21403 ⎘
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
Sub-classes:IONIZABLE CATIONIC LIPID FOR RNA DELIVERY
#2SYNERGISTIC ENHANCEMENT OF THE DELIVERY OF NUCLEIC ACIDS VIA BLENDED FORMULATIONS
#3Paracrine Adenoviral Delivery of Biomolecules
#4COMPOSITIONS AND METHODS FOR REPROGRAMMING DISEASED MUSCULOSKELETAL CELLS
#5COMPOSITIONS AND METHODS FOR VIROTHERAPY
#6ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS WITH IMPROVED RETINAL TRANSDUCTION AND USES THEREOF
#7METHODS AND COMPOSITIONS FOR CAS IMMUNE TOLERANCE INDUCTION TO SUPPORT CRISPR-CAS IN VIVO GENE EDITING
#8METHODS AND SYSTEMS FOR TRANSFORMER-BASED BIOLOGICAL SEQUENCE MODELS
#9RNA CONTAINING COMPOSITION FOR TREATMENT OF TUMOR DISEASES
#10COMPOSITION AND METHODS FOR TREATMENT OF ORNITHINE TRANSCARBAMYLASE DEFICIENCY
#11SCAFFOLDS FOR MODIFYING IMMUNE CELLS AND THE USES THEREOF
#12RECOMBINANT AAV CAPSID PROTEINS
#13TRANSGENIC EXPRESSION CASSETTE FOR TREATING MUSCULAR DYSTROPHY
#14METHODS AND COMPOSITIONS FOR SYNTHETIC BIOMARKERS
#15METHODS AND COMPOSITIONS FOR SYNTHETIC BIOMARKERS
#16CELL DELIVERY COMPOSITIONS AND METHODS OF USE THEREOF
#17GENETICALLY MODIFIED BACTERIA AND METHODS FOR GENETIC MODIFICATION OF BACTERIA
#18NUCLEIC ACID CONSTRUCTS, VIRAL VECTORS AND VIRAL PARTICLES
#19MESSENGER RNA THERAPY FOR TREATMENT OF ARTICULAR DISEASE
#20COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
#21IN VIVO DELIVERY OF OLIGONUCLEOTIDES
#22COMPOUNDS, COMPOSITIONS, AND METHODS OF USING THEREOF
#23MINERAL-COATED SUBSTRATES FOR STABILIZATION OF RNA-BASED THERAPEUTIC COMPOSITIONS
#24CAPSID VARIANTS AND METHODS OF USING THE SAME
#25DIFFERENTIAL CXCR4 EXPRESSION ON HEMATOPOIETIC PROGENITOR CELLS VERSUS STEM CELLS DIRECTS HOMING AND LONG-TERM ENGRAFTMENT
#26COMPOSITIONS AND METHODS FOR DELIVERY OF NUCLEIC ACIDS TO CELLS
#27COMPOSITIONS AND METHODS FOR DELIVERY OF NUCLEIC ACIDS TO CELLS
#28PROMOTER-ENHANCER SEQUENCES OF THE HUMAN TROPONIN T GENE FOR SELECTIVE EXPRESSION IN CARDIAC MYOCYTES
#29Compositions and Methods for Treating Skeletal Muscle Disease
#30COMPOSITIONS AND METHODS FOR OPTIMIZING TROPISM OF DELIVERY SYSTEMS FOR RNA
#31VARIANT-SPECIFIC EXOGENOUS DNA TEMPLATE-FREE CORRECTION OF PATHOGENIC VARIANTS
#32FIBRIL PEPTIDES
#33PRODUCTION OF CIRCULAR POLYRIBONUCLEOTIDES IN A EUKARYOTIC SYSTEM
#34COMPOUNDS, COMPOSITIONS, AND METHODS OF USING THEREOF
#35High-transducing HSV vectors
#36ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS WITH IMPROVED RETINAL TRANSDUCTION AND USES THEREOF
#37USE OF CYP4V2 AND RDCVF IN THE MANUFACTURE OF MEDICAMENT
#38POLYNUCLEOTIDES ENCODING INTERLEUKIN-12 (IL12) AND USES THEREOF
#39RATIONAL POLYPLOID ADENO-ASSOCIATED VIRUS VECTORS FOR THE TREATMENT OF DISEASE
#40LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#41EXTRACELLULAR VESICLES WITH IMMUNE MODULATORS
#42COMPOSITION FOR PREVENTING OR TREATING OBESITY-RELATED DISEASE CONTAINING AMPHIREGULIN-SPECIFIC DOUBLE-STRANDED OLIGONUCLEOTIDE STRUCTURE
#43RNA CONTAINING COMPOSITION FOR TREATMENT OF TUMOR DISEASES
#44PLAKOPHILLIN-2 GENE THERAPY METHODS AND COMPOSITIONS
#45METHODS FOR MODULATING RNA SPLICING
#46Bacterial Vehicle for Engineering of Non-Phagocytic Immune Cells
#47AAV VECTORS ENCODING PARKIN AND USES THEREOF
