JP2017512466A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2017512466A5 JP2017512466A5 JP2016556768A JP2016556768A JP2017512466A5 JP 2017512466 A5 JP2017512466 A5 JP 2017512466A5 JP 2016556768 A JP2016556768 A JP 2016556768A JP 2016556768 A JP2016556768 A JP 2016556768A JP 2017512466 A5 JP2017512466 A5 JP 2017512466A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- hotc
- sequence
- seq
- acid sequence
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- 239000013603 viral vector Substances 0.000 claims 16
- 150000007523 nucleic acids Chemical group 0.000 claims 14
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 10
- 102100028200 Ornithine transcarbamylase, mitochondrial Human genes 0.000 claims 8
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 claims 6
- 241000700605 Viruses Species 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 108091026890 Coding region Proteins 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 206010016654 Fibrosis Diseases 0.000 claims 2
- 101000986595 Homo sapiens Ornithine transcarbamylase, mitochondrial Proteins 0.000 claims 2
- 102100027105 Lymphocyte-specific protein 1 Human genes 0.000 claims 2
- 208000000599 Ornithine Carbamoyltransferase Deficiency Disease Diseases 0.000 claims 2
- 206010052450 Ornithine transcarbamoylase deficiency Diseases 0.000 claims 2
- 208000035903 Ornithine transcarbamylase deficiency Diseases 0.000 claims 2
- 102000002248 Thyroxine-Binding Globulin Human genes 0.000 claims 2
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims 2
- 239000004480 active ingredient Substances 0.000 claims 2
- 210000003494 hepatocyte Anatomy 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 108010040838 lymphocyte-specific protein p50 Proteins 0.000 claims 2
- 201000011278 ornithine carbamoyltransferase deficiency Diseases 0.000 claims 2
- XUIIKFGFIJCVMT-UHFFFAOYSA-N thyroxine-binding globulin Natural products IC1=CC(CC([NH3+])C([O-])=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-UHFFFAOYSA-N 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 230000007882 cirrhosis Effects 0.000 claims 1
- 208000019425 cirrhosis of liver Diseases 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000004761 fibrosis Effects 0.000 claims 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 claims 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020054236A JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| US61/950,157 | 2014-03-09 | ||
| PCT/US2015/019513 WO2015138348A1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020054236A Division JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017512466A JP2017512466A (ja) | 2017-05-25 |
| JP2017512466A5 true JP2017512466A5 (enExample) | 2018-03-29 |
| JP6822841B2 JP6822841B2 (ja) | 2021-01-27 |
Family
ID=52737408
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556768A Active JP6822841B2 (ja) | 2014-03-09 | 2015-03-09 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
| JP2020054236A Active JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020054236A Active JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
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) | BR122023023004A2 (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 (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6491879B2 (ja) | 2011-04-29 | 2019-03-27 | セレクタ バイオサイエンシーズ インコーポレーテッドＳｅｌｅｃｔａ Ｂｉｏｓｃｉｅｎｃｅｓ，Ｉｎｃ． | 制御性ｂ細胞を誘導するための寛容原性合成ナノキャリア |
| EA201592106A3 (ru) | 2013-05-03 | 2016-08-31 | Селекта Байосайенсиз, Инк. | Локальное сопутствующее введение толерогенных синтетических наноносителей для снижения гиперчувствительности типа i и гиперчувствительности типа iv |
| US9867885B2 (en) | 2013-07-30 | 2018-01-16 | Phaserx, Inc. | Block copolymers |
| LT3116900T (lt) * | 2014-03-09 | 2020-12-10 | The Trustees Of The University Of Pennsylvania | Kompozicijos, naudotinos ornitino transkarbamilazės (otc) nepakankamumo gydymui |
| KR20240123409A (ko) | 2014-09-07 | 2024-08-13 | 셀렉타 바이오사이언시즈, 인크. | 유전자 편집 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물 |
| BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
| CA2974503A1 (en) | 2015-01-21 | 2016-07-28 | Phaserx, Inc. | 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 |
| EP3452101A2 (en) | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
| SG11201811603WA (en) | 2016-07-26 | 2019-02-27 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| 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 |
