JP2023520764A5 - - Google Patents

Info

Publication number
JP2023520764A5
JP2023520764A5 JP2022557749A JP2022557749A JP2023520764A5 JP 2023520764 A5 JP2023520764 A5 JP 2023520764A5 JP 2022557749 A JP2022557749 A JP 2022557749A JP 2022557749 A JP2022557749 A JP 2022557749A JP 2023520764 A5 JP2023520764 A5 JP 2023520764A5
Authority
JP
Japan
Prior art keywords
cedna vector
itrs
cedna
adjacent
group
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022557749A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023520764A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/023891 external-priority patent/WO2021195214A1/en
Publication of JP2023520764A publication Critical patent/JP2023520764A/ja
Publication of JP2023520764A5 publication Critical patent/JP2023520764A5/ja
Priority to JP2025212226A priority Critical patent/JP2026065641A/ja
Pending legal-status Critical Current

Links

JP2022557749A 2020-03-24 2021-03-24 第ix因子治療薬を発現するための非ウイルス性dnaベクター及びその使用 Pending JP2023520764A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025212226A JP2026065641A (ja) 2020-03-24 2025-12-01 第ix因子治療薬を発現するための非ウイルス性dnaベクター及びその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062993857P 2020-03-24 2020-03-24
US62/993,857 2020-03-24
PCT/US2021/023891 WO2021195214A1 (en) 2020-03-24 2021-03-24 Non-viral dna vectors and uses thereof for expressing factor ix therapeutics

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025212226A Division JP2026065641A (ja) 2020-03-24 2025-12-01 第ix因子治療薬を発現するための非ウイルス性dnaベクター及びその使用

Publications (2)

Publication Number Publication Date
JP2023520764A JP2023520764A (ja) 2023-05-19
JP2023520764A5 true JP2023520764A5 (https=) 2024-04-04

Family

ID=75539973

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022557749A Pending JP2023520764A (ja) 2020-03-24 2021-03-24 第ix因子治療薬を発現するための非ウイルス性dnaベクター及びその使用
JP2025212226A Pending JP2026065641A (ja) 2020-03-24 2025-12-01 第ix因子治療薬を発現するための非ウイルス性dnaベクター及びその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025212226A Pending JP2026065641A (ja) 2020-03-24 2025-12-01 第ix因子治療薬を発現するための非ウイルス性dnaベクター及びその使用

Country Status (10)

Country Link
US (1) US20230138409A1 (https=)
EP (1) EP4127187A1 (https=)
JP (2) JP2023520764A (https=)
KR (1) KR20230003477A (https=)
CN (1) CN115667530A (https=)
AU (1) AU2021244555A1 (https=)
CA (1) CA3172572A1 (https=)
IL (1) IL296660A (https=)
MX (1) MX2022011805A (https=)
WO (1) WO2021195214A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220288231A1 (en) * 2019-08-12 2022-09-15 Generation Bio Co. Methods and compositions for reducing gene or nucleic acid therapy-related immune responses
WO2023069948A1 (en) 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
CN119072330A (zh) * 2022-03-14 2024-12-03 世代生物公司 异源初免加强疫苗组合物和使用方法
CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技(杭州)有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
AU2024257493A1 (en) * 2023-04-18 2025-12-04 Siren Biotechnology, Inc. Adeno-associated viral vector compositions and methods of use