#48COMPOSITIONS AND METHODS FOR DELIVERY OF NUCLEIC ACIDS TO CELLS
#49RNA FORMULATION FOR IMMUNOTHERAPY
#50FUSOGENIC LIPID NANOPARTICLES AND METHODS FOR THE MANUFACTURE AND USE THEREOF FOR THE TARGET CELL-SPECIFIC PRODUCTION OF A THERAPEUTIC PROTEIN AND FOR THE TREATMENT OF A DISEASE, CONDITION, OR DISORDER ASSOCIATED WITH A TARGET CELL
#51AAV Gene Therapy for Spastic Paraplegia
#52Treatments for retinal disease
#53MODIFIED MINI-NUCLEOSOME CORE PROTEINS AND USE IN NUCLEIC ACID DELIVERY
#54METHODS OF EDITING SINGLE NUCLEOTIDE POLYMORPHISM USING PROGRAMMABLE BASE EDITOR SYSTEMS
#55METHODS AND SYSTEMS FOR NONINVASIVE CONTROL OF BRAIN CELLS AND RELATED VECTORS AND COMPOSITIONS
#56TREATING AUTOIMMUNE DISEASES WITH GENETICALLY MODIFIED CELLS
#57USE OF CYP4V2 AND RDCVF IN THE MANUFACTURE OF MEDICAMENT
#58NON-VIRAL DNA VECTORS AND USES THEREOF FOR EXPRESSING GAUCHER THERAPEUTICS
#59NANOPARTICLES FOR EXPRESSION OF GENES OF INTEREST AND/OR REGULATION OF SIGNALING PATHWAYS
#60COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY
#61Compositions and methods for delivery of nucleic acids to cells
#62Compositions and methods for delivery of nucleic acids to cells
#63DAMAGE-TARGETED TREATMENTS OF DISEASE
#64NOVEL AAV3B VARIANTS THAT TARGET HUMAN HEPATOCYTES IN THE LIVER OF HUMANIZED MICE
#65Recombinant CDKL5 Proteins, Gene Therapy and Production Methods
#66Compositions and methods for delivery of nucleic acids to cells
#67METHOD OF IN VIVO ADMINISTRATION OF THE CODING SEQUENCE OF THE SIRT6 GENE VIA ADENO-ASSOCIATED VIRUS
#68AAV CAPSID-PROMOTER INTERACTIONS AND CELL SELECTIVE GENE EXPRESSION
#69VACCINIA VIRUSES AND METHODS FOR USING VACCINIA VIRUSES
#70ORNITHINE TRANSCARBAMYLASE (OTC) CONSTRUCTS AND METHODS OF USING THE SAME
#71Treatments for retinal disease
#72FACTOR VIII POLYPEPTIDE
#73ON-BIPOLAR CELL-SPECIFIC PROMOTERS FOR OCULAR GENE DELIVERY
#74COMPOSITIONS AND METHODS FOR TREATING GLYCOGEN STORAGE DISORDERS
#75TRANSCRIPTION REGULATORY ELEMENTS
#76GENE THERAPY TARGETING COCHLEAR CELLS
#77PHASE-SEGREGATED VESICLES FOR SPATIALLY CONTROLLED PROTEIN-CONJUGATION AND CELL THERAPY
#78COMPOSITIONS AND METHODS FOR TREATING SUCCINIC SEMIALDEHYDE DEHYDROGENASE DEFICIENCY (SSADHD)
#79PROTEIN PRODUCING NANOLIPOSOMES AND USES THEREOF
#80NON-VIRAL IMMUNO-TARGETING
#81Polynucleotides encoding interleukin-12 (IL12) and uses thereof
#82Adeno-Associated Virus Virions with Variant Capsid and Methods of Use Thereof
#83ANTICANCER COMBINATIONS
#84PREPARING TISSUES FOR DELIVERY OF THERAPEUTIC AND DIAGNOSTIC AGENTS AND DELIVERING THE AGENTS
#85ANTITUMOR IMMUNITY ENHANCING COMPOSITION CONTAINING ADENOVIRUS SIMULTANEOUSLY EXPRESSING IL-12 AND SHVEGF
#86IN VIVO DELIVERY OF OLIGONUCLEOTIDES
#87Recombinant adenoviruses carrying transgenes
#88COMPOSITIONS AND METHODS FOR REPROGRAMMING DISEASED MUSCULOSKELETAL CELLS
#89COMPOSITION FOR DELIVERY OF GENETIC MATERIAL
#90Composition and methods for treatment of ornithine transcarbamylase deficiency
#91METHODS OF PREPARING LIPID NANOPARTICLES
#92Optimized mRNA encoding CAS9 for use in LNPs
#93RECOMBINANT ADENO-ASSOCIATED VECTORS
#94Biodegradable lipids for delivery of nucleic acids
#95Exosomes Comprising RNA Therapeutics
#96THERAPEUTIC NANOPARTICLES AND METHODS OF USE THEREOF
