AR124216A1 - Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen - Google Patents
Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienenInfo
- Publication number
- AR124216A1 AR124216A1 ARP210103330A ARP210103330A AR124216A1 AR 124216 A1 AR124216 A1 AR 124216A1 AR P210103330 A ARP210103330 A AR P210103330A AR P210103330 A ARP210103330 A AR P210103330A AR 124216 A1 AR124216 A1 AR 124216A1
- Authority
- AR
- Argentina
- Prior art keywords
- capsid
- protein
- motif
- seq
- acid sequence
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 5
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 7
- 210000000234 capsid Anatomy 0.000 abstract 5
- 241000702421 Dependoparvovirus Species 0.000 abstract 4
- 108090000623 proteins and genes Proteins 0.000 abstract 4
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 3
- 239000007900 aqueous suspension Substances 0.000 abstract 3
- 239000000546 pharmaceutical excipient Substances 0.000 abstract 3
- 229920001184 polypeptide Polymers 0.000 abstract 3
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 3
- 102000004169 proteins and genes Human genes 0.000 abstract 3
- 210000002889 endothelial cell Anatomy 0.000 abstract 2
- 230000003511 endothelial effect Effects 0.000 abstract 2
- 230000004927 fusion Effects 0.000 abstract 2
- 230000008685 targeting Effects 0.000 abstract 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 abstract 1
- 108090000565 Capsid Proteins Proteins 0.000 abstract 1
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 150000001413 amino acids Chemical class 0.000 abstract 1
- 210000004556 brain Anatomy 0.000 abstract 1
- 210000004899 c-terminal region Anatomy 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 108020001507 fusion proteins Proteins 0.000 abstract 1
- 102000037865 fusion proteins Human genes 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 238000003780 insertion Methods 0.000 abstract 1
- 230000037431 insertion Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000002105 nanoparticle Substances 0.000 abstract 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 abstract 1
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
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- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—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
- A61K48/0025—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
- A61K48/0041—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 the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
- A61K39/215—Coronaviridae, e.g. avian infectious bronchitis virus
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
- A61K39/42—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum viral
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/525—Virus
- A61K2039/5256—Virus expressing foreign proteins
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Communicable Diseases (AREA)
- Mycology (AREA)
- Marine Sciences & Fisheries (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pulmonology (AREA)
- Oncology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063119863P | 2020-12-01 | 2020-12-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR124216A1 true AR124216A1 (es) | 2023-03-01 |
Family
ID=80050581
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP210103330A AR124216A1 (es) | 2020-12-01 | 2021-12-01 | Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240024507A1 (https=) |
| EP (1) | EP4256065A2 (https=) |
| JP (1) | JP2023551903A (https=) |
| KR (1) | KR20230117157A (https=) |
| CN (1) | CN116670152A (https=) |
| AR (1) | AR124216A1 (https=) |
| AU (1) | AU2021390480A1 (https=) |
| CA (1) | CA3200014A1 (https=) |
| IL (1) | IL303236A (https=) |
| MX (1) | MX2023006444A (https=) |
| TW (1) | TW202237850A (https=) |
| WO (1) | WO2022119871A2 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3947700A4 (en) | 2019-04-01 | 2023-01-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| BR112023006305A2 (pt) | 2020-10-09 | 2023-05-09 | Tenaya Therapeutics Inc | Métodos e composições de terapia gênica com placofilina-2 |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| AU2023307223A1 (en) * | 2022-07-14 | 2025-02-20 | The Broad Institute, Inc. | Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor |
| WO2024207061A1 (en) * | 2023-04-04 | 2024-10-10 | Children's Medical Research Institute | Aav capsids and vectors for transduction of cells of the peripheral nervous system |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| IL326390A (en) | 2023-08-10 | 2026-04-01 | Univ Pennsylvania | Compositions and methods for treating spinal muscular atrophy |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
| WO2025137290A1 (en) * | 2023-12-19 | 2025-06-26 | The Broad Institute, Inc. | Methods and systems directed to therapeutic delivery of the human gba1 gene |
Family Cites Families (97)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| EP0682717A4 (en) | 1993-02-05 | 1997-04-23 | Laporte Group Australia | ANTI-FOAMING COMPOSITE FOR DAIRY. |
| US5869337A (en) | 1993-02-12 | 1999-02-09 | President And Fellows Of Harvard College | Regulated transcription of targeted genes and other biological events |
| US5834266A (en) | 1993-02-12 | 1998-11-10 | President & Fellows Of Harvard College | Regulated apoptosis |
| US6140120A (en) | 1993-02-12 | 2000-10-31 | Board Of Trustees Of Leland Stanford Jr. University | Regulated transcription of targeted genes and other biological events |
| EP0804561B1 (en) | 1993-02-12 | 2009-12-30 | The Board Of Trustees Of The Leland Stanford Junior University | Regulated transcription of targeted genes and other biological events |
| US20020173474A1 (en) | 1993-02-12 | 2002-11-21 | President And Fellows Of Harvard College | Methods & materials involving dimerization-mediated regulation of biological events |
| US5830462A (en) | 1993-02-12 | 1998-11-03 | President & Fellows Of Harvard College | Regulated transcription of targeted genes and other biological events |
| US6972193B1 (en) | 1993-02-12 | 2005-12-06 | Board Of Trustees Of Leland Stanford Junior University | Regulated transcription of targeted genes and other biological events |
| US6150137A (en) | 1994-05-27 | 2000-11-21 | Ariad Pharmaceuticals, Inc. | Immunosuppressant target proteins |
| US6476200B1 (en) | 1994-06-27 | 2002-11-05 | The Johns Hopkins University | Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion |
| US6492106B1 (en) | 1994-06-27 | 2002-12-10 | The Johns Hopkins University | Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6150527A (en) | 1994-08-18 | 2000-11-21 | Ariad Pharmaceuticals, Inc. | Synthetic multimerizing agents |
| US6133456A (en) | 1994-08-18 | 2000-10-17 | Ariad Gene Therapeutics, Inc. | Synthetic multimerizing agents |
| DE69534300T2 (de) | 1994-08-18 | 2006-05-18 | Ariad Gene Therapeutics, Inc., Cambridge | Neues multimerisierendes reagenz |
| US6326166B1 (en) | 1995-12-29 | 2001-12-04 | Massachusetts Institute Of Technology | Chimeric DNA-binding proteins |
| AU719001B2 (en) | 1994-12-29 | 2000-05-04 | Massachusetts Institute Of Technology | Chimeric DNA-binding proteins |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| WO1996041865A1 (en) | 1995-06-07 | 1996-12-27 | Ariad Gene Therapeutics, Inc. | Rapamcycin-based regulation of biological events |
| US5811524A (en) | 1995-06-07 | 1998-09-22 | Idec Pharmaceuticals Corporation | Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic use thereof |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6187757B1 (en) | 1995-06-07 | 2001-02-13 | Ariad Pharmaceuticals, Inc. | Regulation of biological events using novel compounds |
| US6506379B1 (en) | 1995-06-07 | 2003-01-14 | Ariad Gene Therapeutics, Inc. | Intramuscular delivery of recombinant AAV |
| AU731826B2 (en) | 1996-02-28 | 2001-04-05 | Ariad Pharmaceuticals, Inc. | Synthetic Multimerizing Agents |
| US6723531B2 (en) | 1996-04-05 | 2004-04-20 | The Salk Institute For Biological Studies | Method for modulating expression of exogenous genes in mammalian systems, and products related thereto |
