HU230406B1 - Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására - Google Patents
Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására Download PDFInfo
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Cited By (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10693918B2 (en) | 2017-06-15 | 2020-06-23 | Palo Alto Networks, Inc. | Radio access technology based security in service provider networks |
| US10708306B2 (en) | 2017-06-15 | 2020-07-07 | Palo Alto Networks, Inc. | Mobile user identity and/or SIM-based IoT identity and application identity based security enforcement in service provider networks |
| US11722532B2 (en) | 2017-06-15 | 2023-08-08 | Palo Alto Networks, Inc. | Security for cellular internet of things in mobile networks based on subscriber identity and application identifier |
| US11805153B2 (en) | 2017-06-15 | 2023-10-31 | Palo Alto Networks, Inc. | Location based security in service provider networks |
| US11838326B2 (en) | 2017-06-15 | 2023-12-05 | Palo Alto Networks, Inc. | Mobile equipment identity and/or IoT equipment identity and application identity based security enforcement in service provider networks |
| US12010148B2 (en) | 2017-06-15 | 2024-06-11 | Palo Alto Networks, Inc. | Access point name and application identity based security enforcement in service provider networks |
Families Citing this family (651)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| DE69939169D1 (de) | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| US20040121309A1 (en) | 2002-12-06 | 2004-06-24 | Ecker David J. | Methods for rapid detection and identification of bioagents in blood, bodily fluids, and bodily tissues |
| WO2004060278A2 (en) | 2002-12-06 | 2004-07-22 | Isis Pharmaceuticals, Inc. | Methods for rapid identification of pathogens in humans and animals |
| US7226739B2 (en) | 2001-03-02 | 2007-06-05 | Isis Pharmaceuticals, Inc | Methods for rapid detection and identification of bioagents in epidemiological and forensic investigations |
| US7666588B2 (en) | 2001-03-02 | 2010-02-23 | Ibis Biosciences, Inc. | Methods for rapid forensic analysis of mitochondrial DNA and characterization of mitochondrial DNA heteroplasmy |
| US20030027135A1 (en) | 2001-03-02 | 2003-02-06 | Ecker David J. | Method for rapid detection and identification of bioagents |
| US7718354B2 (en) | 2001-03-02 | 2010-05-18 | Ibis Biosciences, Inc. | Methods for rapid identification of pathogens in humans and animals |
| US7217510B2 (en) | 2001-06-26 | 2007-05-15 | Isis Pharmaceuticals, Inc. | Methods for providing bacterial bioagent characterizing information |
| US8073627B2 (en) | 2001-06-26 | 2011-12-06 | Ibis Biosciences, Inc. | System for indentification of pathogens |
| 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 |
| US7235393B2 (en) | 2002-04-29 | 2007-06-26 | The Trustees Of The University Of Pennsylvania | Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues |
| US7419817B2 (en) | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
| US7220577B2 (en) * | 2002-08-28 | 2007-05-22 | University Of Florida Research Foundation, Inc. | Modified AAV |
| US8046171B2 (en) | 2003-04-18 | 2011-10-25 | Ibis Biosciences, Inc. | Methods and apparatus for genetic evaluation |
| US8057993B2 (en) | 2003-04-26 | 2011-11-15 | Ibis Biosciences, Inc. | Methods for identification of coronaviruses |
| US8158354B2 (en) | 2003-05-13 | 2012-04-17 | Ibis Biosciences, Inc. | Methods for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture |
| US7964343B2 (en) | 2003-05-13 | 2011-06-21 | Ibis Biosciences, Inc. | Method for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture |
| WO2005017101A2 (en) | 2003-05-19 | 2005-02-24 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH | Avian adenoassociated virus (aaav) and uses thereof |
| PL2657248T3 (pl) * | 2003-06-19 | 2017-09-29 | Genzyme Corporation | Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9233131B2 (en) * | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| AU2010201278B2 (en) * | 2003-09-01 | 2012-11-15 | Academisch Medisch Centrum | AAV vectors for in vivo gene therapy of rheumatoid arthritis |
| US8529885B2 (en) * | 2003-09-01 | 2013-09-10 | Academisch Medisch Centrum | AAV vectors for in vivo gene therapy of rheumatoid arthritis |
| US8097416B2 (en) | 2003-09-11 | 2012-01-17 | Ibis Biosciences, Inc. | Methods for identification of sepsis-causing bacteria |
| US20120122102A1 (en) | 2003-09-11 | 2012-05-17 | Rangarajan Sampath | Compositions for use in identification of bacteria |
| US8546082B2 (en) | 2003-09-11 | 2013-10-01 | Ibis Biosciences, Inc. | Methods for identification of sepsis-causing bacteria |
| 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 |
| AU2011250849B2 (en) * | 2003-09-30 | 2013-09-12 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor |
| WO2005056807A2 (en) | 2003-12-04 | 2005-06-23 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health | Bovine adeno-associated viral (baav) vector and uses thereof |
| US8163895B2 (en) | 2003-12-05 | 2012-04-24 | Ibis Biosciences, Inc. | Compositions for use in identification of orthopoxviruses |
| US7666592B2 (en) | 2004-02-18 | 2010-02-23 | Ibis Biosciences, Inc. | Methods for concurrent identification and quantification of an unknown bioagent |
| US8119336B2 (en) | 2004-03-03 | 2012-02-21 | Ibis Biosciences, Inc. | Compositions for use in identification of alphaviruses |
| WO2006078279A2 (en) * | 2004-04-28 | 2006-07-27 | The Trustees Of The University Of Pennsylvania | Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations |
| US9592284B2 (en) * | 2004-04-28 | 2017-03-14 | The Trustees Of The University Of Pennsylvania | Immunization regimen with E4-deleted adenovirus prime and E1-deleted adenovirus boost |
| JP4810533B2 (ja) | 2004-05-24 | 2011-11-09 | アイビス バイオサイエンシズ インコーポレイティッド | ディジタルスレショルド化による選択的イオン濾過作用を用いた質量分光測定法 |
| US20050266411A1 (en) | 2004-05-25 | 2005-12-01 | Hofstadler Steven A | Methods for rapid forensic analysis of mitochondrial DNA |
| US7811753B2 (en) | 2004-07-14 | 2010-10-12 | Ibis Biosciences, Inc. | Methods for repairing degraded DNA |
| WO2006073496A2 (en) * | 2004-07-30 | 2006-07-13 | Targeted Genetics Corporation | Recombinant aav based vaccine methods |
| US7309589B2 (en) * | 2004-08-20 | 2007-12-18 | Vironix Llc | Sensitive detection of bacteria by improved nested polymerase chain reaction targeting the 16S ribosomal RNA gene and identification of bacterial species by amplicon sequencing |
| EA200700807A1 (ru) * | 2004-10-05 | 2007-08-31 | Мерц Фарма Гмбх Унд Ко. Кгаа | Новые циклические и ациклические пропеноны для лечения заболеваний цнс |
| US8084207B2 (en) | 2005-03-03 | 2011-12-27 | Ibis Bioscience, Inc. | Compositions for use in identification of papillomavirus |
| US20060205040A1 (en) | 2005-03-03 | 2006-09-14 | Rangarajan Sampath | Compositions for use in identification of adventitious viruses |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| US8026084B2 (en) | 2005-07-21 | 2011-09-27 | Ibis Biosciences, Inc. | Methods for rapid identification and quantitation of nucleic acid variants |
| WO2007100397A2 (en) * | 2005-11-28 | 2007-09-07 | Isis Pharmaceuticals, Inc. | Compositions for use in identification of adventitious contaminant viruses |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| CN101528916B (zh) | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| PL2044199T3 (pl) * | 2006-07-25 | 2013-04-30 | Celladon Corp | Przedłużony, podawany z prądem krwi, nasierdziowy, wieńcowy wlew wektorów wirusowych na bazie wirusów towarzyszących adenowirusom, zawierający SERCA2a do terapii genowej |
| WO2008143627A2 (en) | 2006-09-14 | 2008-11-27 | Ibis Biosciences, Inc. | Targeted whole genome amplification method for identification of pathogens |
| JP5680304B2 (ja) | 2007-02-23 | 2015-03-04 | アイビス バイオサイエンシズ インコーポレイティッド | 迅速な法医学的dna分析法 |
| US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
| DK2623605T3 (en) | 2007-04-09 | 2019-03-25 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US9598724B2 (en) | 2007-06-01 | 2017-03-21 | Ibis Biosciences, Inc. | Methods and compositions for multiple displacement amplification of nucleic acids |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| EP2910637A1 (en) | 2008-05-20 | 2015-08-26 | University of Florida Research Foundation, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| EP2349549B1 (en) | 2008-09-16 | 2012-07-18 | Ibis Biosciences, Inc. | Mixing cartridges, mixing stations, and related kits, and system |
| EP2347254A2 (en) | 2008-09-16 | 2011-07-27 | Ibis Biosciences, Inc. | Sample processing units, systems, and related methods |
| EP2344893B1 (en) | 2008-09-16 | 2014-10-15 | Ibis Biosciences, Inc. | Microplate handling systems and methods |
| EP2396803A4 (en) | 2009-02-12 | 2016-10-26 | Ibis Biosciences Inc | IONIZATION PROBE ASSEMBLIES |
| DK2425000T3 (da) | 2009-04-30 | 2019-05-13 | Univ Pennsylvania | Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| WO2010143761A1 (ko) * | 2009-06-12 | 2010-12-16 | (주)바이오니아 | 미지시료 내 감염성 미생물을 신속하게 검출하는 방법 |
| US8950604B2 (en) | 2009-07-17 | 2015-02-10 | Ibis Biosciences, Inc. | Lift and mount apparatus |
| WO2011008972A1 (en) | 2009-07-17 | 2011-01-20 | Ibis Biosciences, Inc. | Systems for bioagent identification |
| US20120244127A1 (en) | 2009-10-01 | 2012-09-27 | The Trustees Of The University Of Pennsylvania | AAV Vectors Expressing SEC10 for Treating Kidney Damage |
| US9890408B2 (en) | 2009-10-15 | 2018-02-13 | Ibis Biosciences, Inc. | Multiple displacement amplification |
| 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 |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| CA3050894C (en) | 2010-04-23 | 2022-10-18 | University Of Massachusetts | Multicistronic expression constructs |
| CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| US9309534B2 (en) | 2010-07-12 | 2016-04-12 | Universidad Autonoma De Barcelona | Gene therapy composition for use in diabetes treatment |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| EP3699286A1 (en) | 2011-04-20 | 2020-08-26 | The Trustees of the University of Pennsylvania | Regimens and compositions for aav-mediated passive immunization of airborne pathogens |
| WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| DK3693025T3 (da) | 2011-04-22 | 2022-01-10 | Univ California | Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf |
| EP3795581A3 (en) * | 2011-08-24 | 2021-06-09 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| US20130136729A1 (en) * | 2011-11-11 | 2013-05-30 | University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group | Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| WO2013130393A1 (en) * | 2012-02-28 | 2013-09-06 | Cornell University | Aav-directed persistent expression of an anti-nicotine antibody gene for smoking cessation |
| US10004811B2 (en) * | 2012-04-13 | 2018-06-26 | Cornell University | Development of a highly efficient second generation nicotine-conjugate vaccine to treat nicotine addiction |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| EP2692868A1 (en) | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| WO2014124282A1 (en) | 2013-02-08 | 2014-08-14 | The Trustees Of The University Of Pennsylvania | Enhanced aav-mediated gene transfer for retinal therapies |
| RS61387B2 (sr) | 2013-02-15 | 2024-06-28 | Bioverativ Therapeutics Inc | Gen optimizovanog faktora viii |
| US8957044B2 (en) | 2013-03-01 | 2015-02-17 | Wake Forest University Health Sciences | Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM) |
| BR112015021036A2 (pt) | 2013-03-15 | 2017-10-10 | Univ Pennsylvania | composições e métodos para tratar mps1 |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| NZ716102A (en) | 2013-07-22 | 2021-12-24 | The Children’S Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| WO2015117010A2 (en) * | 2014-01-31 | 2015-08-06 | Temple University Of The Commonwealth System Of Higher Education | Bag3 as a target for therapy of heart failure |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| 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 |
| ES2822551T3 (es) | 2014-03-17 | 2021-05-04 | Adverum Biotechnologies Inc | Composiciones y métodos para la expresión génica mejorada en células de los conos |
| JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのrAAVベースの組成物および方法 |
| PT3628334T (pt) | 2014-03-21 | 2023-09-26 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| EP2933335A1 (en) | 2014-04-18 | 2015-10-21 | Genethon | A method of treating peripheral neuropathies and motor neuron diseases |
| WO2015164723A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| US11555059B2 (en) | 2014-04-25 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | LDLR variants and their use in compositions for reducing cholesterol levels |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| CR20160519A (es) | 2014-05-13 | 2017-01-27 | The Trustees Of The Univ Of Pennysylvania | Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos |
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| PL3164394T3 (pl) | 2014-07-03 | 2020-09-07 | Board Of Regents, The University Of Texas System | Inhibitory GLS1 do leczenia chorób |
| PT4012035T (pt) | 2014-09-16 | 2025-02-11 | Genzyme Corp | Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc) |
| EP4012035B1 (en) | 2014-09-16 | 2024-11-06 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| WO2016054557A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Novel high efficiency library-identified aav vectors |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| EP3242945B1 (en) | 2015-01-07 | 2021-09-01 | Universitat Autònoma de Barcelona | Single-vector gene construct comprising insulin and glucokinase genes |
| EP3632923A1 (en) | 2015-01-16 | 2020-04-08 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
| IL310375B2 (en) | 2015-01-20 | 2025-05-01 | Genzyme Corp | Analytical ultracentrifugation for characterization of recombinant viral particles |
| WO2016126857A1 (en) * | 2015-02-03 | 2016-08-11 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| EA201791803A1 (ru) | 2015-02-10 | 2017-11-30 | Джензим Корпорейшн | Улучшенная доставка вирусных частиц в полосатое тело и кору больших полушарий |
| CA3255657A1 (en) | 2015-02-10 | 2025-12-01 | Genzyme Corporation | VARIANT RNAi |
| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
| FI3265178T3 (fi) | 2015-03-02 | 2026-08-03 | Adverum Biotechnologies Inc | Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin |
| MX2017011615A (es) | 2015-03-10 | 2018-04-11 | Univ Columbia | Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1. |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | アデノ随伴ウイルス変異体及びその使用方法 |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| EP3285788B1 (en) | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| JP6851319B2 (ja) | 2015-04-27 | 2021-03-31 | 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 | ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2016200543A2 (en) | 2015-05-13 | 2016-12-15 | The Trustees Of The University Of Pennsylvania | Aav-mediated expression of anti-inluenza antibodies and methods of use thereof |
| PL3298134T3 (pl) | 2015-05-16 | 2023-09-18 | Genzyme Corporation | Edycja genów mutacji głęboko intronowych |
| EP4378487A3 (en) | 2015-06-23 | 2024-09-04 | The Children's Hospital of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| ES3009026T3 (en) | 2015-07-22 | 2025-03-25 | Univ Duke | High-throughput screening of regulatory element function with epigenome editing technologies |
| WO2017024198A1 (en) | 2015-08-06 | 2017-02-09 | The Trustees Of The University Of Pennsylvania | Glp-1 and use thereof in compositions for treating metabolic diseases |
| KR20240132120A (ko) | 2015-08-25 | 2024-09-02 | 듀크 유니버시티 | Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법 |
| HK1257024A1 (zh) | 2015-08-31 | 2019-10-11 | 宾夕法尼亚大学 | 用於治疗伴侣动物的aav-epo |
| EP3359663A4 (en) | 2015-09-24 | 2019-08-14 | The Trustees of The University of Pennsylvania | COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE |
| IL314171A (en) * | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| WO2017062750A1 (en) | 2015-10-09 | 2017-04-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful in treating stargardt's disease and other ocular disorders |
| WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| EP3364997B1 (en) | 2015-10-22 | 2024-01-17 | University of Massachusetts | Aspartoacylase gene therapy in the treatment of canavan disease |
| WO2017070516A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| US20180230489A1 (en) | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| US11135313B2 (en) | 2015-10-28 | 2021-10-05 | The Trustees Of The University Of Pennsylvania | Intrathecal administration of adeno-associated-viral vectors for gene therapy |
| CA3001941A1 (en) | 2015-10-30 | 2017-05-04 | Nbe-Therapeutics Ag | Anti-ror1 antibodies |
| AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| US11015174B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
| EP4085934A1 (en) | 2015-12-11 | 2022-11-09 | The Trustees of The University of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| EP3992283A1 (en) | 2015-12-11 | 2022-05-04 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| EP3387138B2 (en) | 2015-12-11 | 2026-05-27 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| AU2016370590B2 (en) | 2015-12-14 | 2023-11-02 | The Trustees Of The University Of Pennsylvania | Composition for treatment of Crigler-Najjar syndrome |
| CN109069668B (zh) | 2015-12-14 | 2023-04-18 | 宾夕法尼亚州大学信托人 | 用于眼病的基因疗法 |
| SG10201912935WA (en) | 2015-12-15 | 2020-02-27 | Genzyme Corp | Adeno-associated viral vectors for treating mucolipidosis type ii |
| KR102777513B1 (ko) | 2015-12-22 | 2025-03-06 | 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 | (r)-1-(4-(6-(2-(4-(3,3-디플루오로시클로부톡시)-6-메틸피리딘-2-일)아세트아미도)피리다진-3-일)-2-플루오로부틸)-n-메틸-1h-1,2,3-트리아졸-4-카르복사미드의 염 형태 및 다형체 |
| MX2018008926A (es) | 2016-01-20 | 2019-01-10 | Scripps Research Inst | Composiciones de anticuerpo contra ror1 y métodos relacionados. |
| KR20180118659A (ko) | 2016-02-01 | 2018-10-31 | 바이오버라티브 테라퓨틱스 인크. | 최적화된 viii 인자 유전자 |
| CA3011939A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| CA3012344A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| US10729789B2 (en) * | 2016-03-01 | 2020-08-04 | University Of Virginia Patent Foundation | Compositions and methods for adeno-associated virus mediated gene expression in myofibroblast-like cells |
| EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN |
| WO2017180587A2 (en) | 2016-04-11 | 2017-10-19 | Obsidian Therapeutics, Inc. | Regulated biocircuit systems |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| US11191847B2 (en) | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| PE20181922A1 (es) | 2016-04-15 | 2018-12-11 | Univ Pennsylvania | Terapia genica para tratar la hemofilia a |
| CN109661470A (zh) * | 2016-04-15 | 2019-04-19 | 宾夕法尼亚州大学信托人 | 新型aav8突变衣壳和含有其的组合物 |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| IL262207B2 (en) | 2016-04-15 | 2026-01-01 | Univ Pennsylvania | Preparations for the treatment of wet age-related macular degeneration |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| WO2017184463A1 (en) | 2016-04-17 | 2017-10-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for prophylaxis of organophosphates |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| GB201608046D0 (en) * | 2016-05-09 | 2016-06-22 | Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The | Treatment of complement-mediated disorders |
