Owner:UNIV OF FLORIDA RES FOUNDATION INC
Isolation of novel AAV'S and uses thereof
ActiveUS9217155B2Avoid time-consume and costly processVectorsSpecial deliveryBinding siteAdeno associate virus
The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.
Owner:UNIV OF MASSACHUSETTS
AAV8 Vector with Enhanced Functional Activity and Methods of Use Thereof
ActiveUS20140037585A1Reduced integrityReduce rigidityBiocidePeptide/protein ingredientsFunctional activityGene
Owner:THE CHILDRENS HOSPITAL OF PHILADELPHIA
Selectively altering microbiota for immune modulation
ActiveUS20180140698A1Enhancing cell based therapyDampening down cell based therapyOrganic active ingredientsPeptide/protein ingredientsPopulationBiological body
The invention relates to methods of modulating immune cells in a patient by altering microbiota of the patient. The invention also relates to methods of modulating treatments or therapies in a subject organism by altering microbiota of the subject. The invention also relates to cell populations, systems, arrays, cells, RNA, kits and other means for effecting this. In an example, advantageously selective targeting of a particular species in a human gut microbiota using guided nucleic acid modification is carried out to effect the alteration.
Owner:SNIPR TECH
Compositions and methods for topical delivery of oligonucleotides
InactiveUS6841539B1Simpler and easy and effective deliverySimple and efficient and convenient deliveryOrganic active ingredientsTissue cultureCuticleLiposome
Owner:IONIS PHARMA INC
Compositions and methods for gene therapy
InactiveUS20060063732A1Improve hydrophobicityEffective occlusionPowder deliveryGranular deliveryTransfectionBlood vessel
Owner:BIOSPHERE MEDICAL INC
Adeno-associated virus variants and methods of use thereof
ActiveUS20160017295A1Improve the immunityEfficient deliveryAntibacterial agentsVirusesHeterologousGene product
The present disclosure provides infectious recombinant adeno-associated virus (rAAV) virions that comprise a variant capsid protein and a heterologous nucleic acid. The present disclosure further provides the variant adeno-associated virus (AAV) capsid proteins (and / or a nucleic acid encoding the variant AAV capsid proteins), which confer to an infectious rAAV virion an increased resistance to human AAV neutralizing antibodies. The present disclosure further provides host cells comprising an infectious rAAV virion and / or a nucleic acid encoding a subject variant AAV capsid protein. The present disclosure further provides methods of delivering a heterologous nucleic acid to a target cell where the target cell is contacted with a subject infectious rAAV virion. The present disclosure further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.
Owner:RGT UNIV OF CALIFORNIA
Pharmaceutical compositions comprising an oligonucleotide as an active agent
A pharmaceutical composition is disclosed, which composition comprises an oligonucleotide as an active agent, the oligonucleotide being adapted to target nucleic acids encoding CD40 thereby to modulate the expression of CD40 in mammalian cells, and a liposome as an excipient. Said liposome is an amphoteric liposome. Also disclosed is a method for the treatment or prophylaxis of a disease or condition associated with the expression of CD40 in a human or non-human animal patient by administering to said patient a therapeutically or prophylactically effective amount of such a composition.
Owner:NOVOSOM
Adeno-associated virus virions with variant capsid and methods of use thereof
The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
Owner:RGT UNIV OF CALIFORNIA
Targeted Gene Delivery to Non-Phagocytic Mammalian Cells Via Bacterially Derived Intact Minicells
ActiveUS20070237744A1High expressionFunction increaseOrganic active ingredientsBacteriaGene deliveryPhagocytic Cell
A method of targeting bacterially-derived, intact minicells to specific, non-phagocytic mammalian cells employs bispecific ligands to deliver nucleic acids efficiently to the mammalian cells. Bispecific ligands, comprising (i) a first arm that carries specificity for a bacterially-derived minicell surface structure and (ii) a second arm that carries specificity for a non-phagocytic mammalian cell surface receptor are useful for targeting minicells to specific, non-phagocytic mammalian cells and causing endocytosis of minicells by non-phagocytic cells.
Owner:ENGENEIC MOLECULAR DELIVERY PTY LTD
Delivering functional nucleic acids to mammalian cells via bacterially-derived, intact minicells
ActiveUS20070298056A1Eliminate side effectsPowder deliveryOrganic active ingredientsEukaryotic plasmidsDrug resistance
Intact bacterially derived minicells containing functional nucleic acids or plasmids encoding functional nucleic acids can reduce, in targeted mammalian cells, drug resistance, apoptosis resistance, and neoplasticity, respectively. Methodology that employs minicells to deliver functional nucleic acids, targeting the transcripts of proteins that contribute to drug resistance or apoptosis resistance, inter alia, can be combined with chemotherapy to increase the effectiveness of the chemotherapy.
Owner:ENGENEIC MOLECULAR DELIVERY PTY LTD
Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
InactiveUS20050186179A1Down-regulating angiogenesisSenses disorderVirusesCell specificPromoter activity
Isolated polynucleotide sequences exhibiting endothelial cell specific promoter activity, novel cis regulatory elements and methods of use thereof enabling treatment of diseases characterized by aberrant neovascularization or cell growth are disclosed.
Owner:VASCULAR BIOGENICS
Combination therapy for treating protein deficiencies
InactiveUS7446098B2High biological efficiencyImprove cost efficiencyBiocideMetabolism disorderReactive siteCombined Modality Therapy
This application provides methods of improving gene therapy by combining gene therapy with active site-specific chaperones (ASSCs). The ASSC increases the stability and efficiency of the protein encoded by the recombinant gene that is administered.
Owner:MT SINAI SCHOOL OF MEDICINE
Nucleic Acid Formulations for Gene Delivery and Methods of Use
InactiveUS20070213287A1Improve topical bioavailabilityHigh expressionOrganic active ingredientsPeptide/protein ingredientsGene deliveryD-glutamic acid
A nucleic acid formulation for use in gene delivery comprising a nucleic acid and an anionic polymer is disclosed. Examples of the anionic polymer includes anionic amino acid polymer or poly-amino acid (such as poly-L-glutamic acid, poly-D-glutamic acid, poly-L-aspartic acid, poly-D-aspartic acid), poly-acrylic acid, polynucleotides, poly galacturonic acid, and poly vinyl sulfate.
Owner:INOVIO PHARMA
Compositions of stabilized DNA for coating microprojctions
InactiveUS20050090009A1Reduce degradationAvoid rapid degradationMicroinjection basedNon-active genetic ingredientsCompound (substance)Biology
Owner:ALZA CORP
ARPE-19 as a platform cell line for encapsulated cell-based delivery
InactiveUS7115257B1Enhance cell viabilitySignificant adverse effectBiocideSenses disorderSurvivabilityIn vivo
ARPE-19 cells were evaluated as a platform cell line for encapsulated and unencapsulated cell-based delivery technology. ARPE-19 cells were found to be hardy (the cell line is viable under stringent conditions, such as in central nervous system or intra-ocular environment); can be genetically modified to secrete the protein of choice; have a long life span; are of human origin; have good in vivo device viability; deliver efficacious quantity of growth factor; trigger no or low level host immune reaction, and are non-tumorigenic.
Owner:NEUROTECH USA
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