JP2003525912A5 - - Google Patents

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JP2003525912A5
JP2003525912A5 JP2001564801A JP2001564801A JP2003525912A5 JP 2003525912 A5 JP2003525912 A5 JP 2003525912A5 JP 2001564801 A JP2001564801 A JP 2001564801A JP 2001564801 A JP2001564801 A JP 2001564801A JP 2003525912 A5 JP2003525912 A5 JP 2003525912A5
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Japan
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acid
polymer
preparation according
nucleic acid
formulation
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JP2001564801A
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Japanese (ja)
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JP4987205B2 (ja
JP2003525912A (ja
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Priority claimed from PCT/US2001/006953 external-priority patent/WO2001066149A2/en
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Publication of JP2003525912A5 publication Critical patent/JP2003525912A5/ja
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JP2001564801A 2000-03-03 2001-03-02 遺伝子送達用核酸製剤および使用方法 Expired - Lifetime JP4987205B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US18723600P 2000-03-03 2000-03-03
US60/187,236 2000-03-03
US26175101P 2001-01-16 2001-01-16
US60/261,751 2001-01-16
PCT/US2001/006953 WO2001066149A2 (en) 2000-03-03 2001-03-02 Nucleic acid formulations for gene delivery and methods of use

Publications (3)

Publication Number Publication Date
JP2003525912A JP2003525912A (ja) 2003-09-02
JP2003525912A5 true JP2003525912A5 (enExample) 2008-04-17
JP4987205B2 JP4987205B2 (ja) 2012-07-25

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JP2001564801A Expired - Lifetime JP4987205B2 (ja) 2000-03-03 2001-03-02 遺伝子送達用核酸製剤および使用方法

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US (2) US7173116B2 (enExample)
EP (1) EP1259265B1 (enExample)
JP (1) JP4987205B2 (enExample)
AT (1) ATE511400T1 (enExample)
AU (1) AU2001245427A1 (enExample)
BR (1) BR0108962A (enExample)
CA (1) CA2401327C (enExample)
DK (1) DK1259265T3 (enExample)
WO (1) WO2001066149A2 (enExample)