| WO2017197355A2 (en) | 2016-05-13 | 2017-11-16 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| US10584337B2 (en) | 2016-05-18 | 2020-03-10 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| US11158076B2 (en) | 2016-06-08 | 2021-10-26 | Sony Corporation | Imaging control device and method, and vehicle |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| US20190240353A1 (en) | 2016-07-05 | 2019-08-08 | University Of Massachusetts | Aav2-mediated gene delivery of sfasl as a neuroprotective therapy in glaucoma |
| US11197936B2 (en) | 2016-07-08 | 2021-12-14 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving RDH12 |
| JP7490211B2 (ja) | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Cpf1に基づくゲノム編集の治療適用 |
| WO2018022511A1 (en) | 2016-07-25 | 2018-02-01 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| 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 |
| CN109640949B (zh) | 2016-07-29 | 2022-11-25 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体和其使用方法 |
| HUE053747T2 (hu) | 2016-08-15 | 2021-07-28 | Genzyme Corp | Eljárások AAV kimutatására |
| 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 |
| EP3831281B1 (en) | 2016-08-30 | 2026-02-25 | The Regents of The University of California | Devices and systems for biomedical targeting and delivery |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| US11078238B2 (en) * | 2016-09-29 | 2021-08-03 | University Of Florida Research Foundation, Incorporated | AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties |
| KR20190075964A (ko) | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 설계 |
| AU2017345470B2 (en) | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| ES2931960T3 (es) | 2016-12-01 | 2023-01-05 | Inst Nat Sante Rech Med | Composiciones farmacéuticas para el tratamiento de enfermedades degenerativas retinianas |
| WO2018112225A1 (en) * | 2016-12-14 | 2018-06-21 | The J. David Gladstone Institutes | Methods and compositions for generating a deletion library and for identifying a defective interfering particle (dip) |
| EP3562494A4 (en) | 2016-12-30 | 2020-08-19 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS |
| JP2020505317A (ja) | 2017-01-20 | 2020-02-20 | ユニバーシティ オブ ピッツバーグ − オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | 臍帯血移植(ucbt)および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置 |
| JP2020508289A (ja) | 2017-01-31 | 2020-03-19 | レジェンクスバイオ インコーポレーテッド | 完全ヒトグリコシル化ヒトα−L−イズロニダーゼ(IDUA)を用いたムコ多糖症I型の治療 |
| JP2020510648A (ja) | 2017-02-20 | 2020-04-09 | 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 | 家族性高コレステロール血症を処置するための遺伝子治療 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| TW201837170A (zh) | 2017-02-28 | 2018-10-16 | 賓州大學委員會 | 新穎aav媒介的流感疫苗 |
| RS65241B1 (sr) | 2017-02-28 | 2024-03-29 | Univ Pennsylvania | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
| WO2018160849A1 (en) | 2017-03-01 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| EP3589311A1 (en) | 2017-03-02 | 2020-01-08 | Genethon | Method for removing anti-aav antibodies from a blood-derived composition |
| CN110913954A (zh) | 2017-03-03 | 2020-03-24 | 黑曜石疗法公司 | 免疫疗法的组合物和方法 |
| AU2018227583B2 (en) | 2017-03-03 | 2023-06-01 | Obsidian Therapeutics, Inc. | CD19 compositions and methods for immunotherapy |
| EP3606544B1 (en) | 2017-04-05 | 2025-06-04 | University of Massachusetts | Minigene therapy |
| EP3610016A1 (en) | 2017-04-10 | 2020-02-19 | Genethon | Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies |
| KR20240093919A (ko) | 2017-04-14 | 2024-06-24 | 리젠엑스바이오 인크. | 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료 |
| US11879133B2 (en) | 2017-04-24 | 2024-01-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
| BR112019023303A2 (pt) | 2017-05-11 | 2020-06-16 | The Trustees Of The University Of Pennsylvania | Terapia de gene para lipofuscinoses ceroides neuronais |
| CA3099990A1 (en) | 2017-05-12 | 2018-11-15 | University Of Massachusetts | Viral vector production |
| CN108103058A (zh) * | 2017-05-12 | 2018-06-01 | 北京五加和分子医学研究所有限公司 | 一种i型糖尿病的基因治疗药物 |
| KR102765034B1 (ko) | 2017-05-24 | 2025-02-11 | 유니버시타트 아우토노마 데 바르셀로나 | 섬유아세포 성장 인자 21 (fgf21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트 |
| US11793887B2 (en) | 2017-05-31 | 2023-10-24 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating peroxisomal disorders |
| EP3635122A4 (en) | 2017-06-06 | 2021-03-31 | University of Massachusetts | SELF-REGULATING AAV VECTORS FOR THE SAFE EXPRESSION OF MECP2 IN RETT SYNDROME |
| US11827898B2 (en) | 2017-06-14 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| AU2018288863A1 (en) * | 2017-06-22 | 2020-01-30 | Board Of Regents, The University Of Texas System | Methods for producing regulatory immune cells and uses thereof |
| KR20250023600A (ko) | 2017-06-30 | 2025-02-18 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법 |
| CA3068328A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| PL3648783T3 (pl) | 2017-07-07 | 2024-11-18 | Genethon | Nowe polinukleotydy kodujące ludzkie białko fkrp |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| US11512327B2 (en) | 2017-08-03 | 2022-11-29 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
| KR20230008269A (ko) | 2017-08-07 | 2023-01-13 | 엔비이-테라퓨틱스 아게 | 생체 내 내성이 높은 항체 약물 결합체 |
| MX2020001997A (es) * | 2017-08-28 | 2020-07-20 | Univ California | Variantes de capside de virus adenoasociado y metodos de uso de estas. |
| DK3684423T5 (da) | 2017-09-20 | 2024-08-26 | 4D Molecular Therapeutics Inc | Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf |
| MX2020003095A (es) | 2017-09-22 | 2020-10-15 | Genzyme Corp | Arni variante. |
| CN118006607A (zh) | 2017-09-22 | 2024-05-10 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| MX2020004005A (es) * | 2017-10-03 | 2020-10-05 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
| US12441998B2 (en) | 2017-10-12 | 2025-10-14 | The Trustees Of Columbia University In The City Of New York | SLC2A1 lncRNA as a biologic and related treatments and methods |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| KR20200067195A (ko) | 2017-10-18 | 2020-06-11 | 리젠엑스바이오 인크. | 완전-인간 번역후 변형된 항체 치료제 |
| US10722487B2 (en) | 2017-10-18 | 2020-07-28 | Board Of Regents, The University Of Texas System | Glutaminase inhibitor therapy |
| MX2020003945A (es) | 2017-10-18 | 2020-11-09 | Regenxbio Inc | Tratamiento de enfermedades oculares y cancer de colon metastasico con vegf-trap humano modificado post-traduccionalmente . |
| US11766489B2 (en) | 2017-11-27 | 2023-09-26 | 4D Molecular Therapeutics, Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
| EA202091352A1 (ru) | 2017-11-30 | 2020-08-27 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Генотерапия мукополисахаридоза iiib |
| US11555206B2 (en) | 2017-11-30 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis IIIA |
| CA3086045A1 (en) | 2017-12-19 | 2019-06-27 | Akouos, Inc. | Aav-mediated delivery of therapeutic antibodies to the inner ear |
| AU2019215152A1 (en) | 2018-02-01 | 2020-08-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof |
| WO2019152841A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for pah gene transfer and methods of use thereof |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| EP3768695A4 (en) | 2018-02-27 | 2022-04-06 | The Trustees of the University of Pennsylvania | NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
| EP3762500A1 (en) | 2018-03-06 | 2021-01-13 | Voyager Therapeutics, Inc. | Insect cell manufactured partial self-complementary aav genomes |
| EP4169576A1 (en) | 2018-03-23 | 2023-04-26 | University of Massachusetts | Gene therapeutics for treating bone disorders |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| WO2019200016A1 (en) | 2018-04-10 | 2019-10-17 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
| EP3781677A4 (en) | 2018-04-16 | 2022-01-19 | University of Massachusetts | COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING |
| CA3099958A1 (en) | 2018-04-16 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating duchenne muscular dystrophy |
| CA3098448A1 (en) | 2018-04-27 | 2019-10-31 | University Of Massachusetts | Aav capsids identified by in vivo library selection |
| IL278213B2 (en) | 2018-04-27 | 2026-04-01 | Rocket Pharmaceuticals Ltd | A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd) |
| CA3098565A1 (en) | 2018-04-29 | 2019-11-07 | Claire G. ZHANG | Scalable clarification process for recombinant aav production |
| CA3098566A1 (en) | 2018-04-29 | 2019-11-07 | Zhuchun WU | Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles |
| KR102877876B1 (ko) | 2018-05-08 | 2025-10-28 | 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 | Aav-상용성 라미닌-링커 중합 단백질 |
| 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 |
| 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 |
| WO2019222329A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| US20210207167A1 (en) | 2018-05-16 | 2021-07-08 | Voyager Therapeutics, Inc. | Aav serotypes for brain specific payload delivery |
| WO2019222444A2 (en) | 2018-05-16 | 2019-11-21 | Voyager Therapeutics, Inc. | Directed evolution |
| WO2019236995A1 (en) | 2018-06-08 | 2019-12-12 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| US20220403001A1 (en) | 2018-06-12 | 2022-12-22 | Obsidian Therapeutics, Inc. | Pde5 derived regulatory constructs and methods of use in immunotherapy |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| IL319002A (en) | 2018-06-14 | 2025-04-01 | Regenxbio Inc | Anion exchange chromatography for recombinant AAV production |
| US20210254103A1 (en) | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| GB201811368D0 (en) | 2018-07-11 | 2018-08-29 | Ucb Biopharma Sprl | Antibody |