| AU735648B2 (en) | 1996-07-12 | 2001-07-12 | Ariad Pharmaceuticals, Inc. | Materials and method for treating or preventing pathogenic fungal infection |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| US6015709A (en) | 1997-08-26 | 2000-01-18 | Ariad Pharmaceuticals, Inc. | Transcriptional activators, and compositions and uses related thereto |
| JP2003524368A (ja) | 1997-08-26 | 2003-08-19 | アリアド ジーン セラピューティクス インコーポレイテッド | ニ量化ドメイン、三量化ドメインまたは四量化ドメインおよび補足的非相同転写活性化ドメイン、転写抑制ドメイン、ｄｎａ結合ドメインまたはリガンド結合ドメインを含む融合蛋白 |
| US6479653B1 (en) | 1997-08-26 | 2002-11-12 | Ariad Gene Therapeutics, Inc. | Compositions and method for regulation of transcription |
| JP2001514007A (ja) | 1997-08-27 | 2001-09-11 | アリアド ジーン セラピューティクス インコーポレイテッド | キメラ転写アクチベーター、ならびにそれに関連する組成物および使用 |
| JP2002508971A (ja) | 1998-01-15 | 2002-03-26 | アリアド・ジーン・セラピューティクス・インコーポレーテッド | 多量体キメラ蛋白質を使用する生物学的イベントの調節 |
| US6984635B1 (en) | 1998-02-13 | 2006-01-10 | Board Of Trustees Of The Leland Stanford Jr. University | Dimerizing agents, their production and use |
| JP2002503667A (ja) | 1998-02-13 | 2002-02-05 | プレジデント・アンド・フェローズ・オブ・ハーバード・カレッジ | 新規な二量体化剤、その製造および使用 |
| US6333318B1 (en) | 1998-05-14 | 2001-12-25 | The Salk Institute For Biological Studies | Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| US6258603B1 (en) | 1998-06-17 | 2001-07-10 | Rohm And Haas Company | Ligands for modulating the expression of exogenous genes via an ecdysone receptor complex |
| US7109317B1 (en) | 1998-11-06 | 2006-09-19 | President And Fellows Of Harvard College | FK506-based regulation of biological events |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| ATE264863T1 (de) | 1999-08-24 | 2004-05-15 | Ariad Gene Therapeutics Inc | 28-epirapaloge |
| US7067526B1 (en) | 1999-08-24 | 2006-06-27 | Ariad Gene Therapeutics, Inc. | 28-epirapalogs |
| AU2001249315A1 (en) | 2000-03-22 | 2001-10-03 | Rheogene, Inc. | Ecdysone receptor-based inducible gene expression system |
| US20040033600A1 (en) | 2001-03-21 | 2004-02-19 | Palli Subba Reddy | Ecdysone receptor-based inducible gene expression system |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| US8105825B2 (en) | 2000-10-03 | 2012-01-31 | Intrexon Corporation | Multiple inducible gene regulation system |
| EP1373470B1 (en) | 2001-02-20 | 2013-04-24 | Intrexon Corporation | Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system |
| WO2002066614A2 (en) | 2001-02-20 | 2002-08-29 | Rheogene Holdings, Inc. | Chimeric retinoid x receptors and their use in a novel ecdysone receptor-based inducible gene expression system |
| EP1534738B1 (en) | 2001-02-20 | 2012-06-20 | Intrexon Corporation | Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system |
| JP4955904B2 (ja) | 2001-02-20 | 2012-06-20 | イントレキソン コーポレーション | 新規エクジソン受容体／無脊椎動物レチノイドｘ受容体−ベースの誘導性遺伝子発現システム |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US20060246079A1 (en) | 2003-11-14 | 2006-11-02 | Morrow Phillip R | Neutralizing human antibodies to anthrax toxin |
| US7935510B2 (en) | 2004-04-30 | 2011-05-03 | Intrexon Corporation | Mutant receptors and their use in a nuclear receptor-based inducible gene expression system |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US7879326B2 (en) | 2007-06-15 | 2011-02-01 | The Board Of Trustees Of The Leland Stanford Junior University | Human neutralizing monoclonal antibodies to H5N1 influenza A virus |
| ITTO20080204A1 (it) | 2008-03-17 | 2009-09-18 | Pomona Biotechnologies Llc | Anticorpi monoclonali atti a reagire con una pluralita di sottotipi del virus influenzale a |
| ITTO20080398A1 (it) | 2008-05-27 | 2009-11-28 | Pomona Biotechnologies Llc | Anticorpi monoclonali aventi proprieta' di cross-neutralizzazione omosubtipica per virus influenzali di tipo a sottotipo h1 |
| CA2731686C (en) | 2008-07-25 | 2020-04-07 | Institute For Research In Biomedicine | Neutralizing anti-influenza a virus antibodies and uses thereof |
| GB0813784D0 (en) | 2008-07-28 | 2008-09-03 | Ct Integrated Photonics Ltd | Optical intergration system |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| CA2761648C (en) | 2009-05-11 | 2019-03-12 | Crucell Holland B.V. | Human binding molecules capable of neutralizing influenza virus h3n2 and uses thereof |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| EP2435575A2 (en) * | 2009-05-28 | 2012-04-04 | Deutsches Krebsforschungszentrum | Modified aav capsid polypeptides |
