MX2022009883A - Vectores de genoterapia para tratar enfermedades cardíacas. - Google Patents
Vectores de genoterapia para tratar enfermedades cardíacas.Info
- Publication number
- MX2022009883A MX2022009883A MX2022009883A MX2022009883A MX2022009883A MX 2022009883 A MX2022009883 A MX 2022009883A MX 2022009883 A MX2022009883 A MX 2022009883A MX 2022009883 A MX2022009883 A MX 2022009883A MX 2022009883 A MX2022009883 A MX 2022009883A
- Authority
- MX
- Mexico
- Prior art keywords
- heart disease
- gene therapy
- therapy vectors
- treating heart
- prevention
- Prior art date
Links
- 208000019622 heart disease Diseases 0.000 title abstract 4
- 239000013598 vector Substances 0.000 title abstract 2
- 238000001415 gene therapy Methods 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 230000002265 prevention Effects 0.000 abstract 2
- 208000031229 Cardiomyopathies Diseases 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 102000004987 Troponin T Human genes 0.000 abstract 1
- 108090001108 Troponin T Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 210000002845 virion Anatomy 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0276—Knock-out vertebrates
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4716—Muscle proteins, e.g. myosin, actin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0657—Cardiomyocytes; Heart cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0375—Animal model for cardiovascular diseases
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Cardiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Heart & Thoracic Surgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Urology & Nephrology (AREA)
- Vascular Medicine (AREA)
- Cell Biology (AREA)
- Environmental Sciences (AREA)
- Rheumatology (AREA)
- Transplantation (AREA)
Abstract
La presente descripción proporciona métodos y composiciones útiles para el tratamiento o la prevención enfermedades cardiacas. En particular, la presente descripción proporciona un vector que comprende un promotor de troponina T modificado enlazado operativamente a un producto génico terapéutico para el tratamiento o la prevención de enfermedades cardíacas, p. ej., miocardiopatía. El producto génico puede ser MYBPC3. La descripción también proporciona viriones de virus adenoasociados recombinantes (rAAV), genomas virales de rAAV, y casetes de expresión y composiciones farmacéuticas de estos. La descripción proporciona además métodos para tratar una enfermedad o trastorno, tal como una enfermedad cardíaca.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202062976160P | 2020-02-13 | 2020-02-13 | |
US202063047633P | 2020-07-02 | 2020-07-02 | |
PCT/US2021/017699 WO2021163357A2 (en) | 2020-02-13 | 2021-02-11 | Gene therapy vectors for treating heart disease |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022009883A true MX2022009883A (es) | 2022-10-03 |
Family
ID=77273691
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022009883A MX2022009883A (es) | 2020-02-13 | 2021-02-11 | Vectores de genoterapia para tratar enfermedades cardíacas. |
Country Status (11)
Country | Link |
---|---|
US (5) | US11129908B2 (es) |
EP (1) | EP4103214A2 (es) |
JP (1) | JP2023513749A (es) |
KR (1) | KR20220139956A (es) |
CN (1) | CN116096394A (es) |
AU (1) | AU2021218411A1 (es) |
BR (1) | BR112022015979A2 (es) |
CA (1) | CA3171166A1 (es) |
CO (1) | CO2022012917A2 (es) |
IL (1) | IL295529A (es) |
MX (1) | MX2022009883A (es) |
Families Citing this family (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR20220139956A (ko) | 2020-02-13 | 2022-10-17 | 테나야 테라퓨틱스, 인코포레이티드 | 심장 질환을 치료하기 위한 유전자 요법 벡터 |
WO2023108157A1 (en) * | 2021-12-10 | 2023-06-15 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating mybpc3 related hypertrophic cardiomyopathy with a viral vector |
TW202404993A (zh) | 2022-04-11 | 2024-02-01 | 美商特納亞治療股份有限公司 | 具經工程化蛋白殼之腺相關病毒 |
WO2024054864A1 (en) | 2022-09-06 | 2024-03-14 | Tenaya Therapeutics, Inc. | Cardioprotective heart disease therapies |
WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