| EP3820537A4 (en) | 2018-07-12 | 2022-04-13 | Spacecraft Seven, LLC | GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE |
| CA3106078A1 (en) | 2018-07-17 | 2020-01-23 | Junghun Lee | Treatment of neuropathy with dna constructs expressing igf-1 isoforms |
| WO2020023612A1 (en) | 2018-07-24 | 2020-01-30 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| JPWO2020026968A1 (ja) * | 2018-07-30 | 2021-08-12 | 株式会社遺伝子治療研究所 | Aavベクターによる遺伝子発現を増強する方法 |
| TW202509215A (zh) | 2018-08-03 | 2025-03-01 | 美商健臻公司 | 針對α-突觸核蛋白的變體RNAi |
| WO2020033842A1 (en) | 2018-08-10 | 2020-02-13 | Regenxbio Inc. | Scalable method for recombinant aav production |
| KR20210102870A (ko) | 2018-08-30 | 2021-08-20 | 테나야 테라퓨틱스, 인코포레이티드 | 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍 |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020069339A1 (en) | 2018-09-28 | 2020-04-02 | President And Fellows Of Harvard College | Mutant reverse tetracycline transactivators for expression of genes |
| US12274733B2 (en) | 2018-09-28 | 2025-04-15 | President And Fellows Of Harvard College | Cellular reprogramming to reverse aging and promote organ and tissue regeneration |
| WO2020069373A1 (en) | 2018-09-28 | 2020-04-02 | President And Fellows Of Harvard College | Cellular reprogramming to reverse aging and promote organ and tissue regeneration |
| EP3860659A1 (en) | 2018-10-01 | 2021-08-11 | The Trustees of the University of Pennsylvania | Compositions useful for treating gm1 gangliosidosis |
| AU2019354296A1 (en) | 2018-10-01 | 2021-04-29 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| WO2020072849A1 (en) | 2018-10-04 | 2020-04-09 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| AU2019354793A1 (en) | 2018-10-05 | 2021-05-13 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding AAV production proteins |
| CN120349985A (zh) | 2018-10-12 | 2025-07-22 | 建新公司 | 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生 |
| US20210371470A1 (en) | 2018-10-12 | 2021-12-02 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| WO2020081490A1 (en) | 2018-10-15 | 2020-04-23 | Voyager Therapeutics, Inc. | EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM |
| JP2022514112A (ja) | 2018-10-15 | 2022-02-09 | リジェネクスバイオ インコーポレイテッド | 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法 |
| UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
| US20210363509A1 (en) | 2018-10-22 | 2021-11-25 | University Of Rochester | Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas9 Fusion Protein |
| US20210386788A1 (en) | 2018-10-24 | 2021-12-16 | Obsidian Therapeutics, Inc. | Er tunable protein regulation |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| MX2021005720A (es) | 2018-11-16 | 2021-07-21 | Astellas Pharma Inc | Metodo para tratar la distrofia muscular mediante el direccionamiento del gen de utrofina. |
| US12391947B2 (en) | 2018-11-29 | 2025-08-19 | University Of Massachusetts | Modulation of SPTLC1 via recombinant adeno-associated vectors |
| WO2020140007A1 (en) | 2018-12-28 | 2020-07-02 | University Of Rochester | Gene therapy for best1 dominant mutations |
| KR20210112339A (ko) | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
| CN113544267B (zh) | 2019-01-14 | 2025-04-18 | 罗切斯特大学 | 使用CRISPR-Cas进行靶向核RNA裂解和聚腺苷酸化 |
| CA3125770A1 (en) | 2019-01-18 | 2020-07-23 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
| 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 |
| MX2021010134A (es) | 2019-02-22 | 2021-09-23 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn. |
| CA3131023A1 (en) | 2019-02-22 | 2020-08-27 | Michael R. Volkert | Oxr1 gene therapy |
| US20220154210A1 (en) | 2019-02-25 | 2022-05-19 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
| CN113710693B (zh) | 2019-02-25 | 2025-07-15 | 马萨诸塞大学 | Dna结合结构域反式激活因子及其用途 |
| EP3931335A2 (en) | 2019-02-25 | 2022-01-05 | Novartis AG | Compositions and methods to treat bietti crystalline dystrophy |
| EP3931337A1 (en) | 2019-02-26 | 2022-01-05 | The Trustees of The University of Pennsylvania | Compositions useful in treatment of krabbe disease |
| EP3930694A1 (en) | 2019-02-28 | 2022-01-05 | Regeneron Pharmaceuticals, Inc. | Adeno-associated virus vectors for the delivery of therapeutics |
| AU2020232692A1 (en) | 2019-03-04 | 2021-09-30 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the AKT pathway |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| EP3947427A1 (en) | 2019-03-25 | 2022-02-09 | Genethon | Production of large-sized quasidystrophins using overlapping aav vectors |
| CN114450411A (zh) | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
| EP4667578A3 (en) | 2019-04-03 | 2026-04-01 | REGENXBIO Inc. | Gene therapy for eye pathologies |
| TW202102526A (zh) | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
| ES2965341T3 (es) | 2019-04-11 | 2024-04-12 | Regenxbio Inc | Métodos de cromatografía de exclusión por tamaño para la caracterización de composiciones de virus adenoasociados recombinantes |
| US12553036B2 (en) | 2019-04-12 | 2026-02-17 | University Of Massachusetts | GM3 synthase vectors and uses thereof |
| TWI887234B (zh) | 2019-04-14 | 2025-06-21 | 美國公爵大學 | 治療杜興氏(duchenne)肌肉失養症之大型突變熱點之aav載體介導之刪除 |
| WO2020215010A1 (en) | 2019-04-17 | 2020-10-22 | Codiak Biosciences, Inc. | Compositions of exosomes and aav |
| US12516097B2 (en) | 2019-04-19 | 2026-01-06 | University Of Massachusetts | Gene therapies for Stargardt disease (ABCA4) |
| JP7630443B2 (ja) | 2019-04-19 | 2025-02-17 | レジェンクスバイオ インコーポレーテッド | アデノ随伴ウイルスベクター製剤及び方法 |
| US20220195462A1 (en) * | 2019-04-24 | 2022-06-23 | Regenxbio Inc. | Fully-human post-translationally modified antibody therapeutics |
| EP3959324B1 (en) | 2019-04-26 | 2025-09-17 | President and Fellows of Harvard College | Aav vectors encoding mini-pcdh15 and uses thereof |
| WO2020223232A1 (en) * | 2019-04-29 | 2020-11-05 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| US20220243225A1 (en) | 2019-04-29 | 2022-08-04 | Voyager Therapeutics, Inc. | SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS |
| JP7715640B2 (ja) | 2019-05-03 | 2025-07-30 | 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 | 異染性白質ジストロフィーの治療に有用な組成物 |
| EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| US12390539B2 (en) | 2019-05-20 | 2025-08-19 | University Of Massachusetts | Minigene therapy |
| AU2020283537A1 (en) * | 2019-05-24 | 2021-12-16 | Regeneron Pharmaceuticals, Inc. | Modified viral particles and uses thereof |
| JP7565620B2 (ja) | 2019-05-28 | 2024-10-11 | 株式会社モダリス | Dmpk遺伝子を標的とした筋ジストロフィーの治療方法 |
| WO2020257684A1 (en) | 2019-06-20 | 2020-12-24 | University Of Massachusetts | Compositions and methods for improved gene editing |
| BR112021026866A2 (pt) | 2019-07-02 | 2022-03-03 | M6P Therapeutics | Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico |
| ES2948620T3 (es) | 2019-07-10 | 2023-09-14 | Masonic Medical Res Laboratory | Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo |
| US20220251567A1 (en) | 2019-07-10 | 2022-08-11 | Inserm (Institut National De La Santè Et De La Recherche Médicale) | Methods for the treatment of epilepsy |
| EP3997214B1 (en) | 2019-07-11 | 2023-08-02 | Centre National de la Recherche Scientifique | Chemically-modified adeno-associated virus |
| EP3997226A1 (en) | 2019-07-11 | 2022-05-18 | Tenaya Therapeutics, Inc. | Cardiac cell reprogramming with micrornas and other factors |
| US10653731B1 (en) * | 2019-07-15 | 2020-05-19 | Vigene Biosciences Inc. | Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency |
| US10557149B1 (en) * | 2019-07-15 | 2020-02-11 | Vigene Biosciences, Inc. | Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus |
| WO2021016453A1 (en) | 2019-07-23 | 2021-01-28 | University Of Rochester | Targeted rna cleavage with crispr-cas |
| EP4004214A1 (en) | 2019-07-26 | 2022-06-01 | RegenxBio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
| EP4007814A1 (en) | 2019-07-26 | 2022-06-08 | Akouos, Inc. | Methods of treating hearing loss using a secreted target protein |
| EP4010465A1 (en) | 2019-08-09 | 2022-06-15 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| JP7785352B2 (ja) * | 2019-08-14 | 2025-12-15 | ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. | 遺伝子治療のためのaavカプシドバリアント |
| CA3149401A1 (en) | 2019-08-26 | 2021-03-04 | Stephen Joseph Pakola | Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab |
| EP4022070A1 (en) | 2019-08-26 | 2022-07-06 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| EP4022057A1 (en) | 2019-08-27 | 2022-07-06 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of disorders associated with repetitive dna |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| WO2021046451A1 (en) | 2019-09-06 | 2021-03-11 | Obsidian Therapeutics, Inc. | Compositions and methods for dhfr tunable protein regulation |
| CN114555814A (zh) | 2019-09-13 | 2022-05-27 | 罗特格斯新泽西州立大学 | Aav相容的层粘连蛋白-连接子聚合蛋白 |
| EP4031673A1 (en) | 2019-09-19 | 2022-07-27 | Genethon | Gene therapy expression system alleviating cardiac toxicity of fkrp |
| US11834659B2 (en) | 2019-09-26 | 2023-12-05 | Massachusetts Institute Of Technology | Trans-activated functional RNA by strand displacement and uses thereof |
| WO2021062096A1 (en) | 2019-09-26 | 2021-04-01 | Massachusetts Institute Of Technology | Microrna-based logic gates and uses thereof |
| JP7803014B2 (ja) | 2019-09-30 | 2026-01-21 | 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 | レンチウイルスベクター製剤 |
| WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| WO2021071835A1 (en) | 2019-10-07 | 2021-04-15 | Regenxbio Inc. | Adeno-associated virus vector pharmaceutical composition and methods |
| WO2021076656A1 (en) | 2019-10-15 | 2021-04-22 | University Of Massachusetts | Rna editor-enhanced rna trans-splicing |
