ClassID:

21411

A61K48/0091 - CPC Classification

Classification description:

Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy Purification or manufacturing processes for gene therapy compositions

Recent Application in this class:
#1
20260152720
2026-06-04

GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING GENETICALLY CORRECTED AUTOLOGOUS KERATINOCYTES

#2
20260124320
2026-05-07

FORMULATIONS FOR AAV GENE THERAPY

#3
20260098076
2026-04-09

MODIFIED LIGAND-GATED ION CHANNELS AND METHODS OF USE

#4
20260083863
2026-03-26

CAPSID POLYPEPTIDES AND METHODS OF USE THEREOF

#5
20260069713
2026-03-12

Lipid-Coated Nanoparticles

#6
20260053954
2026-02-26

Benzaldehyde acetal acid-degradable amphiphilic lipid and self-assembling peptides

#7
20260041788
2026-02-12

GENE THERAPY FOR TREATING PROPIONIC ACIDEMIA

#8
20260034247
2026-02-05

COMPOSITIONS AND METHODS FOR TREATING CEP290-ASSOCIATED DISEASE

#9
20260021207
2026-01-22

SUPER MINIMAL INVERTED TERMINAL REPEAT (ITR) SEQUENCES AND USES THEREOF

#10
20260014278
2026-01-15

HYBRID PEPTIDE DENDRIMER SYSTEMS AND EXTRAHEPATIC DELIVERY

#11
20260014274
2026-01-15

REVERSIBLE, COVALENT POLYNUCLEOTIDE CONDENSATION APPROACH FOR ENHANCED GENE DELIVERY

#12
20250332286
2025-10-30

CHEMICALLY-MODIFIED ADENO-ASSOCIATED VIRUSES

#13
20250325705
2025-10-23

GENE THERAPY

#14
20250312483
2025-10-09

SILICA PARTICLES FOR ENCAPSULATING NUCLEIC ACIDS

#15
20250295808
2025-09-25

A SYSTEM FOR AN OCULAR GENE THERAPY AND A PROCESS FOR PREPARING THEREOF

#16
20250288698
2025-09-18

COMPOSITIONS AND METHODS FOR PROMOTING HOMOLOGY DIRECTED REPAIR

#17
20250262330
2025-08-21

GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIES WITH CHIMERIC ANTIGEN RECEPTORS

#18
20250257319
2025-08-14

GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING GENETICALLY CORRECTED AUTOLOGOUS KERATINOCYTES

#19
20250243484
2025-07-31

COMPOSITIONS AND METHODS FOR TRANSIENT GENE THERAPY WITH ENHANCED STABILITY

#20
20250223611
2025-07-10

Targeted CRISPR Delivery Platforms

#21
20250222132
2025-07-10

NUCLEIC ACID COMPOSITIONS COMPRISING A MULTIVALENT ANION, SUCH AS AN INORGANIC POLYPHOSPHATE, AND METHODS FOR PREPARING, STORING AND USING THE SAME

#22
20250213731
2025-07-03

TREATING METASTATIC CANCER AND MODEL SYSTEMS FOR METASTATIC DISEASE

#23
20250207130
2025-06-26

Compositions and Methods for TTR Gene Editing and Treating ATTR Amyloidosis

#24
20250197889
2025-06-19

Targeted CRISPR Delivery Platforms

#25
20250195436
2025-06-19

DRY POWDER COMPOSITION COMPRISING LONG-CHAIN RNA

#26
20250179525
2025-06-05

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#27
20250171801
2025-05-29

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#28
20250152735
2025-05-15

Lipid Nanoparticle Compositions and Methods for mRNA Delivery

#29
20250127932
2025-04-24

METHOD FOR DELIVERING RNA TO NEURONS TO TREAT HERPES INFECTIONS

#30
20250127931
2025-04-24

NOVEL IMMUNOSTIMULATING VECTOR SYSTEM

#31
20250114482
2025-04-10

THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING

#32
20250114307
2025-04-10

DRY POWDER COMPOSITION COMPRISING LONG-CHAIN RNA

#33
20250108126
2025-04-03

Lipid Nanoparticle Compositions and Methods for mRNA Delivery

#34
20250101468
2025-03-27

DELIVERY AND USE OF THE CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS FOR HEPATIC TARGETING AND THERAPY

#35
20250101461
2025-03-27

STABILIZED FORMULATIONS OF CHIMPANZEE ADENOVIRUS

#36
20250099624
2025-03-27

METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES

#37
20250082785
2025-03-13

GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIES WITH CHIMERIC ANTIGEN RECEPTORS