#97Method and composition for treating neuropathic pain
#98Heterologous targeting peptide grafted AAVs
#99DIAGNOSTIC METHODS FOR ANTI-ANGIOGENIC AGENT THERAPY
#100Polynucleotides encoding interleukin-12 (IL12) and uses thereof
#101Lipid formulations for nucleic acid delivery
#102COMBINATORIAL GENE THERAPY
#103IONIZABLE CATIONIC LIPID FOR RNA DELIVERY
#104NOVEL LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#105Messenger RNA therapy for treatment of articular disease
#106ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
#107Adeno-associated virus variant capsids and methods of use thereof
#108Rational polyploid adeno-associated virus vectors for the treatment of disease
#109Aminoalcohol lipidoids and uses thereof
#110Methods for modulating RNA splicing
#111Method of using adeno-associated virus with variant capsid
#112Adeno-associated virus variant capsids and methods of use thereof
#113COMPOSITIONS AND METHODS FOR TREATING SUCCINIC SEMIALDEHYDE DEHYDROGENASE DEFICIENCY (SSADHD)
#114Synergistic Enhancement of the Delivery of Nucleic Acids via Blended Formulations
#115DELIVERING CRISPR THERAPEUTICS WITH LIPID NANOPARTICLES
#116Polynucleotides encoding interleukin-12 (IL12) and uses thereof
#117MODIFIED RAAV CAPSID PROTEIN FOR GENE THERAPY
#118Composition for delivery of genetic material
#119Treatment of monogenetic disorders
#120Non-viral gene delivery agent comprising lipopeptide (LP) compounds
#121Biodegradable lipids for delivery of nucleic acids
#122High-transducing HSV vectors
#123Compositions and methods for treatment of peroxisome proliferator-activated receptor gamma (PPARG) activated cancer
#124Collagen-mimetic peptide mediated delivery of nucleic acid carriers for efficient delivery from collagen
#125Genetically modified bacteria and methods for genetic modification of bacteria
#126NOVEL LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#127Rational polyploid adeno-associated virus vectors for the treatment of disease
#128In vivo delivery of oligonucleotides
#129RNA formulation for immunotherapy
#130VIRUS AAV/IGF2, GENETIC TREATMENT METHOD AND USE THEREOF IN PROTEIN MISFOLDING-RELATED DISEASES, SUCH AS HUNTINGTON'S DISEASE
#131Heterologous targeting peptide grafted AAVS
#132Ionizable cationic lipid for RNA delivery
#133Tunable endogenous protein degradation with heterobifunctional compounds
#134AAV-BASED GENE THERAPY FOR GLAUCOMA
#135Semi-synthetic tissue constructs for tissue regeneration
#136NOVEL PROTEIN TRANSDUCTION DOMAIN AND USE THEREOF
#137Expression constructs, fusogenic lipid-based nanoparticles and methods of use thereof
#138Adipocyte-targeting non-viral gene delivery complex comprising dual plasmid vector
#139Adeno-associated virus variant capsids and methods of use thereof
#140NUCLEIC ACIDS, PEPTIDES AND METHODS
#141Adeno-associated virus virions with variant capsid
#142Synergistic enhancement of the delivery of nucleic acids via blended formulations
#143Drug delivery system based on JCV-VLP
#144GENE EDITING AND TARGETED TRANSCRIPTIONAL MODULATION FOR ENGINEERING ERYTHROID CELLS
#145Composition and methods for treatment of ornithine transcarbamylase deficiency
#146Aminoalcohol lipidoids and uses thereof
