PT3116900T - Composições úteis no tratamento da deficiência de ornitina transcarbamilase (otc) - Google Patents
Composições úteis no tratamento da deficiência de ornitina transcarbamilase (otc)Info
- Publication number
- PT3116900T PT3116900T PT157122268T PT15712226T PT3116900T PT 3116900 T PT3116900 T PT 3116900T PT 157122268 T PT157122268 T PT 157122268T PT 15712226 T PT15712226 T PT 15712226T PT 3116900 T PT3116900 T PT 3116900T
- Authority
- PT
- Portugal
- Prior art keywords
- otc
- deficiency
- treatment
- compositions useful
- ornithine transcarbamylase
- Prior art date
Links
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 title 1
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 title 1
- 230000007812 deficiency Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
- C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y201/00—Transferases transferring one-carbon groups (2.1)
- C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
- C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—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
- C12N2750/14032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2750/14033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—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
- C12N2750/14071—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
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT3116900T true PT3116900T (pt) | 2020-10-08 |
Family
ID=52737408
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT157122268T PT3116900T (pt) | 2014-03-09 | 2015-03-09 | Composições úteis no tratamento da deficiência de ornitina transcarbamilase (otc) |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US9890365B2 (enExample) |
| EP (2) | EP3778627A1 (enExample) |
| JP (2) | JP6822841B2 (enExample) |
| KR (1) | KR102390075B1 (enExample) |
| AU (2) | AU2015229750A1 (enExample) |
| BR (2) | BR112016020688B1 (enExample) |
| CA (1) | CA2939950C (enExample) |
| CL (2) | CL2016002235A1 (enExample) |
| CY (1) | CY1123378T1 (enExample) |
| DK (1) | DK3116900T3 (enExample) |
| ES (1) | ES2821938T3 (enExample) |
| HR (1) | HRP20201544T1 (enExample) |
| HU (1) | HUE051311T2 (enExample) |
| IL (2) | IL247329B (enExample) |
| LT (1) | LT3116900T (enExample) |
| MX (1) | MX377804B (enExample) |
| PL (1) | PL3116900T3 (enExample) |
| PT (1) | PT3116900T (enExample) |
| RS (1) | RS60902B1 (enExample) |
| SI (1) | SI3116900T1 (enExample) |
| SM (1) | SMT202000531T1 (enExample) |
| WO (2) | WO2015138348A1 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN107320734A (zh) | 2011-04-29 | 2017-11-07 | 西莱克塔生物科技公司 | 从合成纳米载体中控制释放免疫抑制剂 |
| CN105307641A (zh) | 2013-05-03 | 2016-02-03 | 西莱克塔生物科技公司 | 用于降低不期望体液免疫应答的给药组合 |
| AU2014296278C1 (en) | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| BR112016020688B1 (pt) * | 2014-03-09 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos |
| EA201790551A1 (ru) | 2014-09-07 | 2017-07-31 | Селекта Байосайенсиз, Инк. | Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору |
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| EP3247328B1 (en) | 2015-01-21 | 2026-01-07 | Genevant Sciences Gmbh | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| WO2017059902A1 (en) | 2015-10-07 | 2017-04-13 | Biontech Rna Pharmaceuticals Gmbh | 3' utr sequences for stabilization of rna |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| JP6994018B2 (ja) | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| CA3034089A1 (en) | 2016-08-18 | 2018-02-22 | The Regents Of The University Of California | Crispr-cas genome engineering via a modular aav delivery system |
| JP7560946B2 (ja) | 2016-11-10 | 2024-10-03 | トランスレイト バイオ， インコーポレイテッド | メッセンジャーｒｎａの皮下送達 |
| WO2018126084A1 (en) | 2016-12-30 | 2018-07-05 | Phaserx, Inc. | Branched peg molecules and related compositions and methods |
| EP3562494A4 (en) | 2016-12-30 | 2020-08-19 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS |
| US11104887B2 (en) | 2017-01-03 | 2021-08-31 | Ethris Gmbh | Ornithine transcarbamylase coding polyribonucleotides and formulations thereof |
| US11938197B2 (en) | 2017-01-10 | 2024-03-26 | The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) | Polynucleotides and vectors for the expression of transgenes |
| EP4599848A3 (en) | 2017-03-11 | 2025-12-03 | Cartesian Therapeutics, Inc. | Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant |
| JP7119009B2 (ja) * | 2017-05-31 | 2022-08-16 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| MX2020001593A (es) | 2017-08-09 | 2020-07-13 | Bioverativ Therapeutics Inc | Moleculas de acido nucleico y usos de las mismas. |
| EP3714048B1 (en) * | 2017-11-22 | 2025-04-09 | ModernaTX, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
| KR20210008501A (ko) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 페닐케톤뇨증을 치료하는 방법 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| EP3793689A1 (en) * | 2018-05-15 | 2021-03-24 | Translate Bio, Inc. | Subcutaneous delivery of messenger rna |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| CN112771070A (zh) * | 2018-07-16 | 2021-05-07 | 西莱克塔生物科技公司 | Otc构建体和载体的方法和组合物 |
| CA3106078A1 (en) | 2018-07-17 | 2020-01-23 | Junghun Lee | Treatment of neuropathy with dna constructs expressing igf-1 isoforms |
| JP7602454B2 (ja) | 2018-08-09 | 2024-12-18 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CA3122080A1 (en) * | 2018-12-06 | 2020-06-11 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| CN119390787A (zh) | 2019-01-31 | 2025-02-07 | 俄勒冈健康与科学大学 | 用于aav衣壳的使用转录依赖性定向进化的方法 |
| US12553041B2 (en) | 2019-06-04 | 2026-02-17 | Cartesian Therapeutics Inc. | Formulations and doses of pegylated uricase |
| EP4114470A4 (en) * | 2020-03-03 | 2024-04-17 | Arcturus Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY |
| CN115515646A (zh) | 2020-03-04 | 2022-12-23 | 波赛达治疗公司 | 用于治疗代谢性肝病的组合物和方法 |
| US20230235353A1 (en) * | 2020-03-19 | 2023-07-27 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| IL298138A (en) | 2020-05-12 | 2023-01-01 | Univ Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| EP4328313A4 (en) * | 2021-04-21 | 2025-06-25 | Jichi Medical University | Adeno-associated virus virion for treating ornithine transcarbamylase deficiency |
| AU2022334714A1 (en) * | 2021-08-23 | 2024-04-04 | Bioverativ Therapeutics Inc. | Closed-end dna production with inverted terminal repeat sequences |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP4466367A4 (en) * | 2022-01-21 | 2026-03-18 | Univ Pennsylvania | METHODS FOR THE TREATMENT OF ORNITHIN TRANSCARBAMYLASE (OTC) DEFICIENCY |
| CA3268024A1 (en) * | 2022-10-11 | 2024-04-18 | Pacific Northwest Research Institute | CONSTRUCTIONS RELATED TO GENETIC DISORDERS |
| WO2025229995A1 (ja) * | 2024-04-30 | 2025-11-06 | 学校法人自治医科大学 | 核酸導入用ベクター、核酸導入方法、ポリヌクレオチド、及びベクター |
Family Cites Families (81)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US1016745A (en) | 1911-04-11 | 1912-02-06 | Edwin C Henrikson | Table-caster. |
| 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 |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5652224A (en) | 1995-02-24 | 1997-07-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism |
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US20020037867A1 (en) | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US5866552A (en) | 1996-09-06 | 1999-02-02 | The Trustees Of The University Of Pennsylvania | Method for expressing a gene in the absence of an immune response |
| EP1696036B1 (en) | 1996-09-06 | 2010-04-21 | The Trustees of The University of Pennsylvania | Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells |
| WO1998010086A1 (en) | 1996-09-06 | 1998-03-12 | Trustees Of The University Of Pennsylvania | Methods using cre-lox for production of recombinant adeno-associated viruses |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Aav4 vector and uses thereof |
| CA2274146C (en) | 1996-12-05 | 2007-02-06 | Introgene B.V. | Genetic modification of primate hemopoietic repopulating stem cells |
| EP2327797B1 (en) | 1997-04-01 | 2015-11-25 | Illumina Cambridge Limited | Method of nucleic acid sequencing |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| JP2001517454A (ja) | 1997-09-19 | 2001-10-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株 |
| WO1999014354A1 (en) | 1997-09-19 | 1999-03-25 | The Trustees Of The University Of The Pennsylvania | Methods and vector constructs useful for production of recombinant aav |
| AU9774998A (en) | 1997-09-19 | 1999-04-12 | Trustees Of The University Of Pennsylvania, The | Method for gene transfer using bcl2 and compositions useful therein |
| ATE286138T1 (de) | 1998-03-20 | 2005-01-15 | Univ Pennsylvania | Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren |
| US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| DE69939169D1 (de) | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| DE69936104T2 (de) | 1998-11-05 | 2008-01-17 | The Trustees Of The University Of Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US6387368B1 (en) | 1999-02-08 | 2002-05-14 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| JP2003501067A (ja) | 1999-06-02 | 2003-01-14 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法 |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| US6365394B1 (en) | 1999-09-29 | 2002-04-02 | The Trustees Of The University Of Pennsylvania | Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus |
| WO2001023001A2 (en) | 1999-09-29 | 2001-04-05 | The Trustees Of The University Of Pennsylvania | Rapid peg-modification |
| AU7841400A (en) | 1999-10-01 | 2001-05-10 | Genovo, Incorporated | Production of recombinant aav using adenovirus comprising aav rep/cap genes |
| US7115391B1 (en) | 1999-10-01 | 2006-10-03 | Genovo, Inc. | Production of recombinant AAV using adenovirus comprising AAV rep/cap genes |
| JP2003523180A (ja) | 1999-12-03 | 2003-08-05 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 多重のパッケージングシグナルを用いる組み換えアデノウイルスのパッケージングおよび収量を増加するための組成物および方法 |
| US6821512B1 (en) | 1999-12-03 | 2004-11-23 | The Trustees Of The University Of Pennsylvania | Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals |
| US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| US6468524B1 (en) | 2000-03-22 | 2002-10-22 | The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | AAV4 vector and uses thereof |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| WO2001091802A1 (en) * | 2000-05-30 | 2001-12-06 | Baylor College Of Medicine | Chimeric viral vectors for gene therapy |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CN1293204C (zh) | 2000-08-30 | 2007-01-03 | 戴诺生物技术有限公司 | 等位基因的测定方法 |
| IL158487A0 (en) | 2001-05-18 | 2004-05-12 | Wisconsin Alumni Res Found | Method for the synthesis of dna sequences |
| AU2002324700A1 (en) * | 2001-08-14 | 2003-03-03 | Bayer Ag | Nucleic acid and amino acid sequences involved in pain |
| JP2005526001A (ja) | 2001-10-04 | 2005-09-02 | アグイラルーコルドバ，カリオス，エスツアルド | 遺伝子療法用キメラ型ウイルスベクター |
| CN103589692B (zh) * | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| EP1463805B1 (en) | 2001-12-17 | 2014-10-22 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| AU2002367943A1 (en) | 2001-12-18 | 2003-12-22 | University Of North Carolina At Chapel Hill | Improved reagents and methods for producing parvoviruses |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| 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 |
| DK2094086T3 (da) | 2006-11-08 | 2013-11-25 | Veritas Bio LLC | Indgivelse in vivo af dobbeltstrenget rna til en målcelle |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| US9617548B2 (en) * | 2008-04-22 | 2017-04-11 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| MX2010012238A (es) | 2008-05-13 | 2011-04-11 | Univ Washington | Copolimeros dibloque y sus complejos con polinucleotidos destinados a introducirlos en celulas. |
| EP2364330B1 (en) | 2008-11-06 | 2015-03-25 | University Of Washington | Multiblock copolymers |
| EP2352522A4 (en) | 2008-11-06 | 2014-03-05 | Univ Washington | BISPECIFIC AUXILIARIES FOR INTRA-CELLULAR RELEASE |
| WO2010053572A2 (en) | 2008-11-07 | 2010-05-14 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| EP2456786B2 (en) | 2009-07-24 | 2017-04-19 | Immune Design Corp | Lentiviral vectors pseudotyped with a sindbis virus envelope glycoprotein |
| EP2281579A1 (en) | 2009-08-05 | 2011-02-09 | BioNTech AG | Vaccine composition comprising 5'-Cap modified RNA |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2012153638A1 (ja) | 2011-05-12 | 2012-11-15 | 日立建機株式会社 | 建設機械 |
| KR20220025112A (ko) | 2011-06-08 | 2022-03-03 | 샤이어 휴먼 지네틱 테라피즈 인크. | Mrna 전달을 위한 지질 나노입자 조성물 및 방법 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US9512073B2 (en) | 2011-10-27 | 2016-12-06 | Massachusetts Institute Of Technology | Amino acid-, peptide-and polypeptide-lipids, isomers, compositions, and uses thereof |
| WO2013151664A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| CN104411338A (zh) * | 2012-04-02 | 2015-03-11 | 现代治疗公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| EA037448B1 (ru) | 2012-06-08 | 2021-03-30 | Этрис Гмбх | Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция |
| AU2014296278C1 (en) | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| BR112016020688B1 (pt) * | 2014-03-09 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos |
-
2015
- 2015-03-09 BR BR112016020688-6A patent/BR112016020688B1/pt active IP Right Grant
- 2015-03-09 SI SI201531366T patent/SI3116900T1/sl unknown
- 2015-03-09 RS RS20201154A patent/RS60902B1/sr unknown
- 2015-03-09 US US15/124,620 patent/US9890365B2/en active Active
- 2015-03-09 BR BR122023023004-3A patent/BR122023023004A2/pt not_active Application Discontinuation
- 2015-03-09 JP JP2016556768A patent/JP6822841B2/ja active Active
- 2015-03-09 KR KR1020167027891A patent/KR102390075B1/ko active Active
- 2015-03-09 WO PCT/US2015/019513 patent/WO2015138348A1/en not_active Ceased
- 2015-03-09 CA CA2939950A patent/CA2939950C/en active Active
- 2015-03-09 MX MX2016011686A patent/MX377804B/es active IP Right Grant
- 2015-03-09 US US15/122,853 patent/US10167454B2/en active Active
- 2015-03-09 HR HRP20201544TT patent/HRP20201544T1/hr unknown
- 2015-03-09 PL PL15712226T patent/PL3116900T3/pl unknown
- 2015-03-09 AU AU2015229750A patent/AU2015229750A1/en not_active Abandoned
- 2015-03-09 WO PCT/US2015/019536 patent/WO2015138357A2/en not_active Ceased
- 2015-03-09 PT PT157122268T patent/PT3116900T/pt unknown
- 2015-03-09 DK DK15712226.8T patent/DK3116900T3/da active
- 2015-03-09 ES ES15712226T patent/ES2821938T3/es active Active
- 2015-03-09 HU HUE15712226A patent/HUE051311T2/hu unknown
- 2015-03-09 EP EP20184346.3A patent/EP3778627A1/en not_active Withdrawn
- 2015-03-09 SM SM20200531T patent/SMT202000531T1/it unknown
- 2015-03-09 LT LTEP15712226.8T patent/LT3116900T/lt unknown
- 2015-03-09 EP EP15712226.8A patent/EP3116900B1/en active Active
-
2016
- 2016-08-17 IL IL247329A patent/IL247329B/en active IP Right Grant
- 2016-09-06 CL CL2016002235A patent/CL2016002235A1/es unknown
-
2018
- 2018-01-03 US US15/861,427 patent/US10626382B2/en active Active
- 2018-11-15 US US16/191,709 patent/US10781430B2/en active Active
-
2019
- 2019-08-12 CL CL2019002280A patent/CL2019002280A1/es unknown
-
2020
- 2020-02-19 AU AU2020201190A patent/AU2020201190B2/en active Active
- 2020-03-25 JP JP2020054236A patent/JP6920500B2/ja active Active
- 2020-07-01 IL IL275799A patent/IL275799B2/en unknown
- 2020-08-17 US US16/995,572 patent/US11732246B2/en active Active
- 2020-09-24 CY CY20201100902T patent/CY1123378T1/el unknown
-
2023
- 2023-06-22 US US18/339,457 patent/US20230416700A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL275799B (en) | Useful preparations for the treatment of ornithine transcarbamylase deficiency | |
| IL287789A (en) | Methods of producing mogrosides and compositions comprising same and uses thereof | |
| EP3881680B8 (en) | Methods and compositions relating to microbial treatment | |
| EP3145493A4 (en) | Methods and compositions relating to exosomes | |
| IL250568A0 (en) | Anti-methanogenic preparations and their uses | |
| EP3134130A4 (en) | Compositions and methods to treating hemoglobinopathies | |
| HUE056172T2 (hu) | Szelenoorganikus vegyületeket tartalmazó készítmények és eljárások alkalmazásukra | |
| PT3102555T (pt) | Composições de compostos e seus usos | |
| ZA201408056B (en) | Compositions and methods for the treatment of mucositis | |
| EP3148517A4 (en) | Compositions and methods to promote bone formation | |
| EP3198275A4 (en) | Sterilization compositions and methods | |
| EP3190886A4 (en) | Compositions and methods of use thereof | |
| IL252683A0 (en) | Pyridyl-cycloalkyl-substituted carboxylic acids, preparations containing them and their medical uses | |
| PL3113774T3 (pl) | Kompozycje grapiprantu i sposoby ich zastosowania | |
| ZA201700244B (en) | Reduction of infections in healthcare settings using photocatalytic compositions | |
| PL2968478T3 (pl) | Kompozycje i sposoby do leczenia karboksyhemoglobinemii | |
| IL246587A0 (en) | Preparations for use in the treatment of allergic conditions | |
| IL257406A (en) | Methods and compositions related to long half-life coagulation | |
| HK1231846A1 (en) | Compositions of compounds and uses thereof | |
| HK1233497A1 (en) | Compositions of selenoorganic compounds and methods of use thereof | |
| GB201418410D0 (en) | use of compositions of matter | |
| GB201404391D0 (en) | Novel compositions having use in therapy | |
| GB201502816D0 (en) | Novel Compositions having use in therapy |