#38
20250075178
2025-03-06

GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING GENETICALLY CORRECTED AUTOLOGOUS KERATINOCYTES

#39
20250057984
2025-02-20

CELL-TYPE SPECIFIC MEMBRANE FUSION PROTEINS

#40
20250041458
2025-02-06

METHODS FOR FORMING POLYPLEXES

#41
20240366798
2024-11-07

PROCESSES OF PREPARING MRNA-LOADED LIPID NANOPARTICLES

#42
20240350676
2024-10-24

COMPOSITIONS AND SYSTEMS COMPRISING TRANSFECTION-COMPETENT VESICLES FREE OF ORGANIC-SOLVENTS AND DETERGENTS AND METHODS RELATED THERETO

#43
20240342315
2024-10-17

COMPOSITIONS AND SYSTEMS COMPRISING TRANSFECTION-COMPETENT VESICLES FREE OF ORGANIC-SOLVENTS AND DETERGENTS AND METHODS RELATED THERETO

#44
20240315980
2024-09-26

EXTRACELLULAR VESICLES FOR INHALATION

#45
20240294578
2024-09-05

CAPSID VARIANTS AND METHODS OF USING THE SAME

#46
20240247237
2024-07-25

SCALABLE PRODUCTION METHOD FOR AAV

#47
20240229068
2024-07-11

CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES

#48
20240228941
2024-07-11

ELECTROPORATION, DEVELOPMENTALLY-ACTIVATED CELLS, PLURIPOTENT-LIKE CELLS, CELL REPROGRAMMING AND REGENERATIVE MEDICINE

#49
20240200044
2024-06-20

EVOLUTION OF TALENS

#50
20240191243
2024-06-13

HSV AMPLICON PACKAGING SYSTEM USING ENGINEERED CELLS

#51
20240181079
2024-06-06

PRODUCTION OF CIRCULAR POLYRIBONUCLEOTIDES IN A EUKARYOTIC SYSTEM

#52
20240156989
2024-05-16

Methods and Compositions for Modifying a Mutant Dystrophin Gene in a Cell's Genome