#147Methods and systems for noninvasive control of brain cells and related vectors and compositions
#148Compositions and methods for treating viral infections in shrimps
#149Compositions and methods for treating Huntington's disease
#150Composition for delivery of genetic material
#151Amphipathic peptide
#152Liposomes comprising a calcium phosphate-containing precipitate
#153ANTITUMOR IMMUNITY ENHANCING COMPOSITION CONTAINING ADENOVIRUS SIMULTANEOUSLY EXPRESSING IL-12 AND SHVEGF
#154Targeted gene delivery to non-phagocytic mammalian cells via bacterially derived intact minicells
#155Recombinant adenoviruses carrying transgenes
#156Messenger RNA therapy for treatment of articular disease
#157LIPIDS FOR DELIVERY OF THERAPEUTICS
#158Vectors Conditionally Expressing Protein
#159NOVEL DRUG DELIVERY SYSTEM BASED ON JCV-VLP
#160Biodegradable lipids for delivery of nucleic acids
#161Biodegradable lipids for delivery of nucleic acids
#162Biodegradable lipids for delivery of nucleic acids
#163Biodegradable lipids for delivery of nucleic acids
#164Heterologous targeting peptide grafted AAVS
#165METHODS OF TREATING PARKINSON'S DISEASE USING VIRAL VECTORS
#166Self-assembled ribonucleoprotein nanoparticles
#167Ionizable cationic lipid for RNA delivery
#168Methods for modulating RNA splicing
#169Delivering CRISPR therapeutics with lipid nanoparticles
#170NOVEL LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#171NOVEL LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#172Amphipathic peptide
#173Therapeutic miRNAs for treating heart and skeletal muscle diseases
#174SYSTEM AND METHOD FOR DELIVERING GENETIC MATERIAL OR PROTEIN TO CELLS
#175Aminoalcohol lipidoids and uses thereof
#176Composition for delivery of genetic material
#177Adeno-associated virus virions with variant capsid and methods of use thereof
#178Adeno-associated virus virions with variant capsid and methods of use thereof
#179Therapeutic miRNAs for treating heart and skeletal muscle diseases
#180Vectors Conditionally Expressing Protein
#181Interfering RNA delivery system and uses thereof
#182METHODS OF TREATING PARKINSON'S DISEASE USING VIRAL VECTORS
#183GENE THERAPY FOR DIABETIC ISCHEMIC DISEASE
#184Compositions and methods for treating endocrine, gastrointestinal or autoimmune disorders
#185Tyrosine-based lipids for delivery of therapeutics
#186Protein engineered systems for delivery of molecules
#187Anticancer combinations
#188Adeno-associated virus virions with variant capsid and methods of use thereof
#189Messenger RNA therapy for treatment of articular disease
#190Synergistic enhancement of the delivery of nucleic acids via blended formulations
#191Adipocyte-targeting non-viral gene delivery system
#192Composition for delivery of genetic material
#193Vectors comprising stuffer/filler polynucleotide sequences and methods of use
#194Genetically modified bacteria and methods for genetic modification of bacteria
#195Targeted gene delivery to non-phagocytic mammalian cells via bacterially derived intact minicells
#196Amphipathic peptide
#197Materials and Methods for Regulating Whole Body Glucose Homeostasis
#198Adeno-associated virus virions with variant capsid and methods of use thereof
#199Aminoalcohol lipidoids and uses thereof