| WO2021073568A1 (en) * | 2019-10-16 | 2021-04-22 | Wuxi Apptec (Shanghai) Co., Ltd. | A novel aav variant |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| MX2022004353A (es) | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| US20230340078A1 (en) | 2019-11-14 | 2023-10-26 | Biomarin Pharmaceutical Inc. | Treatment of hereditary angioedema with liver-specific gene therapy vectors |
| WO2021099394A1 (en) | 2019-11-19 | 2021-05-27 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Antisense oligonucleotides and their use for the treatment of cancer |
| AU2020392243A1 (en) * | 2019-11-28 | 2022-06-16 | Regenxbio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| KR20220112283A (ko) | 2019-12-10 | 2022-08-10 | 다케다 야쿠힌 고교 가부시키가이샤 | Hunter 질환 치료용 아데노-연합된 바이러스 벡터 |
| BR112022011916A2 (pt) | 2019-12-19 | 2022-09-06 | Univ Pennsylvania | Composições para tratar a ataxia de friedreich |
| CN116096904A (zh) | 2019-12-31 | 2023-05-09 | 天鹅生物疗法有限公司 | 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途 |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| KR20220131522A (ko) | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
| US20230088992A1 (en) | 2020-01-29 | 2023-03-23 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells |
| BR112022015036A2 (pt) | 2020-01-29 | 2022-10-11 | Regenxbio Inc | Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| WO2021154839A1 (en) | 2020-01-30 | 2021-08-05 | Umoja Biopharma, Inc. | Bispecific transduction enhancer |
| WO2021155337A1 (en) | 2020-02-02 | 2021-08-05 | The Trustees Of The University Of Pennsylvania | Compositions useful for treating gm1 gangliosidosis |
| EP4100533A1 (en) | 2020-02-07 | 2022-12-14 | University of Rochester | Ribozyme-mediated rna assembly and expression |
| US20230078498A1 (en) | 2020-02-07 | 2023-03-16 | University Of Rochester | Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis |
| AU2021218703A1 (en) | 2020-02-14 | 2022-07-28 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating CDKL5 deficiency disorder |
| US20230089312A1 (en) | 2020-02-25 | 2023-03-23 | University Of Massachusetts | Inducible single aav system and uses thereof |
| TW202142554A (zh) * | 2020-02-25 | 2021-11-16 | 澳洲兒童醫學研究所 | 腺相關病毒蛋白殼多肽及載體 |
| CN115552022A (zh) | 2020-03-02 | 2022-12-30 | 特纳亚治疗股份有限公司 | 由心肌细胞表达的微rna控制的基因载体 |
| EP4127170A1 (en) | 2020-03-27 | 2023-02-08 | University of Rochester | Crispr-cas13 crrna arrays |
| JP2023519576A (ja) | 2020-03-27 | 2023-05-11 | ユーシービー バイオファルマ エスアールエル | 自律ノブドメインペプチド |
| JP2023519307A (ja) | 2020-03-27 | 2023-05-10 | ユニバーシティ オブ ロチェスター | Crispr-cas13によるウイルスrnaの標的化破壊 |
| US12686874B2 (en) | 2020-03-31 | 2026-07-21 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| BR112022019304A2 (pt) | 2020-03-31 | 2022-12-06 | Univ Massachusetts | Variantes de capsídeo de aav e usos das mesmas |
| TW202144575A (zh) | 2020-04-03 | 2021-12-01 | 美商拜奧馬林製藥公司 | 使用aav及治療調配物之苯酮尿症治療 |
| CN115349014A (zh) * | 2020-04-06 | 2022-11-15 | Dh科技发展私人贸易有限公司 | 病毒载体的基因组完整性分析 |
| JP2023520927A (ja) | 2020-04-10 | 2023-05-22 | ソラ・バイオサイエンシズ・エルエルシー | タンパク質凝集障害の処置のための組成物および方法 |
| CA3180222A1 (en) | 2020-04-15 | 2021-10-21 | Voyager Therapeutics, Inc. | Tau binding compounds |
| AU2021259422A1 (en) | 2020-04-20 | 2022-12-08 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| JP7894321B2 (ja) | 2020-04-28 | 2026-07-23 | ソラ・バイオサイエンシズ・エルエルシー | Tdp-43プロテイノパチーの処置のための組成物および方法 |
| IL298138A (en) | 2020-05-12 | 2023-01-01 | Univ Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| AU2021271635A1 (en) | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
| EP4165194A2 (en) | 2020-05-13 | 2023-04-19 | Akouos, Inc. | Compositions and methods for treating kcnq4-associated hearing loss |
| KR20230041965A (ko) | 2020-05-13 | 2023-03-27 | 아카우오스, 인크. | Slc26a4-연관 청력손실을 치료하기 위한 조성물 및 방법 |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復pah基因功能的腺相關病毒組成物及其使用方法 |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| US20230234997A1 (en) | 2020-06-05 | 2023-07-27 | Sola Biosciences Llc | Compositions and Methods for the Treatment of Synucleinopathies |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| CN115996758A (zh) | 2020-06-19 | 2023-04-21 | 吉尼松公司 | 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统 |
| JP2023537565A (ja) | 2020-06-24 | 2023-09-04 | 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 | タンパク質を発現するように改変されたレンチウイルスベクターの調製物から遊離第viii因子を除去する方法 |
| KR20230050336A (ko) | 2020-07-10 | 2023-04-14 | 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) | 뇌전증을 치료하기 위한 방법과 조성물 |
| BR112023000305A2 (pt) | 2020-07-10 | 2023-03-21 | Genethon | Promotor, sistema de expressão e composição farmacêutica |
| WO2022015715A1 (en) | 2020-07-13 | 2022-01-20 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| EP4182455A1 (en) | 2020-07-15 | 2023-05-24 | University of Rochester | Targeted rna cleavage with dcasl3-rnase fusion proteins |
| WO2022020548A2 (en) | 2020-07-23 | 2022-01-27 | President And Fellows Of Harvard College | Variant txnip compositions and methods of use thereof for the treatment of degenerative ocular diseases |
| WO2022017630A1 (en) | 2020-07-23 | 2022-01-27 | Ucl Business Ltd | GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF |
| US20230285596A1 (en) | 2020-07-27 | 2023-09-14 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
| AU2021321412A1 (en) | 2020-08-05 | 2023-04-06 | Spacecraft Seven, Llc | Adeno-associated viral vector for GLUT1 expression and uses thereof |
| EP4192514A1 (en) | 2020-08-06 | 2023-06-14 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| CN116209430A (zh) | 2020-08-07 | 2023-06-02 | 阿米库斯治疗学公司 | 囊泡靶向蛋白及其用途 |
| WO2022032226A1 (en) | 2020-08-07 | 2022-02-10 | Spacecraft Seven, Llc | Plakophilin-2 (pkp2) gene therapy using aav vector |
| CN114075610B (zh) * | 2020-08-11 | 2023-11-17 | 北京荷塘生华医疗科技有限公司 | 检测野生型腺相关病毒的通用引物及其应用 |
| WO2022036220A1 (en) | 2020-08-14 | 2022-02-17 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| JP2023542614A (ja) | 2020-08-19 | 2023-10-11 | サレプタ セラピューティクス, インコーポレイテッド | レット症候群の治療のためのアデノ随伴ウイルスベクター |
| GB202013194D0 (en) | 2020-08-24 | 2020-10-07 | Combigene Ab | Gene therapy for lipodystrophy |
| US20230372539A1 (en) | 2020-08-24 | 2023-11-23 | The Trustees Of The University Of Pennsylvania | Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases |
| AU2021332331A1 (en) | 2020-08-26 | 2023-03-09 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of GRN-associated adult-onset neurodegeneration |
| US20220096606A1 (en) | 2020-09-09 | 2022-03-31 | Vertex Pharmaceuticals Incorporated | Compositions and Methods for Treatment of Duchenne Muscular Dystrophy |
| AU2021339003A1 (en) | 2020-09-10 | 2023-02-09 | Ludwig-Maximilians-Universität München | Engineered AAV vectors |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| EP4214242A1 (en) | 2020-09-15 | 2023-07-26 | RegenxBio Inc. | Vectorized antibodies for anti-viral therapy |
| US20250326861A1 (en) | 2020-09-15 | 2025-10-23 | Regenxbio Inc. | Vectorized lanadelumab and administration thereof |
| US20230383278A1 (en) | 2020-09-18 | 2023-11-30 | The United States Of America,As Represented By The Secretary,Department Of Health And Human Services | Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency |
| EP4217376A4 (en) | 2020-09-24 | 2024-10-09 | University of Massachusetts | AAV VECTORS ENCODING NF1 AND USES THEREOF |
| KR20230079172A (ko) | 2020-10-01 | 2023-06-05 | 젠자임 코포레이션 | 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트 |
| EP4225381A1 (en) | 2020-10-07 | 2023-08-16 | RegenxBio Inc. | Formulations for suprachoroidal administration such as formulations with aggregate formation |
| TW202228646A (zh) | 2020-10-07 | 2022-08-01 | 美商銳進科斯生物股份有限公司 | 諸如凝膠調配物之用於脈絡膜上投與之調配物 |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| JP2023544803A (ja) | 2020-10-07 | 2023-10-25 | レジェンクスバイオ インコーポレーテッド | Cln2疾患の眼症状に対する遺伝子療法 |
| TW202228648A (zh) | 2020-10-07 | 2022-08-01 | 美商銳進科斯生物股份有限公司 | 諸如高黏度調配物之用於脈絡膜上投與之調配物 |
| EP4225777A2 (en) | 2020-10-07 | 2023-08-16 | RegenxBio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
| AU2021356684A1 (en) | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| JP7415042B2 (ja) | 2020-10-09 | 2024-01-16 | テナヤ セラピューティクス, インコーポレイテッド | プラコフィリン2遺伝子治療の方法および組成物 |
| US11781156B2 (en) | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
| WO2022082109A1 (en) | 2020-10-18 | 2022-04-21 | The Trustees Of The University Of Pennsylvania | Improved adeno-associated virus (aav) vector and uses therefor |
| WO2022094157A1 (en) | 2020-10-28 | 2022-05-05 | Regenxbio Inc. | Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof |
| AU2021371307A1 (en) | 2020-10-28 | 2023-06-01 | Regenxbio, Inc. | VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS |
| WO2022094078A1 (en) | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| KR20230098176A (ko) | 2020-10-29 | 2023-07-03 | 리젠엑스바이오 인크. | 안구 적응증을 위한 벡터화된 tnf-알파 길항제 |
| US20230390418A1 (en) | 2020-10-29 | 2023-12-07 | Regenxbio Inc. | Vectorized factor xii antibodies and administration thereof |
| WO2022098933A1 (en) | 2020-11-06 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of dm1 with slucas9 and sacas9 |
| CN112322791B (zh) * | 2020-11-27 | 2023-10-27 | 福建省农业科学院畜牧兽医研究所 | 一种新型鸭依赖属病毒环介导等温扩增检测引物组及试剂盒 |
| IL303317A (en) | 2020-12-01 | 2023-07-01 | Akouos Inc | ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA |
| AR124216A1 (es) | 2020-12-01 | 2023-03-01 | Univ Pennsylvania | Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen |
| WO2022119890A1 (en) | 2020-12-01 | 2022-06-09 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
| EP4263841A1 (en) | 2020-12-16 | 2023-10-25 | REGENXBIO Inc. | Method of producing a recombinant adeno-associated virus particle |