#53
20240156733
2024-05-16

METHODS OF PREPARATION OF NOVEL PAN TLR ANTAGONISTIC LIPOSOMAL-LNP FORMULATIONS AND USES THEREOF

#54
20240139344
2024-05-02

COMPOSITIONS AND METHODS FOR TREATING CLRN1-ASSOCIATED HEARING LOSS AND/OR VISION LOSS

#55
20240132912
2024-04-25

CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES

#56
20240117378
2024-04-11

Method of Increasing the Function of an AAV Vector

#57
20240117339
2024-04-11

RAPID DESIGN, BUILD, TEST, AND LEARN TECHNOLOGIES FOR IDENTIFYING AND USING NON-VIRAL CARRIERS

#58
20240115513
2024-04-11

METHODS FOR PREPARATION OF PLASMID DNA/LIPID PARTICLES WITH DEFINED SIZE FOR IN VITRO AND IN VIVO TRANSFECTION

#59
20240067926
2024-02-29

Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

#60
20240041786
2024-02-08

AMINO ALCOHOL IONIZABLE LIPIDS

#61
20240024239
2024-01-25

AMINO ALCOHOL IONIZABLE LIPIDS

#62
20230416780
2023-12-28

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#63
20230414788
2023-12-28

FORMULATIONS FOR SUPRACHOROIDAL ADMINISTRATION SUCH AS GEL FORMULATIONS

#64
20230414786
2023-12-28

PERFUSION-GUIDED GENE THERAPY FOR IMPROVING CANCER TREATMENT

#65
20230340532
2023-10-26

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#66
20230340397
2023-10-26

ELECTROPORATION, DEVELOPMENTALLY-ACTIVATED CELLS, PLURIPOTENT-LIKE CELLS, CELL REPROGRAMMING AND REGENERATIVE MEDICINE

#67
20230330262
2023-10-19

Lipid nanoparticle compositions and methods for mRNA delivery

#68
20230321282
2023-10-12

ADENO-ASSOCIATED VIRUS VIRIONS WITH VARIANT CAPSID AND METHODS OF USE THEREOF

#69
20230293723
2023-09-21

LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY

#70
20230287452
2023-09-14

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#71
20230285593
2023-09-14

Lipid nanoparticle compositions and methods for MRNA delivery

#72
20230285592
2023-09-14

Lipid nanoparticle compositions and methods for MRNA delivery

#73
20230272424
2023-08-31

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#74
20230257747
2023-08-17

Compositions and methods for TTR gene editing and treating ATTR amyloidosis

#75
20230201377
2023-06-29

COMPOSITION AND METHODS OF GENOME EDITING OF B-CELLS

#76
20230201370
2023-06-29

EXOSOMES COMPRISING THERAPEUTIC POLYPEPTIDES

#77
20230190963
2023-06-22

Treating metastatic cancer and model systems for metastatic disease

#78
20230158114
2023-05-25

METHODS FOR THE TREATMENT OF INFLAMMATORY JOINT DISEASE

#79
20230151341
2023-05-18

METHOD FOR SPECIFICALLY EDITING GENOMIC DNA AND APPLICATION THEREOF

#80
20230149566
2023-05-18

COMPOSITIONS AND METHODS FOR TREATING MACULAR DYSTROPHY

#81
20230132391
2023-04-27

Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12

#82
20230118592
2023-04-20

Compositions and methods for TTR gene editing and treating ATTR amyloidosis

#83
20230116968
2023-04-20

SCALABLE PRODUCTION METHOD FOR AAV

#84
20230073819
2023-03-09

Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto

#85
20230050813
2023-02-16

NUCLEIC ACID LOADED RED BLOOD CELL EXTRACELLULAR VESICLES

#86
20230048730
2023-02-16

PLATELETS TRANSFECTED BY EXOGENOUS GENETIC MATERIAL AND PLATELET MICROPARTICLES OBTAINED BY SAID TRANSFECTED PLATELETS, METHOD FOR THE PREPARATION AND USES THEREOF

#87
20230037283
2023-02-02

Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12

#88
20230009257
2023-01-12

Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12

#89
20220411823
2022-12-29

PLATELETS TRANSFECTED BY EXOGENOUS GENETIC MATERIAL AND PLATELET MICROPARTICLES OBTAINED BY SAID TRANSFECTED PLATELETS, METHOD FOR THE PREPARATION AND USES THEREOF

#90
20220401575
2022-12-22

COMPOSITIONALLY DEFINED PLASMID DNA/POLYCATION NANOPARTICLES AND METHODS FOR MAKING THE SAME

#91
20220389447
2022-12-08

Targeted CRISPR Delivery Platforms

#92
20220387320
2022-12-08

Dry powder composition comprising long-chain RNA

#93
20220380808
2022-12-01

Method for delivering RNA to neurons to treat herpes infections

#94
20220378939
2022-12-01

Lipid nanoparticle compositions and methods for mRNA delivery

#95
20220362409
2022-11-17

ADENO-ASSOCIATED VIRUS VIRIONS WITH VARIANT CAPSID AND METHODS OF USE THEREOF

#96
20220362406
2022-11-17

LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY

#97
20220347116
2022-11-03

NANOPARTICLE COMPLEX FOR TREATING DISEASES AND METHOD FOR MANUFACTURING THE SAME

#98
20220333097
2022-10-20

Rapid design, build, test, and learn technologies for identifying and using non-viral carriers

