HRP20201544T1 - Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) - Google Patents
Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) Download PDFInfo
- Publication number
- HRP20201544T1 HRP20201544T1 HRP20201544TT HRP20201544T HRP20201544T1 HR P20201544 T1 HRP20201544 T1 HR P20201544T1 HR P20201544T T HRP20201544T T HR P20201544TT HR P20201544 T HRP20201544 T HR P20201544T HR P20201544 T1 HRP20201544 T1 HR P20201544T1
- Authority
- HR
- Croatia
- Prior art keywords
- hotc
- nucleic acid
- sequence
- acid sequence
- viral vector
- Prior art date
Links
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)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Hematology (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| EP15712226.8A EP3116900B1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
| PCT/US2015/019513 WO2015138348A1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20201544T1 true HRP20201544T1 (hr) | 2021-02-19 |
Family
ID=52737408
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20201544TT HRP20201544T1 (hr) | 2014-03-09 | 2015-03-09 | Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US10167454B2 (enExample) |
| EP (2) | EP3116900B1 (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) | WO2015138357A2 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9987354B2 (en) | 2011-04-29 | 2018-06-05 | Selecta Biosciences, Inc. | Tolerogenic synthetic nanocarriers for antigen-specific deletion of T effector cells |
| CN110251675A (zh) | 2013-05-03 | 2019-09-20 | 西莱克塔生物科技公司 | 降低或预防响应于非变应原性抗原的过敏反应的致耐受性合成纳米载体 |
| EP3027221A1 (en) | 2013-07-30 | 2016-06-08 | PhaseRx, Inc. | Block copolymers and their conjugates or complexes with oligonucleotides |
| WO2015138357A2 (en) * | 2014-03-09 | 2015-09-17 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of otc deficency |
| WO2016037161A2 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| JP2018509387A (ja) | 2015-01-21 | 2018-04-05 | フェーズアールエックス インコーポレイテッド | 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム |
| WO2017059902A1 (en) | 2015-10-07 | 2017-04-13 | Biontech Rna Pharmaceuticals Gmbh | 3' utr sequences for stabilization of rna |
| US11078247B2 (en) | 2016-05-04 | 2021-08-03 | Curevac Ag | RNA encoding a therapeutic protein |
| IL263801B2 (en) | 2016-07-26 | 2024-01-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| AU2017313917B2 (en) | 2016-08-18 | 2023-12-21 | The Regents Of The University Of California | CRISPR-Cas genome engineering via a modular AAV delivery system |
| US11400109B2 (en) | 2016-11-10 | 2022-08-02 | Translate Bio, Inc. | Subcutaneous delivery of messenger RNA |
| 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 |
| EP3565605A1 (en) * | 2017-01-03 | 2019-11-13 | ethris GmbH | Ornithine transcarbamylase coding polyribonucleotides and formulations thereof |
| ES3052719T3 (en) * | 2017-01-10 | 2026-01-13 | Childrens Medical Res Institute | Polynucleotides and vectors for the expression of transgenes |
| ES3036482T3 (en) | 2017-03-11 | 2025-09-19 | Cartesian Therapeutics Inc | Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant |
| AU2018275549B2 (en) * | 2017-05-31 | 2024-12-19 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor IX gene expression cassettes and their use |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| TWI904068B (zh) | 2017-08-09 | 2025-11-11 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| JP7423522B2 (ja) * | 2017-11-22 | 2024-01-29 | モダーナティエックス・インコーポレイテッド | 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド |
| JP2021522811A (ja) | 2018-05-09 | 2021-09-02 | ビオマリン プハルマセウトイカル インコーポレイテッド | フェニルケトン尿症の治療方法 |
| 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 |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| CA3106639A1 (en) * | 2018-07-16 | 2020-01-23 | Selecta Biosciences, Inc. | Methods and compositions of otc constructs and vectors |
| AU2019304569B2 (en) | 2018-07-17 | 2023-07-06 | Helixmith Co., Ltd | Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms |
| WO2020033863A1 (en) * | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | 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 |
| KR20210130158A (ko) | 2019-01-31 | 2021-10-29 | 오레곤 헬스 앤드 사이언스 유니버시티 | Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법 |
| AU2020287627A1 (en) | 2019-06-04 | 2021-11-18 | Selecta Biosciences, Inc. | Formulations and doses of PEGylated uricase |
| WO2021178510A1 (en) * | 2020-03-03 | 2021-09-10 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| JP7817176B2 (ja) | 2020-03-04 | 2026-02-18 | ポセイダ セラピューティクス，インコーポレイティド | 代謝性肝障害の治療のための組成物と方法 |
| 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 |