| CN117157108A (zh) | 2020-12-29 | 2023-12-01 | 阿库斯股份有限公司 | 用于治疗clrn1相关听力损失和/或视力损失的组合物和方法 |
| US20240309076A1 (en) | 2020-12-29 | 2024-09-19 | Regenxbio Inc. | Tau-specific antibody gene therapy compositions, methods and uses thereof |
| WO2022155500A1 (en) | 2021-01-14 | 2022-07-21 | Senti Biosciences, Inc. | Secretable payload regulation |
| AU2022210592A1 (en) | 2021-01-21 | 2023-07-13 | Regenxbio Inc. | Improved production of recombinant polypeptides and viruses |
| WO2022159722A1 (en) | 2021-01-22 | 2022-07-28 | University Of Massachusetts | Use of novel mirna-binding site cassettes for antigen-presenting cell detargeting of transgene expression by raav gene therapy vectors |
| WO2022164935A1 (en) | 2021-01-27 | 2022-08-04 | Umoja Biopharma, Inc. | Lentivirus for generating cells expressing anti-cd19 chimeric antigen receptor |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| AU2022221284A1 (en) | 2021-02-10 | 2023-08-17 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) |
| GB202101958D0 (en) | 2021-02-12 | 2021-03-31 | Ucl Business Ltd | Gene therapy for dopamine transporter deficiency syndrome |
| EP4298222A1 (en) | 2021-02-26 | 2024-01-03 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/sacas9 |
| AR124981A1 (es) | 2021-02-26 | 2023-05-24 | Takeda Pharmaceuticals Co | Composición y métodos para el tratamiento de la enfermedad de fabry |
| WO2022182959A1 (en) | 2021-02-26 | 2022-09-01 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/slucas9 |
| WO2022187548A1 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2022187473A2 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| CN117460832A (zh) | 2021-03-22 | 2024-01-26 | 建新公司 | 空aav衣壳和完整aav衣壳的尺寸排阻色谱分析 |
| WO2022204476A1 (en) | 2021-03-26 | 2022-09-29 | The Board Of Regents Of The University Of Texas System | Nucleotide editing to reframe dmd transcripts by base editing and prime editing |
| IL307633A (en) | 2021-04-12 | 2023-12-01 | Univ Pennsylvania | Useful preparations in the treatment of spinal muscular atrophy (SBMA) |
| WO2022226296A2 (en) | 2021-04-23 | 2022-10-27 | University Of Rochester | Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment |
| AU2022262771A1 (en) | 2021-04-23 | 2023-11-02 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| WO2022229851A1 (en) | 2021-04-26 | 2022-11-03 | Crispr Therapeutics Ag | Compositions and methods for using slucas9 scaffold sequences |
| EP4330409A4 (en) | 2021-04-26 | 2025-04-09 | University of Massachusetts | GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4) |
| IL307902A (en) | 2021-04-26 | 2023-12-01 | Regenxbio Inc | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
| EP4334454A2 (en) | 2021-05-04 | 2024-03-13 | RegenxBio Inc. | Novel aav vectors and methods and uses thereof |
| WO2022234519A1 (en) | 2021-05-05 | 2022-11-10 | Crispr Therapeutics Ag | Compositions and methods for using sacas9 scaffold sequences |
| US20240358857A1 (en) | 2021-05-11 | 2024-10-31 | Regenxbio Inc. | Treatment of duchenne muscular dystrophy and combinations thereof |
| US20230055381A1 (en) | 2021-06-25 | 2023-02-23 | Oxford Biomedica Solutions Llc | Adeno-associated virus packaging systems |
| KR20240051112A (ko) | 2021-07-01 | 2024-04-19 | 인답타 세라뷰틱스 인코포레이티드 | 조작된 자연 살해(nk) 세포 및 관련 방법 |
| US20240318157A1 (en) | 2021-07-01 | 2024-09-26 | Amicus Therapeutics, Inc. | Neurotensin Variants And Tagged Proteins Comprising Neurotensin Or Sortilin Propeptide |
| TW202317768A (zh) | 2021-07-08 | 2023-05-01 | 美商特納亞治療股份有限公司 | 用於基因療法的優化表現卡匣 |
| AU2022313258A1 (en) | 2021-07-19 | 2024-02-08 | New York University | Auf1 combination therapies for treatment of muscle degenerative disease |
| AU2022318664A1 (en) | 2021-07-30 | 2024-02-29 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| US20240254483A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
| WO2023018637A1 (en) | 2021-08-09 | 2023-02-16 | Vertex Pharmaceuticals Incorporated | Gene editing of regulatory elements |
| AU2022325231A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions |
| CA3227108A1 (en) | 2021-08-11 | 2023-02-16 | Xiaomeng HU | Genetically modified primary cells for allogeneic cell therapy |
| AU2022326565A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy |
| AU2022325955A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions |
| KR20240107201A (ko) | 2021-08-31 | 2024-07-08 | 스카우트 바이오, 인코포레이티드 | 항원-결합 분자 및 이의 용도 |
| WO2023039444A2 (en) | 2021-09-08 | 2023-03-16 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy |
| US20250288692A1 (en) | 2021-09-30 | 2025-09-18 | Akouos, Inc. | Compositions and methods for treating kcnq4-associated hearing loss |
| TW202332472A (zh) | 2021-10-01 | 2023-08-16 | 美商拜奧馬林製藥公司 | 利用aav基因療法載體進行之遺傳性血管水腫治療及治療性調配物 |
| US20240384298A1 (en) | 2021-10-02 | 2024-11-21 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| CN118202060A (zh) | 2021-10-05 | 2024-06-14 | 再生生物股份有限公司 | 用于重组aav生产的组合物和方法 |
| WO2023060113A1 (en) | 2021-10-05 | 2023-04-13 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| EP4413019A2 (en) | 2021-10-07 | 2024-08-14 | RegenxBio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
| EP4413018A1 (en) | 2021-10-07 | 2024-08-14 | RegenxBio Inc. | Recombinant adeno-associated viruses for targeted delivery |
| WO2023060221A2 (en) | 2021-10-08 | 2023-04-13 | Sola Biosciences Llc | Compositions and methods for the treatment of proteopathies |
| WO2023060248A1 (en) | 2021-10-08 | 2023-04-13 | Sola Biosciences Llc | Compositions and methods for the treatment of p53-mediated cancers |
| JP2024540987A (ja) | 2021-10-21 | 2024-11-06 | バーテックス ファーマシューティカルズ インコーポレイテッド | 低免疫細胞 |
| EP4423285A1 (en) | 2021-10-28 | 2024-09-04 | RegenxBio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP4437113A4 (en) | 2021-11-23 | 2026-04-08 | Univ Massachusetts | GENE THERAPY FOR SPINAL MUSCULAR ATROPHY |
| KR20240126860A (ko) | 2021-11-30 | 2024-08-21 | 재규어 진 테라피, 엘엘씨 | 당뇨병을 치료하기 위한 유전자 요법 방법 |
| US20240408157A1 (en) | 2021-12-02 | 2024-12-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| CA3239578A1 (en) | 2021-12-17 | 2023-06-22 | Stephane GAILLARD | Peptides and methods for use in treating pain |
| WO2023125481A1 (zh) | 2021-12-28 | 2023-07-06 | 成都弘基生物科技有限公司 | 经过修饰的aav衣壳蛋白及其用途 |
| US20250177495A1 (en) | 2022-01-10 | 2025-06-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| US20250082777A1 (en) | 2022-01-10 | 2025-03-13 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
| TW202340467A (zh) | 2022-01-10 | 2023-10-16 | 賓州大學委員會 | 有用於治療c9orf72介導之病症之組成物及方法 |
| US20250084129A1 (en) * | 2022-01-10 | 2025-03-13 | University Of Florida Research Foundation, Incorporated | Aav5 capsid variants |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| CN119317718A (zh) | 2022-01-25 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于改善心脏转导和肝脏去靶向的aav衣壳 |
| AR128422A1 (es) | 2022-02-02 | 2024-05-08 | Akouos Inc | Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular |
| WO2023150647A1 (en) | 2022-02-02 | 2023-08-10 | Sana Biotechnology, Inc. | Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses |
| EP4472657A4 (en) | 2022-02-04 | 2026-02-18 | M6P Therapeutics Inc | COMPOSITIONS AND METHODS OF USE OF A TWO-PROMOTER VECTOR FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS |
| JP2025507338A (ja) | 2022-02-08 | 2025-03-18 | ボイジャー セラピューティクス インコーポレイテッド | Aavカプシドバリアント及びその使用 |
| WO2023158836A1 (en) | 2022-02-17 | 2023-08-24 | Sana Biotechnology, Inc. | Engineered cd47 proteins and uses thereof |
| WO2023156530A1 (en) | 2022-02-17 | 2023-08-24 | Lysogene | Gene therapy for neurodegenerative diseases |
| TW202345911A (zh) | 2022-03-08 | 2023-12-01 | 美商維泰克斯製藥公司 | 用於治療杜興氏肌肉失養症(duchenne muscular dystrophy)之部分外顯子44、50及53之精確切除 |
| WO2023172926A1 (en) | 2022-03-08 | 2023-09-14 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exons for treatment of duchenne muscular dystrophy |
| WO2023173123A1 (en) | 2022-03-11 | 2023-09-14 | Sana Biotechnology, Inc. | Genetically modified cells and compositions and uses thereof |
| EP4493226A1 (en) | 2022-03-13 | 2025-01-22 | RegenxBio Inc. | Modified muscle-specific promoters |
| US20250213727A1 (en) | 2022-03-25 | 2025-07-03 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
| EP4504950A1 (en) | 2022-04-01 | 2025-02-12 | Takeda Pharmaceutical Company Limited | Gene therapy for diseases with cns manifestations |
| KR20240169111A (ko) | 2022-04-06 | 2024-12-02 | 젠자임 코포레이션 | Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법 |
| US20250250326A1 (en) | 2022-04-06 | 2025-08-07 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| AU2023250660A1 (en) | 2022-04-06 | 2024-10-24 | Regenxbio Inc. | Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration |
| CA3247109A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers |
| TW202404651A (zh) | 2022-04-06 | 2024-02-01 | 美商銳進科斯生物股份有限公司 | 用於脈絡膜上投與之調配物諸如形成聚集體之調配物 |
| TW202345913A (zh) | 2022-04-06 | 2023-12-01 | 美商銳進科斯生物股份有限公司 | 用於脈絡膜上投與之調配物諸如凝膠調配物 |
| WO2023196851A1 (en) | 2022-04-06 | 2023-10-12 | President And Fellows Of Harvard College | Reversing aging of the central nervous system |
| WO2023201207A1 (en) | 2022-04-11 | 2023-10-19 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| EP4508222A1 (en) | 2022-04-11 | 2025-02-19 | Centre National de la Recherche Scientifique | Chemically-modified adeno-associated viruses |
| EP4507719A1 (en) | 2022-04-13 | 2025-02-19 | Universitat Autònoma de Barcelona | Treatment of neuromuscular diseases via gene therapy that expresses klotho protein |
| WO2023201277A1 (en) | 2022-04-14 | 2023-10-19 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
| US20250249127A1 (en) | 2022-04-14 | 2025-08-07 | Regenxbio Inc. | Gene therapy for treating an ocular disease |