#99
20220331451
2022-10-20

Adeno-associated virus virions with variant capsid and methods of use thereof

#100
20220331450
2022-10-20

Adeno-associated virus virions with variant capsid and methods of use thereof

#101
20220313785
2022-10-06

METHODS FOR THE TREATMENT OF INFLAMMATORY JOINT DISEASE

#102
20220288240
2022-09-15

Use of Thermostable RNA Polymerases to Produce RNAs Having Reduced Immunogenicity

#103
20220273818
2022-09-01

COMPOSITIONS AND METHODS FOR TREATING CEP290-ASSOCIATED DISEASE

#104
20220265852
2022-08-25

Allele-specific inactivation of mutant HTT via gene editing at coding region single nucleotide polymorphisms

#105
20220257796
2022-08-18

RECOMBINANT AD35 VECTORS AND RELATED GENE THERAPY IMPROVEMENTS

#106
20220193267
2022-06-23

ENGINEERED NUCLEASES USEFUL FOR TREATMENT OF HEMOPHILIA A

#107
20220193252
2022-06-23

RAAV WITH CHEMICALLY MODIFIED CAPSID

#108
20220177921
2022-06-09

GENE THERAPY USING GENETICALLY MODIFIED VIRAL VECTORS

#109
20220168225
2022-06-02

Dry powder composition comprising long-chain RNA

#110
20220154208
2022-05-19

CONSTRUCTS AND METHODS FOR DELIVERING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES

#111
20220118112
2022-04-21

Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto

#112
20220118111
2022-04-21

Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12

#113
20220118110
2022-04-21

Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12

#114
20220111073
2022-04-14

Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes

#115
20220111072
2022-04-14

Lipid nanoparticle compositions and methods for mRNA delivery

#116
20220111071
2022-04-14

LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY

#117
20220111070
2022-04-14

Lipid nanoparticle compositions and methods for MRNA delivery

#118
20220106550
2022-04-07

CELL PROCESSING UNIT, CELL PROCESSING SYSTEM AND METHODS OF USE THEREOF

#119
20220105201
2022-04-07

Lipid nanoparticle compositions and methods for mRNA delivery

#120
20220096660
2022-03-31

METHODS FOR FORMING POLYPLEXES

#121
20220096541
2022-03-31

METHODS TO TREAT MITOCHONDRIAL-ASSOCIATED DYSFUNCTIONS OR DISEASES

#122
20220072160
2022-03-10

Compositions and methods for promoting homology directed repair

#123
20220064580
2022-03-03

CELL PROCESSING DEVICE CELL PROCESSING SYSTEM AND METHODS OF USE THEREOF

#124
20220047521
2022-02-17

Drug Delivery Particle and Method for Producing the Same

#125
20220040330
2022-02-10

Lipid nanoparticle compositions and methods for mRNA delivery

#126
20220031865
2022-02-03

COMBINATION THERAPY FOR TREATING MUSCULAR DYSTROPHY

#127
20210403887
2021-12-30

Evolution of TALENs

#128
20210386665
2021-12-16

INHALATION POWDER MEDICINE, EVALUATION METHOD THEREOF, AND USE THEREOF

#129
20210369869
2021-12-02

NUCLEIC ACID MOLECULES CONTAINING SPACERS AND METHODS OF USE THEREOF

#130
20210369865
2021-12-02

Exosomes Comprising RNA Therapeutics

#131
20210324343
2021-10-21

RECOMBINANT ADENO-ASSOCIATED VIRUS PARTICLE PURIFICATION COMPRISING AN AFFINITY PURIFICATION STEP

#132
20210319870
2021-10-14

Smart multidosing

#133
20210316016
2021-10-14

Vector for gene silencing and replacement and methods of use thereof

#134
20210308067
2021-10-07

Extracellular vesicles for inhalation

#135
20210300755
2021-09-30

Lipid nanoparticle compositions and methods for mRNA delivery

#136
20210299280
2021-09-30

Compositions and methods for transient gene therapy with enhanced stability

#137
20210285013
2021-09-16

GENE THERAPY FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA

#138
20210283274
2021-09-16

Adeno-associated virus virions with variant capsid and methods of use thereof

#139
20210283272
2021-09-16

GENE THERAPY FOR TREATING PROPIONIC ACIDEMIA

#140
20210283271
2021-09-16

rAAV with chemically modified capsid

#141
20210275689
2021-09-09

Processes of preparing mRNA-loaded lipid nanoparticles

#142
20210253644
2021-08-19

CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND METHODS OF USE IN GENE THERAPY OF HUMAN LIVER CANCER

#143
20210252164
2021-08-19

COMBINATORIAL GENE THERAPY

#144
20210220486
2021-07-22

Immunostimulating vector system

#145
20210214217
2021-07-15

Nucleic acid loaded extracellular vesicles

#146
20210206831
2021-07-08

Modified ligand-gated ion channels and methods of use

#147
20210205467
2021-07-08

RAAV with chemically modified capsid

#148
20210198636
2021-07-01

Use of Syncytin for Targeting Drug and Gene Delivery to Lung Tissue

#149
20210147836
2021-05-20

Use of thermostable RNA polymerases to produce RNAs having reduced immunogenicity

#150
20210060177
2021-03-04

Lipid nanoparticle compositions and methods for mRNA delivery

#151
20210054407
2021-02-25

Dry Transfection Compositions and Methods for Making and Using the Same

#152
20210040504
2021-02-11

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#153
20210040156
2021-02-11

CAPSID-MODIFIED RAAV VECTORS AND METHODS OF USE

#154
20210030683
2021-02-04

Dry powder composition comprising long-chain RNA

#155
20200405845
2020-12-31

Scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy

#156
20200392541
2020-12-17

Delivery and use of the CRISPR-CAS systems, vectors and compositions for hepatic targeting and therapy

#157
20200385689
2020-12-10

Scalable production method for AAV

#158
20200383919
2020-12-10

Dry powder composition comprising long-chain RNA

#159
20200354409
2020-11-12

Adenoviral Polypeptide IX Increases Adenoviral Gene Therapy Vector Productivity and Infectivity

#160
20200338214
2020-10-29

Lipid nanoparticle compositions and methods for mRNA delivery

#161
20200338131
2020-10-29

Method

#162
20200325491
2020-10-15

Method of increasing the function of an AAV vector

#163
20200277587
2020-09-03

Evolution of TALENs

#164
20200239869
2020-07-30

Analytical HPLC methods

#165
20200230259
2020-07-23

ISOLATION OF EXTRACELLULAR VESICLES (EVS) FROM RED BLOOD CELLS FOR GENE THERAPY

#166
20200216860
2020-07-09

DELIVERY OF A GENE-EDITING SYSTEM WITH A SINGLE RETROVIRAL PARTICLE AND METHODS OF GENERATION AND USE

#167
20200208176
2020-07-02

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

#168
20200197535
2020-06-25

EXTRACELLULAR VESICLES FOR AGENT DELIVERY

#169
20200138977
2020-05-07

NUCLEIC ACID-CATIONIC POLYMER COMPOSITIONS AND METHODS OF MAKING AND USING THE SAME

#170
20200093892
2020-03-26

METHODS FOR THE TREATMENT OF INFLAMMATORY JOINT DISEASE

#171
20200085973
2020-03-19

LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY

#172
20200071725
2020-03-05

Nucleic acid application primers

#173
20200061210
2020-02-27

NOVEL METHOD FOR GENE THERAPY USING INTRANASAL ADMINISTRATION OF GENETICALLY MODIFIED VIRAL VECTORS

#174
20200060971
2020-02-27

Dry powder composition comprising long-chain RNA

#175
20200054768
2020-02-20

Factor IX encoding nucleotides

#176
20200054767
2020-02-20

Factor IX encoding nucleotides

#177
20200048600
2020-02-13

Electroporation, developmentally-activated cells, pluripotent-like cells, cell reprogramming and regenerative medicine