#200Lipid formulations for nucleic acid delivery
#201DELIVERY OF NUCLEIC ACID-LIKE COMPOUNDS
#202RNA formulation for immunotherapy
#203Therapeutic delivery and expression system, methods and uses thereof
#204A NOVEL DRUG DELIVERY SYSTEM BASED ON JCV-VLP
#205Arginine-based lipids for delivery of therapeutics
#206Drug delivery product and methods
#207Vectors conditionally expressing protein
#208PHOSPHOLIPID-DETERGENT CONJUGATES AND USES THEREOF
#209Down regulation of the gene expression by means of nucleic acid-loaded virus-like particles
#210Interfering RNA delivery system and uses thereof
#211Amino acid lipids and uses thereof
#212Delivery of nucleic acid-like compounds
#213Multiplexed supramolecular assemblies for non-viral delivery of genetic material
#214DNA origami devices
#215Hybrid superparamagnetic iron oxide nanoparticles and polyethylenimine as a magnetocomplex for gene transfection
#216INTERFERING RNA DELIVERY SYSTEM AND USES THEREOF
#217Composition for nucleic acid transfection
#218Composition For Delivery Of Genetic Material
#219SYSTEM FOR IMPROVED DELIVERY OF GENE MODULATING COMPOUNDS
#220Localized Delivery of Gold Nanoparticles for Therapeutic and Diagnostic Applications
#221NOVEL CATIONIC LIPIDS WITH SHORT LIPID CHAINS FOR OLIGONUCLEOTIDE DELIVERY
#222Immunogenic minicells and methods of use
#223ISOLATED STROMAL CELLS AND METHODS OF USING THE SAME
#224Liposomes comprising a calcium phosphate-containing precipitate
#225Lipid nanoparticles for gene therapy
#226Lipid formulations for nucleic acid delivery
#227Polyion complex comprising PHD2 expression inhibiting substance
#228Hybrid superparamagnetic iron oxide nanoparticles and polyethylenimine as a magnetoplex for gene transfection
#229AMPHOTERIC LIPOSOMAL COMPOSITIONS FOR CELLULAR DELIVERY OF SMALL RNA MOLECULES FOR USE IN RNA INTERFERENCE
#230Down regulation of the gene expression by means of nucleic acid-loaded virus-like particles
#231POLYACRIDINE NUCLEIC ACID DELIVERY PEPTIDE COMPLEXES
#232Oral Administration Of Therapeutic Agent Coupled To Transporting Agent
#233Aminoalcohol lipidoids and uses thereof
#234CATIONIC LIPIDS AND METHODS OF USE
#235INCREASING EFFICIENCY OF NUCLEIC ACID DELIVERY IN VIVO USING TARGETING CONJUGATES
#236Lipophilic nucleic acid delivery vehicle and methods of use thereof
#237COMPOSITIONS AND METHODS FOR TOPICAL DELIVERY OF OLIGONUCLEOTIDES
#238Antigenic compositions and use of same in the targeted delivery of nucleic acids
#239Amino acid lipids and uses thereof
#240Bottom hole assembly with ported completion and methods of fracturing therewith
#241NANOPARTICLE COMPOSITIONS FOR NUCLEIC ACIDS DELIVERY SYSTEM
#242POLYMERIC SYSTEMS CONTAINING INTRACELLULAR RELEASABLE DISULFIDE LINKER FOR THE DELIVERY OF OLIGONUCLEOTIDES
#243HISTONE OCTAMERS FOR INCREASED NUCLEIC ACID TRANSFER
#244GENE THERAPY FOR DIABETIC ISCHEMIC DISEASE
#245POLYPEPTIDE-NUCLEIC ACID CONJUGATES AND USES THEREOF
#246Aminoalcohol lipidoids and uses thereof
#247Lipid
#248Methods and models for rapid, widespread delivery of genetic material to the CNS using non-viral, cationic lipid-mediated vectors
#249Drug delivery product and methods
#250Materials and complexes for the delivery of biologically-active materials to cells