| EP4162059A1 (en) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| JPWO2022224372A1 (enExample) * | 2021-04-21 | 2022-10-27 | ||
| AR126845A1 (es) * | 2021-08-23 | 2023-11-22 | Bioverativ Therapeutics Inc | Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| MX2024009027A (es) * | 2022-01-21 | 2024-08-06 | Univ Pennsylvania | Metodos para el tratamiento de deficiencia de ornitina transcarbamilasa (otc). |
| JP2025534674A (ja) * | 2022-10-11 | 2025-10-17 | パシフィック・ノースウェスト・リサーチ・インスティテュート | 遺伝性障害に関する構築物 |
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| 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 |
| EP0950111A1 (en) | 1996-09-06 | 1999-10-20 | The Trustees Of The University Of Pennsylvania | Methods using cre-lox for production of recombinant adeno-associated viruses |
| CN1233291A (zh) | 1996-09-06 | 1999-10-27 | 宾西法尼亚大学托管会 | 重组腺伴随病毒定向基因治疗的方法 |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | 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 |
| US20020037867A1 (en) | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav4 vector and uses thereof |
| EP0938578B1 (en) | 1996-12-05 | 2004-02-04 | Crucell Holland 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 |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | 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 |
| WO1999015677A1 (en) | 1997-09-19 | 1999-04-01 | The Trustees Of The University Of Pennsylvania | Method for gene transfer using bcl2 and compositions useful therein |
| EP1015619A1 (en) | 1997-09-19 | 2000-07-05 | The Trustees Of The University Of Pennsylvania | Methods and cell line useful for production of recombinant adeno-associated viruses |
| CA2303768C (en) | 1997-09-19 | 2009-11-24 | The Trustees Of The University Of Pennsylvania | Methods and vector constructs useful for production of recombinant aav |
| DE69922934T2 (de) | 1998-03-20 | 2005-12-08 | The Trustees Of The University Of Pennsylvania | Zusammensetzungen und Verfahren zur helfer-freien Herstellung von rekombinanten 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 |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| 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 |
| PT1127150E (pt) | 1998-11-05 | 2007-08-22 | Univ Pennsylvania | ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm'' |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| 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 |
| AU2409200A (en) | 1999-06-02 | 2000-12-28 | Trustees Of The University Of Pennsylvania, The | Compositions and methods useful for production of recombinant viruses which require helper viruses |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| EP1218035A2 (en) | 1999-09-29 | 2002-07-03 | The Trustees Of The University Of Pennsylvania | Rapid peg-modification of viral vectors |
| 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 |
| JP2003511037A (ja) | 1999-10-01 | 2003-03-25 | ジェノボ， インコーポレイテッド | ＡＡＶｒｅｐ／ｃａｐ遺伝子を含むアデノウイルスを使用する組換えＡＡＶの産生 |
| US7115391B1 (en) | 1999-10-01 | 2006-10-03 | Genovo, Inc. | Production of recombinant AAV using adenovirus comprising AAV rep/cap genes |
| 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 |
| WO2001040455A2 (en) | 1999-12-03 | 2001-06-07 | The Trustees Of The University Of Pennsylvania | Compositions and methods for increasing packaging and yields of recombinant adenoviruses using multiple packaging signals |
| 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 |
| 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 |
| 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 |
| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| JP2004520812A (ja) | 2000-08-30 | 2004-07-15 | ハプロゲン・エルエルシー | 対立遺伝子を決定するための方法 |
| WO2002095073A1 (en) | 2001-05-18 | 2002-11-28 | Wisconsin Alumni Research Foundation | 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 |
| US20040086485A1 (en) | 2001-10-04 | 2004-05-06 | Aguilar-Cordova Carlos Estuardo | Chemeric viral vectors for gene therapy |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 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 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| PT2573170T (pt) | 2001-12-17 | 2018-03-26 | Univ Pennsylvania | Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações |
| 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 |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP2799547B1 (en) | 2006-11-08 | 2016-12-21 | Veritas Bio, LLC | In Vivo Delivery of RNA to a Target Cell |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| CA2724105C (en) | 2008-05-13 | 2017-09-05 | University Of Washington | Diblock copolymers and polynucleotide complexes thereof for delivery into cells |