#178
20200023075
2020-01-23

Lyophilized pharmaceutical compositions for naked DNA gene therapy

#179
20200018752
2020-01-16

METHODS FOR DETERMINING POTENCY OF ADENO-ASSOCIATED VIRUS PREPARATIONS

#180
20200000938
2020-01-02

Method for the treatment of malignancies

#181
20200000937
2020-01-02

GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIES WITH CHIMERIC ANTIGEN RECEPTORS

#182
20190388557
2019-12-26

RAAV with chemically modified capsid

#183
20190382733
2019-12-19

Recombinant adeno-associated virus particle purification comprising an affinity purification step

#184
20190382724
2019-12-19

Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

#185
20190365930
2019-12-05

Formulation

#186
20190338308
2019-11-07

Targeted CRISPR Delivery Platforms

#187
20190336617
2019-11-07

CRISPRs IN SERIES TREATMENT

#188
20190336531
2019-11-07

NATURAL KILLER T (NKT) CELLS COMPRISING ENGINEERED T CELL RECEPTORS

#189
20190321489
2019-10-24

Lipid nanoparticle compositions and methods for mRNA delivery

#190
20190321463
2019-10-24

SCALABLE MANUFACTURING PLATFORM FOR VIRAL VECTOR PURIFICATION AND VIRAL VECTORS SO PURIFIED FOR USE IN GENE THERAPY

#191
20190321295
2019-10-24

Method of Encapsulating a Nucleic Acid in a Lipid Nanoparticle Host

#192
20190315808
2019-10-17

Adenoviral Polypeptide IX Increases Adenoviral Gene Therapy Vector Productivity and Infectivity

#193
20190307900
2019-10-10

COMPOSITIONS AND METHODS FOR TREATING MACULAR DYSTROPHY

#194
20190298859
2019-10-03

Endosomolytic agents for gene therapy

#195
20190290780
2019-09-26

Compositions and methods for mitochondrial genome editing

#196
20190282710
2019-09-19

B-CELL-MIMETIC CELLS

#197
20190276846
2019-09-12

Seeding An Adherent Cell Bioreactor With Non-Adherent Cells Increases Seeding Density Limit And Reduces Required Expansion Time

#198
20190270989
2019-09-05

Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes

#199
20190262475
2019-08-29

COMPOSITION AND METHODS OF GENOME EDITING OF B-CELLS

#200
20190255513
2019-08-22

Process for recovering viral products using functionalised chromatography media

#201
20190231902
2019-08-01

Method for delivering RNA to neurons to treat herpes infections

#202
20190231899
2019-08-01

Vector for gene silencing and replacement and methods of use thereof

#203
20190218574
2019-07-18

Method of increasing the function of an AAV vector

#204
20190216949
2019-07-18

Adeno-associated virus particle with mutated capsid and methods of use thereof

#205
20190201550
2019-07-04

Compositions and methods for treating CEP290-associated disease

#206
20190201548
2019-07-04

GENE EDITING AND TARGETED TRANSCRIPTIONAL MODULATION FOR ENGINEERING ERYTHROID CELLS

#207
20190192689
2019-06-27

Lipid nanoparticle compositions and methods for mRNA delivery

#208
20190184035
2019-06-20

BCL11A HOMING ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE

#209
20190175764
2019-06-13

Gene therapy

#210
20190175762
2019-06-13

Dependent Component Genome Editing Gene Drives

#211
20190175761
2019-06-13

Lipid nanoparticle compositions and methods for mRNA delivery

#212
20190169264
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Modified ligand-gated ion channels and methods of use

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2019-06-06

Treating metastatic cancer and model systems for metastatic disease

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2019-06-06

METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES

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20190167814
2019-06-06

Treatment And/Or Prevention Of DNA-Triplet Repeat Diseases Or Disorders

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20190167813
2019-06-06

Compositions and methods of replication deficient adenoviral vectors for vaccine applications