| IT1395961B1 (it) | 2009-06-01 | 2012-11-02 | Pomona Biotechnologies Llc | Anticorpi monoclonali come medicamento per il trattamento terapeutico e/o profilattico delle infezioni da virus influenzale a (h1n1) di origine suina (s-oiv) |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130037977A1 (en) | 2010-04-08 | 2013-02-14 | Paul A. Burke | Preparation of Lipid Nanoparticles |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| WO2012145572A1 (en) | 2011-04-20 | 2012-10-26 | The Trustees Of The University Of Pennsylvania | Regimens and compositions for aav-mediated passive immunization of airborne pathogens |
| HRP20180291T1 (hr) | 2011-05-27 | 2018-03-23 | Amicus Therapeutics, Inc. | Postupci za sparivanje ciljajućeg peptida na rekombinantne lizosomalne enzime za poboljšano liječenje lizosomskih bolesti nakupljanja |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| EP4331620A3 (en) | 2012-12-07 | 2024-12-04 | Translate Bio, Inc. | Lipidic nanoparticles for mrna delivery |
| AU2014228231B2 (en) | 2013-03-15 | 2019-02-21 | Amicus Therapeutics, Inc. | Chemical crosslinkers |
| AU2014348212C1 (en) | 2013-11-18 | 2018-11-29 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| EP3134431B1 (en) | 2014-04-25 | 2021-04-07 | The Trustees Of The University Of Pennsylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3237605B1 (en) * | 2014-12-24 | 2021-02-24 | Neuorphan Pty Ltd. | Improvements in oligodendroglial cell culturing methods and in methods for treating neurodegenerative disorders by using thyroid hormones or analogues |
| EA201792500A1 (ru) | 2015-05-13 | 2018-04-30 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Aav-опосредованная экспрессия антител против гриппа и способы их использования |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| UY37620A (es) | 2017-02-28 | 2018-08-31 | Univ Pennsylvania | Vacunas contra la gripe mediadas por aav novedosas |
| EP3609542B1 (en) * | 2017-04-11 | 2023-02-22 | Ruprecht-Karls-Universität Heidelberg | Adeno-associated virus library |
| IL276859B2 (en) | 2018-02-27 | 2025-12-01 | Univ Pennsylvania | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
| WO2020160582A1 (de) | 2019-02-04 | 2020-08-13 | Technische Universität Wien | Tübbing aus bewehrtem beton |
| EP3947700A4 (en) * | 2019-04-01 | 2023-01-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| SG11202111375RA (en) | 2019-04-29 | 2021-11-29 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
-
2021
- 2021-12-01 TW TW110144803A patent/TW202237850A/zh unknown
- 2021-12-01 IL IL303236A patent/IL303236A/en unknown
- 2021-12-01 US US18/255,154 patent/US20240024507A1/en active Pending
- 2021-12-01 KR KR1020237020955A patent/KR20230117157A/ko not_active Withdrawn
- 2021-12-01 MX MX2023006444A patent/MX2023006444A/es unknown
- 2021-12-01 EP EP21848319.6A patent/EP4256065A2/en not_active Withdrawn
- 2021-12-01 AU AU2021390480A patent/AU2021390480A1/en not_active Abandoned
- 2021-12-01 AR ARP210103330A patent/AR124216A1/es unknown
- 2021-12-01 JP JP2023533645A patent/JP2023551903A/ja not_active Withdrawn
- 2021-12-01 CA CA3200014A patent/CA3200014A1/en active Pending
- 2021-12-01 WO PCT/US2021/061312 patent/WO2022119871A2/en not_active Ceased
- 2021-12-01 CN CN202180085871.6A patent/CN116670152A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022119871A3 (en) | 2022-07-14 |
| JP2023551903A (ja) | 2023-12-13 |
| US20240024507A1 (en) | 2024-01-25 |
| WO2022119871A2 (en) | 2022-06-09 |
| CA3200014A1 (en) | 2022-06-09 |
| KR20230117157A (ko) | 2023-08-07 |
| CN116670152A (zh) | 2023-08-29 |
| EP4256065A2 (en) | 2023-10-11 |
| AU2021390480A1 (en) | 2023-06-22 |
| MX2023006444A (es) | 2023-08-10 |
| IL303236A (en) | 2023-07-01 |
| AU2021390480A9 (en) | 2024-02-08 |
| TW202237850A (zh) | 2022-10-01 |
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