| CA3249261A1 (en) | 2022-04-18 | 2023-10-26 | Vertex Pharmaceuticals Incorporated | COMPOSITIONS AND PROCESSES TO IMPROVE ADENE-ASSOCIATED VIRUS (AAV) THERAPY AND REDUCE AAV TROPISM TO THE LIVER |
| KR20230152503A (ko) * | 2022-04-27 | 2023-11-03 | 주식회사 헬릭스미스 | 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터 |
| TW202417633A (zh) | 2022-05-03 | 2024-05-01 | 美商銳進科斯生物股份有限公司 | 用於眼適應症之載體化抗TNF-α抑制劑 |
| TW202400803A (zh) | 2022-05-03 | 2024-01-01 | 美商銳進科斯生物股份有限公司 | 載體化抗補體抗體與補體劑及其投與 |
| CA3255627A1 (en) | 2022-05-06 | 2023-11-09 | Novartis Ag | NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV |
| AR129441A1 (es) | 2022-05-25 | 2024-08-28 | Tafalgie Therapeutics | Péptidos y métodos para el tratamiento del dolor |
| WO2023239627A2 (en) | 2022-06-08 | 2023-12-14 | Regenxbio Inc. | Methods for recombinant aav production |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| CA3260237A1 (en) | 2022-06-24 | 2023-12-28 | Tune Therapeutics, Inc. | Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression |
| TW202409289A (zh) * | 2022-06-27 | 2024-03-01 | 美商安斯泰來基因治療股份有限公司 | 用於治療肌強直性營養不良之組合物及方法 |
| WO2024006741A1 (en) | 2022-06-28 | 2024-01-04 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| US20250375524A1 (en) | 2022-06-30 | 2025-12-11 | Indapta Therapeutics, Inc. | Combination of engineered natural killer (nk) cells and antibody therapy and related methods |
| WO2024011112A1 (en) | 2022-07-06 | 2024-01-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| EP4554967A2 (en) | 2022-07-12 | 2025-05-21 | Tune Therapeutics, Inc. | Compositions, systems, and methods for targeted transcriptional activation |
| WO2024020352A1 (en) | 2022-07-18 | 2024-01-25 | Vertex Pharmaceuticals Incorporated | Tandem guide rnas (tg-rnas) and their use in genome editing |
| WO2024017990A1 (en) | 2022-07-21 | 2024-01-25 | Institut National de la Santé et de la Recherche Médicale | Methods and compositions for treating chronic pain disorders |
| US20260027145A1 (en) | 2022-07-27 | 2026-01-29 | Sana Biotechnology, Inc. | Methods of transduction using a viral vector and inhibitors of antiviral restriction factors |
| CN115354049A (zh) * | 2022-07-29 | 2022-11-18 | 中国科学院深圳先进技术研究院 | 一种基因递送系统在将目的基因经静脉注射递送至肝脏的应用 |
| WO2024044725A2 (en) | 2022-08-24 | 2024-02-29 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| KR20250052387A (ko) | 2022-08-25 | 2025-04-18 | 다케다 야쿠힌 고교 가부시키가이샤 | 파브리병의 치료에서의 사용을 위한 조성물 |
| WO2024054983A1 (en) | 2022-09-08 | 2024-03-14 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| UY40442A (es) | 2022-09-22 | 2024-02-15 | Biomarin Pharm Inc | Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav |
| UY40443A (es) | 2022-09-22 | 2024-02-15 | Biomarin Pharm Inc | Tratamiento de la miocardiopatía con vectores de genoterapia con aav |
| EP4593955A1 (en) | 2022-09-30 | 2025-08-06 | RegenxBio Inc. | Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab |
| JP2025534666A (ja) | 2022-10-11 | 2025-10-17 | リジェネックスバイオ インコーポレイテッド | 操作された核酸調節エレメントならびにその使用方法 |
| WO2024105638A1 (en) | 2022-11-18 | 2024-05-23 | Jcr Pharmaceuticals Co., Ltd. | Recombinant aav vectors and methods for treatment of hunter syndrome |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| IT202200026595A1 (it) | 2022-12-22 | 2024-06-22 | Fond Telethon Ets | Nuovi inibitori di regolatori epigenetici |
| US12252518B2 (en) | 2023-01-06 | 2025-03-18 | Life Biosciences, Inc. | Methods of treating non-arteritic anterior ischemic optic neuropathy |
| WO2024146935A1 (en) | 2023-01-06 | 2024-07-11 | Institut National de la Santé et de la Recherche Médicale | Intravenous administration of antisense oligonucleotides for the treatment of pain |
| WO2024151541A1 (en) | 2023-01-09 | 2024-07-18 | Sana Biotechnology, Inc. | Type-1 diabetes autoimmune mouse |
| WO2024149844A1 (en) | 2023-01-12 | 2024-07-18 | Nantes Université | Chemically-modified adeno-associated virus |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| WO2024163683A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist) |
| AU2023427408A1 (en) | 2023-02-02 | 2025-09-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| AR131771A1 (es) | 2023-02-03 | 2025-04-30 | Janssen Pharmaceutica Nv | Vectores de terapia génica para su uso en la enfermedad de parkinson |
| GB202302480D0 (en) | 2023-02-22 | 2023-04-05 | Drishti Discoveries Ltd | shRNA for the treatment of disease |
| CN116286988A (zh) * | 2023-03-03 | 2023-06-23 | 西咸新区沣厚原创医药科技有限公司 | 一种稳定表达冠状病毒3cl蛋白酶的腺相关病毒体系、动物模型及构建方法和应用 |
| WO2024192281A2 (en) | 2023-03-15 | 2024-09-19 | Regenxbio Inc. | Exon skipping gene therapy constructs, vectors and uses thereof |
| CN120882872A (zh) | 2023-03-17 | 2025-10-31 | 罗切斯特大学 | 核酶介导的rna组装和表达 |
| JP2026513932A (ja) | 2023-04-07 | 2026-05-01 | リジェネクスバイオ インコーポレイテッド | 組換えaav生成のための組成物及び方法 |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| EP4695396A1 (en) | 2023-04-10 | 2026-02-18 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| EP4694904A2 (en) | 2023-04-13 | 2026-02-18 | REGENXBIO Inc. | Targeting aav capsids, methods of manufacturing and using same |
| CN116622908B (zh) * | 2023-04-13 | 2024-02-06 | 武汉珈创生物技术股份有限公司 | 快速检测野生型腺相关病毒的引物探针、试剂盒及方法和应用 |
| CN121311595A (zh) | 2023-04-17 | 2026-01-09 | 阿斯克生物公司 | 转基因在灵长类动物中可调节的受控表达 |
| CN121586778A (zh) | 2023-04-26 | 2026-02-27 | 赛诺菲 | 宿主细胞系以及用于鉴定和使用此类宿主细胞系的方法 |
| EP4705492A1 (en) * | 2023-05-01 | 2026-03-11 | St. Jude Children's Research Hospital, Inc. | Modified aav p5 promoter for improved vector titer and purity |
| CN121843705A (zh) | 2023-05-03 | 2026-04-10 | 萨那生物科技公司 | 工程化胰岛细胞的给药和施用方法 |
| AU2024266138A1 (en) | 2023-05-03 | 2025-11-13 | Manifold Biotechnologies, Inc. | Methods and compositions for high-throughput protein delivery, screening, and detection |
| EP4709866A2 (en) | 2023-05-07 | 2026-03-18 | REGENXBIO Inc. | Compositions and methods for recombinant aav production |
| KR20260010739A (ko) | 2023-05-11 | 2026-01-21 | 유니버시티 하스피탈즈 클리블랜드 메디컬 센터 | 항불안 치료 |
| WO2024233791A1 (en) | 2023-05-11 | 2024-11-14 | Seelos Therapeutics, Inc. | Methods of treating neurodegenerative disorders |
| WO2024238853A1 (en) | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
| WO2024238859A1 (en) | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Vectorized c5 inhibitor agents and administration thereof |
| WO2024238867A1 (en) | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Vectorized anti-complement antibodies and administration thereof |
| WO2024243236A2 (en) | 2023-05-22 | 2024-11-28 | Sana Biotechnology, Inc. | Methods of delivery of islet cells and related methods |
| WO2024241176A1 (en) | 2023-05-24 | 2024-11-28 | Jcr Pharmaceuticals Co., Ltd. | Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders |
| WO2024243563A1 (en) | 2023-05-25 | 2024-11-28 | Juno Therapeutics, Inc. | Aav purification method |
| JP2026522354A (ja) | 2023-06-12 | 2026-07-07 | 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 | ムコ多糖症iiibのためのaav遺伝子療法 |
| WO2025007046A1 (en) | 2023-06-29 | 2025-01-02 | The Trustees Of The University Of Pennsylvania | Mutant aav with central nervous system targeting motifs and compositions containing same |
| WO2025008406A1 (en) | 2023-07-04 | 2025-01-09 | Institut National de la Santé et de la Recherche Médicale | Antisense oligonucleotides and their use for the treatment of cancer |
| WO2025017168A1 (en) | 2023-07-19 | 2025-01-23 | Genethon | Novel optimized utrophin micro-genes |
| WO2025017169A1 (en) | 2023-07-20 | 2025-01-23 | Genethon | Novel mididystrophins |
| IL326390A (en) | 2023-08-10 | 2026-04-01 | Univ Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
| WO2025038430A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2025072604A1 (en) | 2023-09-28 | 2025-04-03 | University Of Rochester | Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1) |
| WO2025075963A1 (en) | 2023-10-02 | 2025-04-10 | Regenxbio Inc. | Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase |
| WO2025080780A1 (en) | 2023-10-10 | 2025-04-17 | University Of Rochester | Delivery and expression of prime editing crispr systems |
| WO2025090962A1 (en) | 2023-10-25 | 2025-05-01 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| WO2025090858A1 (en) | 2023-10-27 | 2025-05-01 | Biogen Ma Inc. | Methods for identifying aav capsid variants with desired characteristics |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025106374A1 (en) | 2023-11-13 | 2025-05-22 | Juno Therapeutics, Inc. | Aav production method |
| KR20260114566A (ko) | 2023-11-14 | 2026-07-22 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도 |
| AU2024386366A1 (en) | 2023-11-23 | 2026-06-04 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Gene therapy compositions and methods for treating glioma |
| WO2025113676A1 (en) | 2023-11-29 | 2025-06-05 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating stroke in primates |
| WO2025128817A1 (en) | 2023-12-15 | 2025-06-19 | Genzyme Corporation | Artificial micrornas targeting tau |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
| WO2025137219A1 (en) | 2023-12-21 | 2025-06-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025147436A1 (en) | 2024-01-03 | 2025-07-10 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2025160429A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| WO2025160434A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
| WO2025179007A1 (en) * | 2024-02-21 | 2025-08-28 | The Regents Of The University Of California | Adeno-associated virus compositions and methods of use thereof for human cells |
| TW202548022A (zh) | 2024-03-03 | 2025-12-16 | 美商帕西奇生物公司 | 用於治療神經退化性病症之重組腺相關病毒 |
| WO2025217230A1 (en) | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Vectorized anti-complement antibodies and complement agents and administration thereof |