Family Cites Families (40)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5266488A (en) | 1988-04-06 | 1993-11-30 | The Regents Of The University Of California | Troponin T gene promoter and derivatives thereof |
US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
EP2623605B1 (en) | 2007-04-09 | 2018-11-28 | University of Florida Research Foundation, Inc. | RAAV vector compositions having tyrosine-modified capsid proteins and methods for use |
US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
WO2009137006A2 (en) | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
US8889641B2 (en) | 2009-02-11 | 2014-11-18 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
EP2714709B1 (en) | 2011-05-26 | 2018-04-18 | University of Washington | Compositions for use in cell and gene based methods to improve cardiac function |
CN103987854A (zh) | 2011-07-21 | 2014-08-13 | 小利兰·斯坦福大学托管委员会 | 来自患者的诱导性多能干细胞的心肌细胞及其使用方法 |
US8933048B2 (en) | 2011-09-21 | 2015-01-13 | Case Western Reserve University | Methods of treating cardiomyopathy |
US20130136729A1 (en) | 2011-11-11 | 2013-05-30 | University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group | Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors |
EP2792742A1 (en) | 2013-04-17 | 2014-10-22 | Universitätsklinikum Hamburg-Eppendorf (UKE) | Gene-therapy vectors for treating cardiomyopathy |
WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
CN107250364A (zh) | 2015-01-16 | 2017-10-13 | 华盛顿大学 | 新的微小肌养蛋白及相关使用方法 |
MX2017012097A (es) | 2015-03-24 | 2018-06-15 | Univ California | Variantes de virus adenoasociado y métodos de uso de estas. |
EP3626274A3 (en) | 2015-04-16 | 2020-06-17 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
SG11201804713VA (en) | 2015-12-11 | 2018-07-30 | California Inst Of Techn | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
MX2018007237A (es) | 2015-12-14 | 2018-12-11 | Univ North Carolina Chapel Hill | Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus. |
US11866462B2 (en) | 2016-05-04 | 2024-01-09 | Oregon Health & Science University | Recombinant adeno-associated viral vectors |
EP3469073B1 (en) | 2016-06-09 | 2024-01-10 | Centre National de la Recherche Scientifique (CNRS) | Raav with chemically modified capsid |
AU2018221738A1 (en) | 2017-02-15 | 2019-08-22 | The University Of North Carolina At Chapel Hill | Methods and compositions for gene transfer across the vasculature |
US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
CA3071978A1 (en) | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
JP6825167B2 (ja) | 2017-09-20 | 2021-02-03 | 4ディー モレキュラー セラピューティクス インコーポレイテッド | アデノ随伴ウイルス変異キャプシドおよびその使用方法 |
AU2019209002A1 (en) * | 2018-01-19 | 2020-09-03 | Agency For Science, Technology And Research | Disrupting the LINC complex for treating laminopathy |
JP2021530218A (ja) | 2018-07-11 | 2021-11-11 | ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. | 血液脳関門を超える薬剤の送達のための方法および組成物 |
US11242368B2 (en) | 2018-09-21 | 2022-02-08 | Arizona Board Of Regents On Behalf Of The University Of Arizona | Methods and compositions for rapidly replacing cardiac myosin binding protein-C in sarcomeres |
MX2021006646A (es) | 2018-12-05 | 2021-12-10 | Abeona Therapeutics Inc | Vector viral adenoasociado recombinante para el suministro de genes. |
EA202192501A1 (ru) | 2019-03-21 | 2021-12-16 | Страйдбайо, Инк. | Рекомбинантные аденоассоциированные вирусные векторы |
JP2022527917A (ja) | 2019-03-28 | 2022-06-07 | ザ ジェネラル ホスピタル コーポレイション | 導入遺伝子発現のための操作されたアデノ随伴(aav)ベクター |
US20200405824A1 (en) | 2019-06-26 | 2020-12-31 | University Of Washington | Use of ribonucleotide reductase alone or in combination with micro-dystrophin to treat duchenne muscular dystrophy striated muscle disease |
JP2022545955A (ja) | 2019-08-27 | 2022-11-01 | ディーエヌエークゥオー アーゲー | 停止していない鼓動している心臓への薬物灌流 |
KR20220139956A (ko) | 2020-02-13 | 2022-10-17 | 테나야 테라퓨틱스, 인코포레이티드 | 심장 질환을 치료하기 위한 유전자 요법 벡터 |
US20220023384A1 (en) | 2020-07-08 | 2022-01-27 | Children's Medical Center Corporation | Mybpc3 polypeptides and uses thereof |
AU2021405790A1 (en) | 2020-12-23 | 2023-07-06 | Boehringer Ingelheim International Gmbh | Viral capsid proteins with specificity to heart tissue cells |