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20190167810
2019-06-06

Exosomes comprising therapeutic polypeptides

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2019-05-30

GENE THERAPY FOR THE TREATMENT OF ALDEHYDE DEHYDROGENASE DEFICIENCY

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2019-05-30

Means and methods to treat dystonia

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Method of engineering drug-specific hypersensitive t-cells for immunotherapy by gene inactivation

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2019-05-23

Therapeutic applications of CPF1-based genome editing

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20190151473
2019-05-23

Methods and compositions for treatment of disorders and diseases involving RDH12

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2019-05-23

NANOPARTICLES COMPRISING PROTEIN-POLYNUCLEOTIDE COMPLEXES AND FOR DELIVERING PROTEIN BASED COMPLEXES

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2019-05-16

Engineered nucleases useful for treatment of hemophilia A

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2019-05-09

GENERATION OF GENETICALLY ENGINEERED ANIMALS BY CRISPR/CAS9 GENOME EDITING IN SPERMATOGONIAL STEM CELLS

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20190133950
2019-05-09

Dry powder composition comprising long-chain RNA

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2019-04-18

TREATMENT OF NEUROPATHY WITH DNA CONSTRUCT EXPRESSING HGF ISOFORMS WITH REDUCED INTERFERENCE FROM GABAPENTINOIDS

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Nanocapsule delivery system for ribonucleoproteins

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2019-03-21

METHODS AND COMPOSITIONS UTILIZING CPF1 FOR RNA-GUIDED GENE EDITING

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SCALABLE PRODUCTION METHOD FOR AAV

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Isolation of extracellular vesicles (EVs) from red blood cells for gene therapy

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Immunostimulating vector system

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2019-02-07

Methods and compositions for modifying a mutant dystrophin gene in a cell's genome

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2019-02-07

MATERIALS AND METHODS FOR TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) OR OMENN SYNDROME

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20190002917
2019-01-03

Gene therapy for treating familial hypercholesterolemia

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20180333486
2018-11-22

Engineered Cells Expressing Multiple Immunomodulators and Uses Thereof

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20180333434
2018-11-22

Use of common gamma chain cytokines for the visualization, isolation and genetic modification of memory T lymphocytes

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20180318448
2018-11-08

Method for determining presence or absence of suffering from malignant lymphoma or leukemia, and agent for treatment and/or prevention of leukemia

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2018-11-08

Method for the treatment of malignancies

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20180296699
2018-10-18

Personalized medicine therapeutic MiniCircle

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20180291351
2018-10-11

Recombinant Influenza Viruses for Vaccines and Gene Therapy

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20180250424
2018-09-06

COMPOSITIONS AND METHODS FOR PROMOTING HOMOLOGY DIRECTED REPAIR

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2018-08-23

Evolution of TALENs

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2018-08-09

Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer

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20180169146
2018-06-21

Compositions related to rna in circularized form

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20180154023
2018-06-07

NUCLEIC ACID-CATIONIC POLYMER COMPOSITIONS AND METHODS OF MAKING AND USING THE SAME

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20180148686
2018-05-31

Intervention for tendinopathy

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20180147146
2018-05-31

Dry powder composition comprising long-chain RNA

#249
20180142031
2018-05-24

Smart car devices, DE car polypeptides, side CARs and uses thereof

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20180126006
2018-05-10

Nucleic acid molecules containing spacers outside ITR

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20180057841
2018-03-01

Method of increasing the function of an AAV vector

#252
20180055939
2018-03-01

EXCIPIENTS FOR USE IN ADENO-ASSOCIATED VIRUS PHARMACEUTICAL FORMULATIONS AND PHARMACEUTICAL FORMULATIONS MADE THEREWITH

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20180051275
2018-02-22

Method for purifying viral vectors

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20180036428
2018-02-08

Capsid modified rAAV vectors and methods of use

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20180028687
2018-02-01

Extracellular vesicles for agent delivery

#256
20180000968
2018-01-04

Poly(Beta-Amino Ester)s With Additives for Drug Delivery

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20170368201
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Scalable manufacturing process to produce recombinant lentiviral vectors in serum-free suspension cell culture system