Family Cites Families (134)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
ATE78010T1 (de) 1987-04-24 1992-07-15 Arturo Broggini Kuenstliche steine und verfahren zur herstellung derselben.
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US6605712B1 (en) 1990-12-20 2003-08-12 Arch Development Corporation Gene transcription and ionizing radiation: methods and compositions
WO1992011033A1 (en) 1990-12-20 1992-07-09 Arch Development Corporation Control of gene expression by ionizing radiation
US5364791A (en) 1992-05-14 1994-11-15 Elisabetta Vegeto Progesterone receptor having C. terminal hormone binding domain truncations
US5571797A (en) 1994-05-11 1996-11-05 Arch Development Corporation Method of inducing gene expression by ionizing radiation
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5834306A (en) 1994-12-23 1998-11-10 Sri International Tissue specific hypoxia regulated therapeutic constructs
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US5928906A (en) 1996-05-09 1999-07-27 Sequenom, Inc. Process for direct sequencing during template amplification
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US7098320B1 (en) 1996-07-29 2006-08-29 Nanosphere, Inc. Nanoparticles having oligonucleotides attached thereto and uses therefor
GB9709421D0 (en) 1997-05-10 1997-07-02 Zeneca Ltd Chemical compounds
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6709858B1 (en) 1997-11-03 2004-03-23 The Arizona Board Of Regents On Behalf Of The University Of Arizona Hyperthermic inducible expression vectors for gene therapy and methods of use thereof
WO1999025385A1 (en) 1997-11-17 1999-05-27 Imarx Pharmaceutical Corp. A method of increasing nucleic acid synthesis with ultrasound
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
GB9810423D0 (en) 1998-05-15 1998-07-15 Cancer Res Campaign Tech Ionizing radiation or diathermy-switched gene therapy vectors and their use in antitumour therapy
EP1155056A4 (en) 1998-12-04 2004-05-12 Mosaic Technologies Inc METHOD OF IMMOBILIZING OLIGONUCLEOTIDES
WO2001064956A2 (en) 2000-03-01 2001-09-07 Amgen Inc. The identification and use of effectors and allosteric molecules for the alteration of gene expression
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US6716622B2 (en) 2000-07-18 2004-04-06 Uab Research Foundation Tissue-specific self-inactivating gene therapy vector
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
FR2824431A1 (fr) 2001-05-03 2002-11-08 Mitsubishi Electric Inf Tech Methode et dispositif de reception de signal
WO2004090108A2 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals Irna conjugates
CA2521464C (en) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Irna conjugates
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
US7840263B2 (en) 2004-02-27 2010-11-23 Cardiac Pacemakers, Inc. Method and apparatus for device controlled gene expression
WO2006069782A2 (en) 2004-12-27 2006-07-06 Silence Therapeutics Ag. Lipid complexes coated with peg and their use
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
AU2005330637B2 (en) 2004-08-04 2012-09-20 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
AU2005306801B2 (en) 2004-11-08 2012-05-17 The Regents Of The University Of Colorado Structure-based compound design involving riboswitches
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US8852916B2 (en) 2010-01-22 2014-10-07 The Invention Science Fund I, Llc Compositions and methods for therapeutic delivery with microorganisms
US20110172826A1 (en) 2005-12-14 2011-07-14 Amodei Dario G Device including altered microorganisms, and methods and systems of use
WO2007100412A2 (en) 2005-12-21 2007-09-07 Yale University Methods and compositions related to the modulation of riboswitches
US8324436B2 (en) 2006-01-03 2012-12-04 Honeywell International Inc. Gas phase synthesis of 2,3,3,3-tetrafluoro-1-propene from 2-chloro-3,3,3-trifluoro-1-propene
US20070190028A1 (en) 2006-02-13 2007-08-16 Jihong Qu Method and apparatus for heat or electromagnetic control of gene expression
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
US9388425B2 (en) 2006-10-20 2016-07-12 Trustees Of Boston University Tunable genetic switch for regulating gene expression
WO2009018332A1 (en) 2007-08-01 2009-02-05 Alnylam Pharmaceuticals, Inc. Single-stranded and double-stranded oligonucleotides comprising a metal-chelating ligand
WO2009082606A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Folate conjugates
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
US8883211B2 (en) 2008-07-10 2014-11-11 Serina Therapeutics, Inc. Polyoxazolines with inert terminating groups, polyoxazolines prepared from protected initiating groups and related compounds
WO2010019257A2 (en) 2008-08-13 2010-02-18 The Johns Hopkins University Prodrug activation in cancer cells using molecular switches
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
US8722082B2 (en) 2008-11-10 2014-05-13 Tekmira Pharmaceuticals Corporation Lipids and compositions for the delivery of therapeutics
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
ES2579936T3 (es) 2009-08-20 2016-08-17 Sirna Therapeutics, Inc. Nuevos lípidos catiónicos con diversos grupos de cabeza para el suministro oligonucleotídico
US9222086B2 (en) 2009-09-23 2015-12-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
WO2011053896A2 (en) 2009-10-30 2011-05-05 University Of Miami Hypoxia regulated conditionally silenced aav expressing angiogenic inducers
US9697460B2 (en) 2009-11-30 2017-07-04 Trustees Of Boston University Biological analog-to-digital and digital-to-analog converters
WO2011066651A1 (en) 2009-12-01 2011-06-09 Protiva Biotherapeutics, Inc. Snalp formulations containing antioxidants
EP3296398A1 (en) 2009-12-07 2018-03-21 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
EP2525781A1 (en) 2010-01-22 2012-11-28 Schering Corporation Novel cationic lipids for oligonucleotide delivery
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
DK2575767T3 (en) 2010-06-04 2017-03-13 Sirna Therapeutics Inc HOWEVER UNKNOWN LOW MOLECULAR CATIONIC LIPIDS TO PROCESS OIGONUCLEOTIDES
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
LT4226941T (lt) 2010-08-31 2025-01-10 Glaxosmithkline Biologicals Sa Pegilintos liposomos, skirtos imunogeną koduojančios rnr pristatymui
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
CA2809858C (en) 2010-09-20 2019-11-12 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
CA2811430A1 (en) 2010-09-30 2012-04-05 Merck Sharp & Dohme Corp. Low molecular weight cationic lipids for oligonucleotide delivery
EP3485913A1 (en) 2010-10-21 2019-05-22 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
WO2012068187A1 (en) 2010-11-19 2012-05-24 Merck Sharp & Dohme Corp. Poly(amide) polymers for the delivery of oligonucleotides
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
GB201107768D0 (en) 2011-05-10 2011-06-22 Univ Manchester Riboswitches
WO2012162210A1 (en) 2011-05-26 2012-11-29 Merck Sharp & Dohme Corp. Ring constrained cationic lipids for oligonucleotide delivery
US9222093B2 (en) 2011-06-30 2015-12-29 The University Of Hong Kong Two-way, portable riboswitch mediated gene expression control device
EP4115875A1 (en) 2011-07-06 2023-01-11 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
MX366055B (es) 2011-08-31 2019-06-26 Novartis Ag Liposomas pegilados para admistracion de acido ribonucleico (arn) que codifica para inmunogeno.
EP3456317B1 (en) 2011-09-27 2025-09-24 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
AU2012347605B2 (en) 2011-12-07 2017-09-21 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
JP6182457B2 (ja) 2011-12-12 2017-08-16 協和発酵キリン株式会社 カチオン性脂質を含有するドラックデリバリーシステムのための脂質ナノ粒子
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
AU2013240051B2 (en) 2012-03-27 2017-11-30 Sirna Therapeutics, Inc. Diether based biodegradable cationic lipids for siRNA delivery
AU2013299717B2 (en) 2012-08-06 2018-06-28 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
US9943611B2 (en) 2012-11-01 2018-04-17 California Institute Of Technology Reversible gene expression
WO2015006740A2 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
JP6620093B2 (ja) 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションArbutus Biopharma Corporation メッセンジャーrnaを送達するための組成物及び方法
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
US10426737B2 (en) 2013-12-19 2019-10-01 Novartis Ag Lipids and lipid compositions for the delivery of active agents
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
WO2016011089A1 (en) 2014-07-14 2016-01-21 President And Fellows Of Harvard College Compositions comprising riboregulators and methods of use thereof
JP6637988B2 (ja) 2014-11-18 2020-01-29 アークトゥルス セラピューティクス, インコーポレイテッド Rna送達のためのイオン化可能カチオン性脂質
US11669953B2 (en) 2015-01-30 2023-06-06 Hitachi High-Tech Corporation Pattern matching device and computer program for pattern matching
GB201506507D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Riboswitch inducible gene expression
MX390848B (es) * 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
JP6948313B6 (ja) 2015-09-17 2022-01-14 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物および組成物
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US10550440B2 (en) 2016-02-26 2020-02-04 Arizona Board Of Regents On Behalf Of Arizona State University Synthetic translation-sensing riboswitches and uses thereof
MX2018010633A (es) * 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
CA3020585A1 (en) 2016-04-11 2017-10-19 Arbutus Biopharma Corporation Targeted nucleic acid conjugate compositions
US20180020547A1 (en) 2016-07-13 2018-01-18 Alcatel-Lucent Canada Inc. Underlying recessed component placement
WO2018026762A1 (en) 2016-08-01 2018-02-08 Arizona Board Of Regents On Behalf Of Arizona State University Ultraspecific riboregulators having robust single-nucleotide specificity and in vitro and in vivo uses thereof
WO2018075486A1 (en) 2016-10-17 2018-04-26 Northwestern University Generation of novel metabolite-responsive transcription regulator biosensors
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
US12442015B2 (en) * 2018-01-19 2025-10-14 Generation Bio Co. Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors
MA51915A (fr) * 2018-02-22 2020-12-30 Generation Bio Co Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna)
EP3810782A2 (en) * 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells

Similar Documents

Publication Publication Date Title
JP2023520764A5 (https=)
JP2022524434A5 (https=)
US20250333763A1 (en) Gene therapy constructs for treating wilson disease
JP2022549380A5 (https=)
JP2021087431A5 (https=)
JP2022525302A5 (https=)
JP2023520763A5 (https=)
JP2024009857A5 (https=)
JP2020532981A5 (https=)
JP2019503649A5 (https=)
US12359219B2 (en) Transgene cassettes designed to express a human MECP2 gene
US20240376498A1 (en) Transgene cassettes, aav vectors and aav viral vectors for the expression of human codon-optimized slc6a1
JP2022551744A5 (https=)
JP2023542131A5 (https=)
JP2024546103A (ja) Aavベクターを用いたジャンクトフィリン-2(jph2)遺伝子治療
JP2023065516A5 (https=)
JP2021534766A5 (https=)
JP2021511020A5 (https=)
Youjin et al. The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapy
JP2023542132A5 (https=)
JP2024515788A5 (https=)
CA3158131A1 (en) Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
IL308404A (en) Non-viral dna vectors expressing therapeutic antibodies and uses thereof
WO2024163335A2 (en) Pten gene therapy vectors and uses thereof
HK40102353A (en) Gene therapy constructs for treating wilson disease