Families Citing this family (93)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040192593A1 (en) * 1999-07-26 2004-09-30 Baylor College Of Medicine Protease resistant ti-growth hormone releasing hormone
KR20020047096A (ko) * 1999-07-26 2002-06-21 크로커 사무엘 에스 초활성 돼지 성장 호르몬 분비 호르몬 유사체
CA2423093C (en) 2000-09-25 2013-09-17 Valentis, Inc. Improved system for regulation of transgene expression
US7338656B2 (en) 2001-10-26 2008-03-04 Baylor College Of Medicine Composition and method to alter lean body mass and bone properties in a subject
WO2003059382A2 (en) * 2001-12-28 2003-07-24 Supratek Pharma Inc. Pharmaceutical compositions comprising polyanionic polymers and amphiphilic block copolymers and methods of use thereof to improve gene expression
US20080269153A1 (en) * 2002-05-28 2008-10-30 Ruxandra Draghia-Akli Increased stability of a dna formulation by including poly-l-glutamate
US20040014645A1 (en) * 2002-05-28 2004-01-22 Advisys, Inc. Increased delivery of a nucleic acid construct in vivo by the poly-L-glutamate ("PLG") system
WO2003099341A1 (en) * 2002-05-28 2003-12-04 Advisys, Inc. Increased delivery of a nucleic acid constrtuct in vivo by the poly-l-glutamate (plg) system
DE60336736D1 (de) * 2002-07-16 2011-05-26 VGX Pharmaceuticals LLC Codon-optimierte synthetische plasmide
AU2003302337A1 (en) * 2002-12-31 2004-07-29 The Johns Hopkins University Wound healing method and kits
CA2513743C (en) * 2003-01-28 2013-06-25 Advisys, Inc. Reducing culling in herd animals growth hormone releasing hormone (ghrh)
ES2309508T3 (es) * 2003-03-11 2008-12-16 Laboratoires Serono Sa Vectores de expresion que comprenden el promotor de mcmv-ie2.
AU2004229465B2 (en) * 2003-04-09 2010-04-29 The United States Of America Department Of Veterans Affairs Compositions and methods related to production of erythropoietin
TW200424214A (en) * 2003-04-21 2004-11-16 Advisys Inc Plasmid mediated GHRH supplementation for renal failures
GB0313916D0 (en) * 2003-06-16 2003-07-23 Glaxosmithkline Biolog Sa Vaccine composition
US20090005333A1 (en) * 2004-01-26 2009-01-01 Vgx Pharmaceuticlas, Inc. Reducing culling in herd animals growth hormone releasing hormone (ghrh)
DK2438931T3 (da) 2004-09-22 2013-12-02 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
TWI294460B (en) * 2004-12-23 2008-03-11 Ind Tech Res Inst Method for stabilizing nucleic acids
US20060094023A1 (en) * 2004-11-02 2006-05-04 Industrial Technology Research Institute Method for isolating nucleic acid by using amino surfactants
JP2009518044A (ja) * 2005-12-07 2009-05-07 ジェネトロニクス,インコーポレイティド 可変容積エレクトロポレーションチャンバー及びその使用方法
KR101529448B1 (ko) 2006-03-03 2015-06-18 온코섹 메디컬 인코포레이티드 외과적 절제술 이후에 조직내 남아 있는 미세 잔류 종양을 치료하는 장치
DK2094086T3 (da) * 2006-11-08 2013-11-25 Veritas Bio LLC Indgivelse in vivo af dobbeltstrenget rna til en målcelle
FR2941152B1 (fr) * 2009-01-20 2013-10-18 Centre Nat Rech Scient Vecteurs comprenant une macromolecule anionique et un lipide cationique pour l'administration de petits acides nucleiques.
WO2012018594A2 (en) * 2010-07-26 2012-02-09 Scott & White Healthcare Plant-derived polysaccharides for delivery of rna-based therapies
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CA2821992A1 (en) 2010-10-01 2012-04-05 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
EP2741785B1 (en) 2011-07-12 2018-09-05 Philadelphia Health and Education Corporation Novel clostridium difficile dna vaccine
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
SG11201400527XA (en) 2011-09-16 2014-04-28 Univ Pennsylvania Rna engineered t cells for the treatment of cancer
US9708384B2 (en) 2011-09-22 2017-07-18 The Trustees Of The University Of Pennsylvania Universal immune receptor expressed by T cells for the targeting of diverse and multiple antigens
HRP20220250T1 (hr) 2011-10-03 2022-04-29 Modernatx, Inc. Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe
WO2013063419A2 (en) 2011-10-28 2013-05-02 The Trustees Of The University Of Pennsylvania A fully human, anti-mesothelin specific chimeric immune receptor for redirected mesothelin-expressing cell targeting
SI2791160T1 (sl) 2011-12-16 2022-07-29 Modernatx, Inc. Sestave modificirane MRNA
SG11201404284SA (en) 2012-02-22 2014-10-30 Univ Pennsylvania Use of the cd2 signaling domain in second-generation chimeric antigen receptors
CA3205751A1 (en) 2012-02-22 2013-08-29 The Trustees Of The University Of Pennsylvania Use of icos-based cars to enhance antitumor activity and car persistence
AU2013243952A1 (en) 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
JP2015524255A (ja) 2012-07-13 2015-08-24 ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア 二重特異性抗体を共導入することによってcart細胞の活性を強化する方法
US9365641B2 (en) 2012-10-01 2016-06-14 The Trustees Of The University Of Pennsylvania Compositions and methods for targeting stromal cells for the treatment of cancer