| US11400109B2 (en) | 2016-11-10 | 2022-08-02 | Translate Bio, Inc. | Subcutaneous delivery of messenger RNA |
| EP3562510A4 (en) | 2016-12-30 | 2021-01-06 | Genevant Sciences GmbH | BRANCHED PEG MOLECULES AND ASSOCIATED COMPOSITIONS AND PROCEDURES |
| BR112019013576A2 (pt) * | 2016-12-30 | 2020-02-04 | Univ Pennsylvania | terapia genica para o tratamento da fenilcetonuria |
| EP3565605A1 (en) * | 2017-01-03 | 2019-11-13 | 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 |
| 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 |
| KR102625470B1 (ko) * | 2017-05-31 | 2024-01-16 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN120905307A (zh) | 2017-08-09 | 2025-11-07 | 比奥维拉迪维治疗股份有限公司 | 核酸分子及其用途 |
| US11859215B2 (en) | 2017-11-22 | 2024-01-02 | Modernatx, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
| SG11202010830WA (en) | 2018-05-09 | 2020-11-27 | Biomarin Pharm Inc | Methods of treating phenylketonuria |
| 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 |
| JP2021523914A (ja) | 2018-05-15 | 2021-09-09 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | パーキンソン病を治療するための組成物および方法 |
| CA3106639A1 (en) * | 2018-07-16 | 2020-01-23 | Selecta Biosciences, Inc. | Methods and compositions of otc constructs and vectors |
| WO2020016655A2 (en) | 2018-07-17 | 2020-01-23 | 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 |
| ES2960692T3 (es) * | 2018-12-06 | 2024-03-06 | Arcturus Therapeutics Inc | Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa |
| 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 |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of 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 |
| WO2021188892A1 (en) * | 2020-03-19 | 2021-09-23 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| CN115803064A (zh) | 2020-05-12 | 2023-03-14 | 宾夕法尼亚州大学信托人 | 用于drg特异性降低转基因表达的组合物 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2022224372A1 (ja) * | 2021-04-21 | 2022-10-27 | 学校法人自治医科大学 | オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン |
| CN117881696A (zh) * | 2021-08-23 | 2024-04-12 | 比奥维拉迪维治疗股份有限公司 | 具有反向末端重复序列的封闭末端dna产生 |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| TW202330914A (zh) * | 2022-01-21 | 2023-08-01 | 賓州大學委員會 | 用於鳥胺酸胺甲醯基轉移酶(otc)缺乏症之活體內核酸酶介導的治療之組成物及方法 |
| WO2024081592A2 (en) * | 2022-10-11 | 2024-04-18 | Pacific Northwest Research Institute | Constructs relating to genetic disorders |
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 |
| US20020037867A1 (en) | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| IL128779A0 (en) | 1996-09-06 | 2000-01-31 | Univ Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| JP2001500014A (ja) | 1996-09-06 | 2001-01-09 | 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生産のためにｃｒｅ―ｌｏｘを用いる方法 |
| 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 |
| 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 |
| AU5070298A (en) | 1996-12-05 | 1998-06-29 | Introgene B.V. | Genetic modification of primate hemopoietic repopulating stem cells |
| ATE364718T1 (de) | 1997-04-01 | 2007-07-15 | Solexa Ltd | Verfahren zur vervielfältigung von nukleinsäure |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | 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 |
| WO1999015685A1 (en) | 1997-09-19 | 1999-04-01 | The Trustees Of The University Of Pennsylvania | Methods and cell line useful for production of recombinant adeno-associated viruses |
| 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 |
| 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 |
| 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 |
| ATE286138T1 (de) | 1998-03-20 | 2005-01-15 | Univ Pennsylvania | Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| 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 |
| JP4573437B2 (ja) | 1998-11-05 | 2010-11-04 | 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 | アデノ随伴ウイルス血清型１核酸配列、ベクターおよび同一物を含有する宿主細胞 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| CA2384814A1 (en) | 1999-09-29 | 2001-04-05 | The Trustees Of The University Of Pennsylvania | Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor |
| 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 |
| US7115391B1 (en) | 1999-10-01 | 2006-10-03 | Genovo, Inc. | Production of recombinant AAV using adenovirus comprising AAV rep/cap genes |
| EP1222299A1 (en) | 1999-10-01 | 2002-07-17 | Genovo, Incorporated | Production of recombinant aav using adenovirus comprising aav rep/cap genes |
| 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 |