| EP2352522A4 (en) | 2008-11-06 | 2014-03-05 | Univ Washington | BISPECIFIC AUXILIARIES FOR INTRA-CELLULAR RELEASE |
| EP2364330B1 (en) | 2008-11-06 | 2015-03-25 | University Of Washington | Multiblock copolymers |
| WO2010053572A2 (en) | 2008-11-07 | 2010-05-14 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| HRP20140327T4 (hr) | 2009-07-24 | 2017-11-03 | Immune Design Corp | Lentiviralni vektori pseudotipizirani sa glikoproteinom ovojnice virusa sindbis |
| 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 |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9181675B2 (en) | 2011-05-12 | 2015-11-10 | Hitachi Construction Machinery Co., Ltd. | Construction machine |
| PT2717893T (pt) | 2011-06-08 | 2019-08-20 | Translate Bio Inc | Composições de nanopartículas lipídicas e métodos para transferência de arnm |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| MX363734B (es) | 2011-10-27 | 2019-03-29 | Massachusetts Inst Technology | Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco. |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| HK1206612A1 (en) * | 2012-04-02 | 2016-01-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of secreted proteins |
| EP2834259A4 (en) * | 2012-04-02 | 2016-08-24 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| EP3027221A1 (en) * | 2013-07-30 | 2016-06-08 | PhaseRx, Inc. | Block copolymers and their conjugates or complexes with oligonucleotides |
| WO2015138357A2 (en) | 2014-03-09 | 2015-09-17 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of otc deficency |
-
2015
- 2015-03-09 WO PCT/US2015/019536 patent/WO2015138357A2/en not_active Ceased
- 2015-03-09 SM SM20200531T patent/SMT202000531T1/it unknown
- 2015-03-09 AU AU2015229750A patent/AU2015229750A1/en not_active Abandoned
- 2015-03-09 HU HUE15712226A patent/HUE051311T2/hu unknown
- 2015-03-09 RS RS20201154A patent/RS60902B1/sr unknown
- 2015-03-09 PT PT157122268T patent/PT3116900T/pt unknown
- 2015-03-09 BR BR112016020688-6A patent/BR112016020688B1/pt active IP Right Grant
- 2015-03-09 JP JP2016556768A patent/JP6822841B2/ja active Active
- 2015-03-09 HR HRP20201544TT patent/HRP20201544T1/hr unknown
- 2015-03-09 LT LTEP15712226.8T patent/LT3116900T/lt unknown
- 2015-03-09 ES ES15712226T patent/ES2821938T3/es active Active
- 2015-03-09 DK DK15712226.8T patent/DK3116900T3/da active
- 2015-03-09 US US15/122,853 patent/US10167454B2/en active Active
- 2015-03-09 PL PL15712226T patent/PL3116900T3/pl unknown
- 2015-03-09 WO PCT/US2015/019513 patent/WO2015138348A1/en not_active Ceased
- 2015-03-09 SI SI201531366T patent/SI3116900T1/sl unknown
- 2015-03-09 BR BR122023023004-3A patent/BR122023023004A2/pt not_active Application Discontinuation
- 2015-03-09 EP EP15712226.8A patent/EP3116900B1/en active Active
- 2015-03-09 EP EP20184346.3A patent/EP3778627A1/en not_active Withdrawn
- 2015-03-09 KR KR1020167027891A patent/KR102390075B1/ko active Active
- 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 US15/124,620 patent/US9890365B2/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 |
|---|---|---|
| HRP20201544T1 (hr) | Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) | |
| JP2017512466A5 (enExample) | ||
| HRP20201063T1 (hr) | Pripravci za liječenje mpsi | |
| JP2016512683A5 (enExample) | ||
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| HRP20240580T1 (hr) | ISPORUKA ADENO-POVEZANIM VIRUSNIM VEKTOROM β-SARKOGLIKANA I MIKRORNA-29 I LIJEČENJE MIŠIĆNE DISTROFIJE | |
| HRP20231077T1 (hr) | Genska terapija za pigmentni retinitis | |
| JP2020522269A5 (enExample) | ||
| JP2018506261A5 (enExample) | ||
| HRP20241180T1 (hr) | Modificirani faktor ix, te pripravci, postupci i upotreba za prijenos gena u stanice, organe i tkiva | |
| JP2018531609A5 (enExample) | ||
| RU2018116076A (ru) | Модифицированные гены атаксии фридрейха и векторы для генной терапии | |
| JP2019503649A5 (enExample) | ||
| JP2018108113A5 (enExample) | ||
| JP2020514286A5 (enExample) | ||
| HRP20201324T1 (hr) | Vektori aav koji sadrže faktor viii koji kodira gen | |
| RU2017125234A (ru) | Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний | |
| JP2018522529A5 (enExample) | ||
| RU2018136611A (ru) | Генная терапия для лечения гемофилии a | |
| JP2020510428A5 (enExample) | ||
| JP2017509632A5 (enExample) | ||
| RU2018119710A (ru) | Кодирующие фактор viii варианты с пониженным содержанием cpg, композиции и способы, и применение в лечении нарушений гемостаза | |
| JP2017532966A5 (enExample) | ||
| HRP20201831T1 (hr) | Optimizirani promoter rpe65 i kodirajuće sekvence | |
| JP2020533276A5 (enExample) |