US10117896B2 (en) 2012-10-05 2018-11-06 The Trustees Of The University Of Pennsylvania Use of a trans-signaling approach in chimeric antigen receptors
US9598489B2 (en) 2012-10-05 2017-03-21 The Trustees Of The Univeristy Of Pennsylvania Human alpha-folate receptor chimeric antigen receptor
HUE058785T2 (hu) 2012-10-08 2022-09-28 Biontech Delivery Tech Gmbh Karboxilált poliamin-származékok, mint transzfekciós reagensek
SMT202200337T1 (it) 2012-11-26 2022-09-14 Modernatx Inc Rna modificato al livello del terminale
PL2956477T5 (pl) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika viii
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
CA2931547A1 (en) 2013-12-09 2015-06-18 Durect Corporation Pharmaceutically active agent complexes, polymer complexes, and compositions and methods involving the same
ES2963718T3 (es) 2014-01-21 2024-04-01 Novartis Ag Capacidad presentadora de antígenos de células CAR-T potenciada mediante introducción conjunta de moléculas co-estimuladoras
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
JP7372728B2 (ja) 2014-10-31 2023-11-01 ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア 改変t細胞に関する方法および組成物
AU2015339743C1 (en) 2014-10-31 2021-04-22 The Trustees Of The University Of Pennsylvania Altering gene expression in modified T cells and uses thereof
CN107106609A (zh) 2014-10-31 2017-08-29 宾夕法尼亚大学董事会 刺激和扩展t细胞的组合物和方法
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CA2975147C (en) 2015-01-31 2025-06-10 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC MOLECULES TO T CELLS
HK1250569A1 (zh) 2015-05-01 2018-12-28 The Regents Of The University Of California 聚醣依赖性免疫治疗分子
EP3340998B1 (en) 2015-08-28 2023-01-11 The Trustees of the University of Pennsylvania Methods and compositions for cells expressing a chimeric intracellular signaling molecule
PT3411478T (pt) 2016-02-01 2022-09-13 Bioverativ Therapeutics Inc Genes do fator viii otimizados
US11549099B2 (en) 2016-03-23 2023-01-10 Novartis Ag Cell secreted minibodies and uses thereof
US10233419B2 (en) 2016-06-30 2019-03-19 Zymergen Inc. Apparatuses and methods for electroporation
JP2018035137A (ja) 2016-07-13 2018-03-08 マブイミューン ダイアグノスティックス エイジーMabimmune Diagnostics Ag 新規な抗線維芽細胞活性化タンパク質(fap)結合薬剤およびその使用
CA3058425A1 (en) 2017-03-28 2018-10-04 The Trustees Of The University Of Pennsylvania Methods to protect transplanted tissue from rejection
EP3684399A1 (en) 2017-12-29 2020-07-29 Cellectis Method for improving production of car t cells
CN118325839A (zh) 2018-03-27 2024-07-12 宾夕法尼亚大学董事会 具有增强功能的修饰的免疫细胞及其筛选方法
AU2019269601A1 (en) 2018-05-17 2020-11-26 Regents Of The University Of Minnesota Drug-resistant immune cells and methods of use thereof
US12497611B2 (en) 2018-08-17 2025-12-16 Yale University Compositions and methods for high-throughput activation screening to boost T cell effector function
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US20220170097A1 (en) 2018-10-29 2022-06-02 The Broad Institute, Inc. Car t cell transcriptional atlas
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
CN120944827A (zh) 2019-03-27 2025-11-14 宾夕法尼亚大学董事会 Tn-MUC1嵌合抗原受体(CAR)T细胞疗法
WO2021067389A1 (en) 2019-09-30 2021-04-08 Bioverativ Therapeutics Inc. Lentiviral vector formulations
US20220387626A1 (en) 2019-10-23 2022-12-08 Yale University Compositions and methods comprising viral vector systems for multiplexed activation of endogenous genes as immunotherapy and viral-based immune-gene therapy
WO2021178896A1 (en) 2020-03-06 2021-09-10 Go Therapeutics, Inc. Anti-glyco-cd44 antibodies and their uses
AU2021244937A1 (en) 2020-03-27 2022-11-03 Mendus B.V. In vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy
CA3172449A1 (en) 2020-03-27 2021-09-30 Erik Hans MANTING Ex vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
CN116133679A (zh) 2020-06-30 2023-05-16 门德斯有限公司 白血病衍生细胞在卵巢癌疫苗中的用途
EP4210832A1 (en) 2020-09-14 2023-07-19 Vor Biopharma, Inc. Chimeric antigen receptors for treatment of cancer
CA3196677A1 (en) 2020-11-05 2022-05-12 Erik Hans MANTING Use of tumor-independent antigens in immunotherapies
CA3203705A1 (en) 2021-01-22 2022-07-28 Erik Hans MANTING Methods of tumor vaccination
US20250326855A1 (en) 2021-03-05 2025-10-23 Go Therapeutics, Inc. Anti-glyco-cd44 antibodies and their uses
WO2022190058A1 (en) 2021-03-12 2022-09-15 Dcprime B.V. Methods of vaccination and use of cd47 blockade
KR20240107093A (ko) 2021-08-05 2024-07-08 고 테라퓨틱스, 인크. 항-글리코-muc4 항체 및 그의 용도
TW202328188A (zh) 2021-09-03 2023-07-16 美商Go治療公司 抗醣化-cmet抗體及其用途
AU2022339819A1 (en) 2021-09-03 2024-04-11 Go Therapeutics, Inc. Anti-glyco-lamp1 antibodies and their uses
US20240002800A1 (en) 2022-05-16 2024-01-04 Mendus B.V. Use of leukemia-derived cells for enhancing natural killer (nk) cell therapy
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
CN119925619A (zh) * 2024-12-31 2025-05-06 首都医科大学 一种递送外源质粒dna的赋形剂及其制备和应用