JP2024510112A (ja) | 2021-02-22 | 2024-03-06 | ディーエヌエークゥオー アーゲー | 腎臓の局所領域灌流 |
CA3208568A1 (en) | 2021-02-22 | 2022-08-25 | Johannes HOLZMEISTER | Loco-regional perfusion of an unarrested beating heart |
JP2024510108A (ja) | 2021-02-22 | 2024-03-06 | ディーエヌエークゥオー アーゲー | 肝臓の局所領域灌流 |
-
2021
- 2021-02-11 KR KR1020227031127A patent/KR20220139956A/ko unknown
- 2021-02-11 BR BR112022015979A patent/BR112022015979A2/pt unknown
- 2021-02-11 CN CN202180028001.5A patent/CN116096394A/zh active Pending
- 2021-02-11 AU AU2021218411A patent/AU2021218411A1/en active Pending
- 2021-02-11 MX MX2022009883A patent/MX2022009883A/es unknown
- 2021-02-11 CA CA3171166A patent/CA3171166A1/en active Pending
- 2021-02-11 EP EP21714285.0A patent/EP4103214A2/en active Pending
- 2021-02-11 IL IL295529A patent/IL295529A/en unknown
- 2021-02-11 JP JP2022548910A patent/JP2023513749A/ja active Pending
- 2021-03-24 US US17/210,882 patent/US11129908B2/en active Active
- 2021-07-22 US US17/383,138 patent/US20210346521A1/en active Pending
- 2021-10-21 US US17/507,362 patent/US11446397B2/en active Active
-
2022
- 2022-01-21 US US17/581,576 patent/US11759531B2/en active Active
- 2022-09-12 CO CONC2022/0012917A patent/CO2022012917A2/es unknown
-
2023
- 2023-07-28 US US18/361,571 patent/US20230372541A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
EP4103214A2 (en) | 2022-12-21 |
IL295529A (en) | 2022-10-01 |
CA3171166A1 (en) | 2021-08-19 |
US11129908B2 (en) | 2021-09-28 |
US20220160896A1 (en) | 2022-05-26 |
AU2021218411A1 (en) | 2022-10-06 |
US20230372541A1 (en) | 2023-11-23 |
US20210252165A1 (en) | 2021-08-19 |
US20210346521A1 (en) | 2021-11-11 |
JP2023513749A (ja) | 2023-04-03 |
CN116096394A (zh) | 2023-05-09 |
KR20220139956A (ko) | 2022-10-17 |
US11759531B2 (en) | 2023-09-19 |
BR112022015979A2 (pt) | 2022-10-11 |
US11446397B2 (en) | 2022-09-20 |
CO2022012917A2 (es) | 2022-09-20 |
US20220031866A1 (en) | 2022-02-03 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MX2022009883A (es) | Vectores de genoterapia para tratar enfermedades cardíacas. | |
WO2021163357A3 (en) | Gene therapy vectors for treating heart disease | |
BR112021021156A2 (pt) | Terapêuticos anticorpos totalmente humanos pós-tradução modificados | |
JP2016500519A5 (es) | ||
RU2018136611A (ru) | Генная терапия для лечения гемофилии a | |
TW201629225A (zh) | 第九因子基因療法 | |
MX2020001402A (es) | Metodos de terapia genica del factor viii (fviii). | |
MX2021013364A (es) | Composiciones utiles para el tratamiento de la enfermedad de pompe. | |
AR118850A1 (es) | Composiciones útiles en el tratamiento de leucodistrofia metacromática | |
JP2018510620A5 (es) | ||
BR112022004027A2 (pt) | Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte | |
CO2024001148A2 (es) | Casetes de expresión optimizada para genoterapia | |
Robert et al. | Gene transfer of ZMapp antibodies mediated by recombinant adeno-associated virus protects against Ebola infections | |
CO2023009633A2 (es) | Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal | |
MX2021010356A (es) | SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR. | |
Mahoney et al. | Viral Vectors in Gene Therapy and Clinical Aplications | |
MX2022000551A (es) | Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica. | |
CL2023001650A1 (es) | Tratamiento de la enfermedad de danon | |
MX2023000995A (es) | Vector de genoterapia para eef1a2 y usos del mismo. | |
Robert et al. | Antibody delivery mediated by recombinant adeno-associated virus for the treatment of various chronic and infectious diseases | |
MX2023007474A (es) | Proteinas de la capside viral con especificidad para las celulas del tejido cardiaco. | |
Lin et al. | Applications of Virus Vector–Mediated Gene Therapy in China | |
Wu et al. | Half-Life Extension Enhances Drug Efficacy in Adeno-Associated Virus Delivered Gene Therapy | |
BR112022003206A2 (pt) | Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte | |
MX2022004799A (es) | Composiciones y metodos para el tratamiento de los trastornos de almacenamiento de glucogeno. |