| WO2025217214A2 (en) | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| WO2025214477A1 (en) | 2024-04-12 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Treatment of genetic cardiomyopathies with aav gene therapy vectors |
| CN118459608B (zh) * | 2024-04-26 | 2025-01-24 | 泰州博莱得利生物科技有限公司 | 犬细小病毒特异性免疫制剂、制备方法和应用 |
| WO2025237990A1 (en) | 2024-05-14 | 2025-11-20 | Institut National de la Santé et de la Recherche Médicale | Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis |
| WO2026006341A1 (en) | 2024-06-24 | 2026-01-02 | Regenxbio Inc. | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
| CN118460614B (zh) * | 2024-07-05 | 2025-03-11 | 凌意(杭州)生物科技有限公司 | 一种腺相关病毒Cap蛋白的表达框 |
| WO2026035687A1 (en) | 2024-08-05 | 2026-02-12 | University Of Rochester | Compositions and methods for stitchr-mediated full-length scn5a expression in vivo |
| WO2026055342A1 (en) | 2024-09-04 | 2026-03-12 | Arsenal Biosciences, Inc. | Synthetic pathway activators |
| WO2026094009A1 (en) | 2024-11-04 | 2026-05-07 | Insmed Incorporated | Compositions and methods for treating duchenne muscular dystrophy |
| WO2026102076A1 (en) | 2024-11-07 | 2026-05-15 | Juno Therapeutics, Inc. | Aav production method |
| WO2026114267A1 (en) | 2024-11-29 | 2026-06-04 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating alzheimer's disease in primates |
| WO2026119983A1 (en) | 2024-12-03 | 2026-06-11 | Tafalgie Therapeutics | Peptides for use in treating pain, wounds and inflammatory diseases |
| WO2026119985A1 (en) | 2024-12-03 | 2026-06-11 | Tafalgie Therapeutics | Peptides for use in treating wounds and inflammatory diseases |
| WO2026122921A1 (en) | 2024-12-05 | 2026-06-11 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| EP4763209A1 (en) | 2024-12-17 | 2026-06-24 | Institut National de la Santé et de la Recherche Médicale | Use of (alpha)s-setmar agonist for preventing or treating glioblastoma |
| WO2026137014A1 (en) | 2024-12-20 | 2026-06-25 | Samsung Bioepis Co., Ltd. | Methods of purifying recombinant adeno-associated viruses |
| WO2026151888A1 (en) | 2025-01-08 | 2026-07-16 | The Trustees Of The University Of Pennsylvania | Compositions for treating friedreich's ataxia |
| WO2026150302A1 (en) | 2025-01-09 | 2026-07-16 | Novartis Ag | Novel aav rep/cap encoding constructs |
| CN120249230B (zh) * | 2025-06-05 | 2025-11-07 | 鼐济医药科技(杭州)有限公司 | 一种用于同时生产多种重组腺相关病毒的方法 |
| CN121142059B (zh) * | 2025-11-17 | 2026-02-17 | 吉林农业大学 | 一种基于dia质谱技术评估微塑料神经毒性的方法及系统 |
Family Cites Families (70)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB8929110D0 (en) * | 1989-12-22 | 1990-02-28 | 3I Res Expl Ltd | Polypeptides and dna encoding therefor |
| US5449616A (en) | 1990-05-23 | 1995-09-12 | University Of Iowa Research Foundation | Nucleic acid encoding dystrophin-associated protein |
| US5173414A (en) * | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| CA2046745A1 (en) | 1991-07-10 | 1993-01-11 | Isaac Neuman | Article including a container containing at least one precious stone |
| US6174666B1 (en) | 1992-03-27 | 2001-01-16 | The United States Of America As Represented By The Department Of Health And Human Services | Method of eliminating inhibitory/instability regions from mRNA |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| 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 |
| US6001650A (en) * | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| JPH11514853A (ja) * | 1995-09-08 | 1999-12-21 | ジエンザイム コーポレイション | 遺伝子治療のための改良されたaavベクター |
| JP3881382B2 (ja) * | 1995-12-01 | 2007-02-14 | イントロヘーネ ベスローテン フェンノートシャップ | 安定にトランスフェクションされた哺乳類細胞における調節されたタンパク質発現 |
| 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 |
| US6083716A (en) | 1996-09-06 | 2000-07-04 | The Trustees Of The University Of Pennsylvania | Chimpanzee adenovirus vectors |
| 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 |
| 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 |
| US20020037867A1 (en) * | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| 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 |
| CA2274146C (en) * | 1996-12-05 | 2007-02-06 | Introgene B.V. | Genetic modification of primate hemopoietic repopulating stem cells |
| US6039942A (en) * | 1996-12-20 | 2000-03-21 | Novo Nordick A/S | Phytase polypeptides |
| 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 |
| 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 |
| 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 |
| 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 |
| DE69939169D1 (de) | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| WO2000011140A1 (en) | 1998-08-20 | 2000-03-02 | The Wistar Institute Of Anatomy And Biology | Methods of augmenting mucosal immunity through systemic priming and mucosal boosting |
| 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 |
| 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 |
| 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 |
| AU781478B2 (en) * | 1999-08-20 | 2005-05-26 | Johns Hopkins University School Of Medicine, The | Methods and compositions for the construction and use of fusion libraries |
| WO2001023001A2 (en) | 1999-09-29 | 2001-04-05 | The Trustees Of The University Of Pennsylvania | Rapid peg-modification |
| 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 |
| 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 |
| EP1228234A2 (en) * | 2000-03-14 | 2002-08-07 | Neurologix, Inc. | Production of chimeric capsid vectors |
| 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 |
| 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 |
| US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| US20020159978A1 (en) * | 2001-02-06 | 2002-10-31 | James Allen | Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions |
| 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 |
| DK2359869T3 (en) * | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| 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 |
| AU2002367943A1 (en) | 2001-12-18 | 2003-12-22 | University Of North Carolina At Chapel Hill | Improved reagents and methods for producing parvoviruses |
| WO2003088899A2 (en) * | 2002-04-05 | 2003-10-30 | The Children's Hospital Of Philadelphia | Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof |
| US7235393B2 (en) * | 2002-04-29 | 2007-06-26 | The Trustees Of The University Of Pennsylvania | Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues |
| PL2657248T3 (pl) | 2003-06-19 | 2017-09-29 | Genzyme Corporation | Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie |
| 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 |
| US20090054823A1 (en) | 2004-09-30 | 2009-02-26 | The Trustees Of The University Of Pennsylvania | Perfusion circuit and use therein in targeted delivery of macromolecules |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| US7788045B2 (en) | 2005-09-01 | 2010-08-31 | Meditasks, Llc | Systems and method for homeostatic blood states |
| CN101528916B (zh) | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| JP2009102988A (ja) | 2007-10-19 | 2009-05-14 | Toyota Motor Corp | 内燃機関の燃料噴射装置 |
| US8236527B2 (en) | 2008-03-14 | 2012-08-07 | Humanzyme Limited | Recombinant production of authentic human proteins using human cell expression systems |
| US20090280103A1 (en) | 2008-04-04 | 2009-11-12 | Martin Flueck | Regulation of muscle repair |
| US8729041B2 (en) | 2008-12-03 | 2014-05-20 | The Johns Hopkins University | Compositions and methods for treating hepatic neoplasia |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| CN103648328B (zh) | 2011-07-15 | 2016-01-06 | 阿尔弗雷德·凯驰两合公司 | 旋转刷和带旋转刷的清扫机 |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| EP3470523A1 (en) | 2012-05-09 | 2019-04-17 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| US9819463B2 (en) | 2016-02-18 | 2017-11-14 | Huawei Technologies Co., Ltd. | Method and apparatus for transmitting data in a wireless communication system |
| RS65241B1 (sr) | 2017-02-28 | 2024-03-29 | Univ Pennsylvania | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
-
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| US10693918B2 (en) | 2017-06-15 | 2020-06-23 | Palo Alto Networks, Inc. | Radio access technology based security in service provider networks |
| US10708306B2 (en) | 2017-06-15 | 2020-07-07 | Palo Alto Networks, Inc. | Mobile user identity and/or SIM-based IoT identity and application identity based security enforcement in service provider networks |
| US11122435B2 (en) | 2017-06-15 | 2021-09-14 | Palo Alto Networks, Inc. | Radio access technology based security in service provider networks |
| US11722532B2 (en) | 2017-06-15 | 2023-08-08 | Palo Alto Networks, Inc. | Security for cellular internet of things in mobile networks based on subscriber identity and application identifier |
| US11805153B2 (en) | 2017-06-15 | 2023-10-31 | Palo Alto Networks, Inc. | Location based security in service provider networks |
| US11838326B2 (en) | 2017-06-15 | 2023-12-05 | Palo Alto Networks, Inc. | Mobile equipment identity and/or IoT equipment identity and application identity based security enforcement in service provider networks |
| US11916967B2 (en) | 2017-06-15 | 2024-02-27 | Palo Alto Networks, Inc. | Mobile user identity and/or sim-based IoT identity and application identity based security enforcement in service provider networks |
| US12010148B2 (en) | 2017-06-15 | 2024-06-11 | Palo Alto Networks, Inc. | Access point name and application identity based security enforcement in service provider networks |
| US12355819B2 (en) | 2017-06-15 | 2025-07-08 | Palo Alto Networks, Inc. | Mobile user identity and/or sim-based IoT identity and application identity based security enforcement in service provider networks |
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