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Method for producing therapeutic corneal endothelial substitute cell sphere

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20170266269
2017-09-21

METHODS OF IDENTIFYING ANTIGENS FOR VACCINES

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2017-08-24

Lipid nanoparticle compositions and methods for MRNA delivery

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2017-08-03

CONDITIONAL CYTOTOXIC GENE THERAPY VECTOR FOR SELECTABLE STEM CELL MODIFICATION FOR ANTI HIV GENE THERAPY

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20170216457
2017-08-03

Messenger RNA nanoparticles and preparation method therefor

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20170196809
2017-07-13

Method of encapsulating a nucleic acid in a lipid nanoparticle host

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20170175135
2017-06-22

Virus Purification

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20170173184
2017-06-22

Treatment of lysosomal storage diseases

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2017-06-22

Drug delivery particle and method for producing the same

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20170128569
2017-05-11

Engineered cells expressing multiple immunomodulators and uses thereof

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20170073702
2017-03-16

Virus purification

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20170051309
2017-02-23

Seeding An Adherent Cell Bioreactor With Non-Adherent Cells Increases Seeding Density Limit And Reduces Required Expansion Time

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2017-02-09

In vivo Gene Engineering with Adenoviral Vectors

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2017-02-09

Lyophilized lentiviral vector particles, compositions and methods

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2016-10-13

Gene therapy to prevent reactions to allergens

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2016-09-08

Methods for genetic modification of stem cells

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2016-09-01

DRY TRANSFECTION COMPOSITIONS AND METHODS FOR MAKING AND USING THE SAME

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Methods for the treatment of inflammatory joint disease

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2016-08-04

Constructs and methods for delivering molecules via viral vectors with blunted innate immune responses

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2016-07-21

Scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy

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2016-06-30

Nucleic acid-encapsulating polymer micelle complex and method for producing same

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2016-06-23

Nanocomplex containing amphipathic peptide useful for efficient transfection of biomolecules

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2016-06-02

Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy

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2016-06-02

DELIVERY, ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETING AND MODELING DISEASES AND DISORDERS OF POST MITOTIC CELLS

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2016-05-19

Recombinant influenza viruses for vaccines and gene therapy

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20160106865
2016-04-21

Capsid-modified rAAV vectors and methods of use

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2016-03-24

Drug delivery system and method

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2016-02-11

Scalable production method for AAV

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2016-02-04

Vectors comprising stuffer/filler polynucleotide sequences and methods of use

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Compositions and Methods for Parkinson's Disease Treatment by BDNF-flag Gene Transfer through Neurotensin Polyplex to Nigral Dopamine Neuro

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Automated generation of genetically modified stem cells

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2015-10-29

Use of common γ chain cytokines for the visualization, isolation and genetic modification of memory T lymphocytes

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Reduction stimulus-responsive gene delivery system and preparation and application thereof

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EXCIPIENTS FOR USE IN ADENO-ASSOCIATED VIRUS PHARMACEUTICAL FORMULATIONS, AND PHARMACEUTICAL FORMULATIONS MADE THEREWITH

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20150166967
2015-06-18

Recombinant influenza vectors with tandem transcription units

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20150159173
2015-06-11

Method of increasing the function of an AAV vector

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20150152393
2015-06-04

Avian adenoassociated virus and uses thereof

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20150110743
2015-04-23

Long lasting drug formulations

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2014-10-30

SCALABLE MANUFACTURING PROCESS TO PRODUCE RECOMBINANT LENTIVIRAL VECTORS IN SERUM-FREE SUSPENSION CELL CULTURE SYSTEM

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2014-09-25

Process for preparing purified nucleic acid and the use thereof

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20140221636
2014-08-07

Bovine adeno-associated viral (BAAV) vector and uses thereof

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2014-07-24

Reassortant influenza viruses for vaccines

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20140065204
2014-03-06

COMPOSITIONS AND METHODS FOR DELIVERING NUCLEIC ACID TO A CELL