#251Protein-coding RNA to correct mitochondrial dysfunction
#252Lipid formulations for nucleic acid delivery
#253Methods of treating Parkinson's disease using viral vectors
#254TRIAZINE DENDRIMERS AND METHODS OF MAKING AND USING THE SAME FOR NUCLEIC ACID TRANSPORT
#255Aqueous formulation for selective targeting and delivering gene to cancer cells
#256COMPOSITION FOR NUCLEIC ACID TRANSFECTION
#257Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications
#258COMPOSITIONS COMPRISING A SIRNA AND LIPIDIC 4,5-DISUBSTITUTED 2-DEOXYSTREPTAMINE RING AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF
#259ISOLATED STROMAL CELLS AND METHODS OF USING THE SAME
#260Compositions and methods for topical delivery of oligonucleotides
#261Transfection of Collagen-Producing Cells
#262DELIVERY OF NUCLEIC ACID-LIKE COMPOUNDS
#263Peptide lipid-containing carrier and method for introducing compound into cells using same
#264METHOD FOR PRODUCING A NUCLEIC-ACID-CONTAINING COMPLEX PREPARATION
#265COMPOSITIONS AND METHODS FOR PERFORMING REVERSE GENE THERAPY
#266Membrane active heteropolymers
#267COMPOSITIONS AND METHODS FOR NUCLEIC ACID DELIVERY SYSTEMS
#268ISOLATED STROMAL CELLS AND METHODS OF USING THE SAME
#269Delivery of siRNA by neutral lipid compositions
#270Immunogenic minicells and methods of use
#271Amino acid lipids and uses thereof
#272Lipids and lipid assemblies comprising transfection enhancer elements
#273Gene therapy for diabetic ischemic disease
#274Methods and Compositions Facilitating Entry of Compounds Into Cells
#275Compositions And Methods For Delivery Of Agents Into Cells
#276COMPOSITION FOR IN VIVO DELIVERY
#277NANOPARTICLE DELIVERY VEHICLE
#278Cationic lipids for the transfection of nucleic acids
#279In Vivo Transformation of Pancreatic Acinar Cells into Insulin-Producing Cells
#280Amphoteric liposomes and their use
#281Materials and Methods Relating to the Treatment of Glioblastomas
#282Compositions and methods for non-parenteral delivery of oligonucleotides
#283Galactose derivative, drug carrier and medicinal composition
#284Targeted gene delivery to non-phagocytic mammalian cells via bacterially derived intact minicells
#285Composition and method for production of transformed cells
#286Shielded micelles for polynucleotide delivery
#287Polyampholytes for delivering polyions to a cell
#288Glucocorticoid modulation of nucleic acid-mediated immune stimulation
#289Systems and methods for manufacturing liposomes
#290Methods of using redox-active surfactants to control polymer interactions
#291Functional synthetic molecules and macromolecules for gene delivery
#292Peptide-enhanced transfections
#293Oral administration of therapeutic agent coupled to transporting agent induces tolerance
#294Process of making a compound by forming a polymer from a template drug
#295Pharmaceutical compositions for local administration
#296Injectable non-aqueous suspension
#297Hollow nanoparticle having modified cysteine residue and drug with the use thereof
#298Gene expression inducing fusion protein and method for controlling gene expression Induction
#299Method for inhibition of pathogenic microorganisms
#300DNA/RNA transduction technology and its clinical and basic applications