| 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 |
| 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 |
| ES2327609T3 (es) | 2000-06-01 | 2009-11-02 | University Of North Carolina At Chapel Hill | Procedimientos y compuestos para controlar la liberacion de vectores de parvovirus reconbinantes. |
| 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 |
| WO2002095073A1 (en) | 2001-05-18 | 2002-11-28 | Wisconsin Alumni Research Foundation | Method for the synthesis of dna sequences |
| WO2003016475A2 (en) * | 2001-08-14 | 2003-02-27 | The General Hospital Corporation | Nucleic acid and amino acid sequences involved in pain |
| AU2002347813A1 (en) | 2001-10-04 | 2003-04-14 | Carlos Estuardo Aguilar-Cordova | Chimeric viral vectors for gene therapy |
| BR122016004546B8 (pt) | 2001-11-13 | 2021-07-27 | Univ Pennsylvania | vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus |
| DK1453547T3 (en) | 2001-12-17 | 2016-12-05 | Univ Pennsylvania | ADENOASSOCATED VIRUS (AAV) SEROTYPE 8 SEQUENCES, VECTORS CONTAINING THESE AND APPLICATIONS THEREOF |
| EP3339430A1 (en) | 2001-12-17 | 2018-06-27 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, 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 |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| CA3072423A1 (en) | 2003-09-30 | 2005-04-14 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| EP3409296A1 (en) | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | 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 |
| CA2722238C (en) | 2008-04-22 | 2017-11-28 | Life Sciences Research Partners Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| EP2620161A1 (en) | 2008-05-13 | 2013-07-31 | 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 |
| KR20110095292A (ko) | 2008-11-06 | 2011-08-24 | 유니버시티 오브 워싱톤 | 다중블록 공중합체 |
| CA3006395C (en) | 2008-11-07 | 2022-05-31 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| SMT201800656T1 (it) | 2009-07-24 | 2019-01-11 | Immune Design Corp | Vettori lentivirali non integranti |
| EP2281579A1 (en) | 2009-08-05 | 2011-02-09 | BioNTech AG | Vaccine composition comprising 5'-Cap modified RNA |
| CN102869779A (zh) | 2010-03-29 | 2013-01-09 | 宾夕法尼亚大学托管会 | 药理学诱导的转基因消融系统 |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | 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 |
| EP4043025A1 (en) | 2011-06-08 | 2022-08-17 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| JP6151707B2 (ja) | 2011-10-27 | 2017-06-21 | マサチューセッツ インスティテュート オブ テクノロジー | 薬物内包微小球の形成が可能なｎ−末端で官能基化されたアミノ酸誘導体 |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| AU2013243955B2 (en) | 2012-04-02 | 2018-02-22 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
| CN108949772A (zh) | 2012-04-02 | 2018-12-07 | 现代泰克斯公司 | 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 |
| CN104349794B (zh) | 2012-06-08 | 2019-01-04 | 埃泽瑞斯公司 | 信使rna的肺递送 |
| US9867885B2 (en) | 2013-07-30 | 2018-01-16 | Phaserx, Inc. | Block copolymers |
| LT3116900T (lt) | 2014-03-09 | 2020-12-10 | The Trustees Of The University Of Pennsylvania | Kompozicijos, naudotinos ornitino transkarbamilazės (otc) nepakankamumo gydymui |
-
2015
- 2015-03-09 LT LTEP15712226.8T patent/LT3116900T/lt unknown
- 2015-03-09 WO PCT/US2015/019513 patent/WO2015138348A1/en not_active Ceased
- 2015-03-09 HR HRP20201544TT patent/HRP20201544T1/hr unknown
- 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 US US15/124,620 patent/US9890365B2/en active Active
- 2015-03-09 MX MX2016011686A patent/MX377804B/es active IP Right Grant
- 2015-03-09 PL PL15712226T patent/PL3116900T3/pl unknown
- 2015-03-09 DK DK15712226.8T patent/DK3116900T3/da active
- 2015-03-09 JP JP2016556768A patent/JP6822841B2/ja active Active
- 2015-03-09 ES ES15712226T patent/ES2821938T3/es active Active
- 2015-03-09 KR KR1020167027891A patent/KR102390075B1/ko active Active
- 2015-03-09 HU HUE15712226A patent/HUE051311T2/hu unknown
- 2015-03-09 AU AU2015229750A patent/AU2015229750A1/en not_active Abandoned
- 2015-03-09 SM SM20200531T patent/SMT202000531T1/it unknown
- 2015-03-09 RS RS20201154A patent/RS60902B1/sr unknown
- 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 US US15/122,853 patent/US10167454B2/en active Active
- 2015-03-09 BR BR112016020688-6A patent/BR112016020688B1/pt active IP Right Grant
- 2015-03-09 EP EP20184346.3A patent/EP3778627A1/en not_active Withdrawn
- 2015-03-09 EP EP15712226.8A patent/EP3116900B1/en active Active
- 2015-03-09 CA CA2939950A patent/CA2939950C/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