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4543252A (en) * 1982-01-21 1985-09-24 The Regents Of The University Of California Cationic oligopeptides having microbicidal activity
ATE26584T1 (de) * 1983-07-01 1987-05-15 Battelle Memorial Institute In-vivo abbaubares polypeptid und dessen anwendung zur verzoegerten freigabe von medikamenten.
ATE60340T1 (de) * 1984-10-19 1991-02-15 Battelle Memorial Institute Durch mikroorganismen abbaubares polypeptid und seine verwendung fuer die fortschreitende abgabe von medikamenten.
US5749847A (en) * 1988-01-21 1998-05-12 Massachusetts Institute Of Technology Delivery of nucleotides into organisms by electroporation
JP3368603B2 (ja) * 1992-02-28 2003-01-20 オリンパス光学工業株式会社 遺伝子治療用処置具
US5439440A (en) * 1993-04-01 1995-08-08 Genetronics, Inc. Electroporation system with voltage control feedback for clinical applications
WO1994024983A2 (en) 1993-04-28 1994-11-10 Ribozyme Pharmaceuticals, Inc. Ocular delivery of nucleic acid
US5679647A (en) * 1993-08-26 1997-10-21 The Regents Of The University Of California Methods and devices for immunizing a host against tumor-associated antigens through administration of naked polynucleotides which encode tumor-associated antigenic peptides
FR2715847B1 (fr) * 1994-02-08 1996-04-12 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
EP0804249A2 (en) 1994-03-15 1997-11-05 Brown University Research Foundation Polymeric gene delivery system
US6271205B1 (en) * 1994-09-21 2001-08-07 University Of Massachusetts Medical Center Cancer treatment by expression of differentiation factor receptor
US6040295A (en) * 1995-01-13 2000-03-21 Genemedicine, Inc. Formulated nucleic acid compositions and methods of administering the same for gene therapy
FR2732218B1 (fr) * 1995-03-28 1997-08-01 Flamel Tech Sa Particules a base de polyaminoacide(s) et susceptibles d'etre utilisees comme vecteurs de principe(s) actif(s) et leurs procedes de preparation
GB9600272D0 (en) * 1996-01-06 1996-03-06 Univ Nottingham Polymers
US5704908A (en) * 1996-10-10 1998-01-06 Genetronics, Inc. Electroporation and iontophoresis catheter with porous balloon
GB9623051D0 (en) 1996-11-06 1997-01-08 Schacht Etienne H Delivery of DNA to target cells in biological systems
US6048551A (en) * 1997-03-27 2000-04-11 Hilfinger; John M. Microsphere encapsulation of gene transfer vectors
FR2766706B1 (fr) * 1997-07-30 2001-05-25 Biovector Therapeutics Sa Complexes particulaires stables de charge globale neutre ou negative de structure multilamellaire composes par au moins une substance biologiquement active globalement anionique et un constituant cationique, leur preparation et utilisation
US6040925A (en) 1997-12-12 2000-03-21 Hewlett-Packard Company Radial and pruned radial interpolation
US6383811B2 (en) * 1997-12-30 2002-05-07 Mirus Corporation Polyampholytes for delivering polyions to a cell
CA2337652C (en) * 1998-07-13 2013-03-26 Genetronics, Inc. Skin and muscle-targeted gene therapy by pulsed electrical field
US7396919B1 (en) * 1998-07-17 2008-07-08 Mirus Bio Corporation Charge reversal of polyion complexes
US5945290A (en) * 1998-09-18 1999-08-31 Isis Pharmaceuticals, Inc. Antisense modulation of RhoA expression
FR2786098B1 (fr) * 1998-11-20 2003-05-30 Flamel Tech Sa Particules a base de polyaminoacide(s) et susceptibles d'etre utilisees comme vecteurs de principe(s) actif(s), suspension colloidale les comprenant et leurs procedes de fabrication
WO2000078791A2 (en) 1999-06-17 2000-12-28 Universiteit Gent FUNCTIONAL POLY-α-AMINOACID DERIVATIVES USEFUL FOR THE MODIFICATION OF BIOLOGICALLY ACTIVE MATERIALS AND THEIR APPLICATION
WO2000078358A2 (en) 1999-06-18 2000-12-28 The Collaborative Group, Ltd. Hyaluronic acid microspheres for sustained gene transfer
EP1209971A4 (en) 1999-08-20 2004-04-14 Mirus Corp POLYION COMPLEX LOAD INVERSION
AU2001241958A1 (en) * 2000-03-03 2001-09-17 Valentis, Inc. Improved poloxamer and poloxamine compositions for nucleic acid delivery
CA2423093C (en) * 2000-09-25 2013-09-17 Valentis, Inc. Improved system for regulation of transgene expression

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