JP2023520763A - ゴーシェ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 - Google Patents

ゴーシェ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 Download PDF

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JP2023520763A
JP2023520763A JP2022557747A JP2022557747A JP2023520763A JP 2023520763 A JP2023520763 A JP 2023520763A JP 2022557747 A JP2022557747 A JP 2022557747A JP 2022557747 A JP2022557747 A JP 2022557747A JP 2023520763 A JP2023520763 A JP 2023520763A
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cedna
itr
gba
cedna vector
itrs
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ダグラス アンソニー カー，
フィリップ サマヨア，
ナサニエル シルバー，
ルーク エス． ハム，
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ジェネレーション バイオ カンパニー
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
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    • C12N9/14—Hydrolases (3)
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    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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JP2022557747A 2020-03-24 2021-03-24 ゴーシェ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 JP2023520763A (ja)

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US202062993866P 2020-03-24 2020-03-24
US62/993,866 2020-03-24
PCT/US2021/023898 WO2021195218A1 (en) 2020-03-24 2021-03-24 Non-viral dna vectors and uses thereof for expressing gaucher therapeutics

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JP2023520763A true JP2023520763A (ja) 2023-05-19
JP2023520763A5 JP2023520763A5 (https=) 2024-04-04

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US (1) US20230134550A1 (https=)
EP (1) EP4127186A1 (https=)
JP (1) JP2023520763A (https=)
KR (1) KR20230003478A (https=)
CN (1) CN115667531A (https=)
AU (1) AU2021244559A1 (https=)
CA (1) CA3172591A1 (https=)
IL (1) IL296662A (https=)
MX (1) MX2022011806A (https=)
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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024546887A (ja) * 2021-12-17 2024-12-26 ジェネンテック， インコーポレイテッド オリゴヌクレオチドｇｂａアゴニスト
WO2023139496A1 (en) * 2022-01-21 2023-07-27 Pfizer Inc. Gene therapy for gaucher disease
CN118715316B (zh) * 2022-10-08 2025-09-19 凌意(杭州)生物科技有限公司 用于治疗GCase缺乏相关疾病的多核苷酸
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019051255A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. CLOSED-ENDED DNA (CEDNA) MODIFIED
WO2019165050A1 (en) * 2018-02-22 2019-08-29 Generation Bio Co. Controlled expression of transgenes using close-ended dna (cedna) vectors
WO2020012164A1 (en) * 2018-07-13 2020-01-16 Ucl Business Ltd Glucocerebrosidase gene therapy

Family Cites Families (131)

* 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
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
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
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
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
US20110172826A1 (en) 2005-12-14 2011-07-14 Amodei Dario G Device including altered microorganisms, and methods and systems of use
US8852916B2 (en) 2010-01-22 2014-10-07 The Invention Science Fund I, Llc Compositions and methods for therapeutic delivery with microorganisms
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
BRPI0810949A2 (pt) * 2007-05-29 2015-10-27 Christopher B Reid "método de preparação de células multipotentes, auto-renovadoras, diferenciadoras ou resistentes a doenças, célula e vetor pra uso do método"
WO2009018332A1 (en) 2007-08-01 2009-02-05 Alnylam Pharmaceuticals, Inc. Single-stranded and double-stranded oligonucleotides comprising a metal-chelating ligand
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
WO2009082606A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Folate conjugates
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
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
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
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric 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
BR112017024514A2 (pt) * 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
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
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
KR20200120649A (ko) * 2018-02-14 2020-10-21 제너레이션 바이오 컴퍼니 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도
EP3810782A2 (en) * 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019051255A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. CLOSED-ENDED DNA (CEDNA) MODIFIED
WO2019165050A1 (en) * 2018-02-22 2019-08-29 Generation Bio Co. Controlled expression of transgenes using close-ended dna (cedna) vectors
WO2020012164A1 (en) * 2018-07-13 2020-01-16 Ucl Business Ltd Glucocerebrosidase gene therapy

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"GenBank", HOMO SAPIENS GLUCOSIDASE, ACCESSION NO. BC003356, vol. [online], [retrieved on 2025.02.07], JPN6025005685, 2006, ISSN: 0005819911 *
UNIPROTKB, LYSOSOMAL ACID GLUCOSYLCERAMIDASE, ACCESSION NO. P04062, vol. [online], [retrieved on 2025.02.07], JPN6025005684, 2004, ISSN: 0005819912 *

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