MX2022015042A - Composiciones de trem y métodos relacionados con las mismas. - Google Patents

Composiciones de trem y métodos relacionados con las mismas.

Info

Publication number
MX2022015042A
MX2022015042A MX2022015042A MX2022015042A MX2022015042A MX 2022015042 A MX2022015042 A MX 2022015042A MX 2022015042 A MX2022015042 A MX 2022015042A MX 2022015042 A MX2022015042 A MX 2022015042A MX 2022015042 A MX2022015042 A MX 2022015042A
Authority
MX
Mexico
Prior art keywords
trem
compositions
methods relating
methods
trna
Prior art date
Application number
MX2022015042A
Other languages
English (en)
Inventor
Neil Kubica
Theonie Anastassiadis
Qingyi Li
David Charles Donnell Butler
Original Assignee
Flagship Pioneering Innovations Vi Llc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Flagship Pioneering Innovations Vi Llc filed Critical Flagship Pioneering Innovations Vi Llc
Publication of MX2022015042A publication Critical patent/MX2022015042A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Detergent Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Immunology (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)

Abstract

La divulgación se refiere en general a métodos para modular un parámetro de producción de un ARN correspondiente a, o un polipéptido codificado por, una secuencia de ácido nucleico que comprende un ORF endógeno que tiene un codón de terminación prematura, que comprende administrar una molécula efectora basada en ARNt que tiene una modificación de origen no natural.
MX2022015042A 2020-05-29 2021-05-28 Composiciones de trem y métodos relacionados con las mismas. MX2022015042A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063031941P 2020-05-29 2020-05-29
PCT/US2021/034999 WO2021243290A1 (en) 2020-05-29 2021-05-28 Trem compositions and methods relating thereto

Publications (1)

Publication Number Publication Date
MX2022015042A true MX2022015042A (es) 2023-03-09

Family

ID=76797080

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022015042A MX2022015042A (es) 2020-05-29 2021-05-28 Composiciones de trem y métodos relacionados con las mismas.

Country Status (10)

Country Link
US (1) US20230203510A1 (es)
EP (1) EP4158031A1 (es)
JP (1) JP2023527413A (es)
KR (1) KR20230029685A (es)
CN (1) CN116018405A (es)
AU (1) AU2021278984A1 (es)
CA (1) CA3180101A1 (es)
IL (1) IL298539A (es)
MX (1) MX2022015042A (es)
WO (1) WO2021243290A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4377457A1 (en) * 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Trem compositions and uses thereof
IL311867A (en) * 2021-10-13 2024-06-01 Flagship Pioneering Innovations Vi Llc TREM compositions and methods of use
CN117138048B (zh) * 2023-10-30 2024-02-02 中山大学中山眼科中心 Dock6在制备防治眼部新生血管性疾病药物中的应用

Family Cites Families (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US99823A (en) 1870-02-15 Improved indigo soap
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6693086B1 (en) 1998-06-25 2004-02-17 National Jewish Medical And Research Center Systemic immune activation method using nucleic acid-lipid complexes
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
JP4380411B2 (ja) 2004-04-30 2009-12-09 澁谷工業株式会社 滅菌方法
EP2992902A1 (en) 2004-12-27 2016-03-09 Silence Therapeutics GmbH Lipid complexes coated with peg and their uses
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US7451428B2 (en) 2005-02-24 2008-11-11 Texas Instruments Incorporated Merging sub-resolution assist features of a photolithographic mask through the use of a merge bar
GB0600948D0 (en) * 2006-01-18 2006-02-22 Univ Dundee Prevention/Treatment Of Ichthyosis Vulgaris, Atopy And Other Disorders
JP2009534690A (ja) 2006-07-10 2009-09-24 メムシック，インコーポレイテッド 磁場センサーを用いて偏揺れを感知するためのシステム、および、前記システムを用いた携帯用の電子装置
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
PL2279254T3 (pl) 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
EP2350043B9 (en) 2008-10-09 2014-08-20 TEKMIRA Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
ES2656516T3 (es) 2008-10-20 2018-02-27 Alnylam Pharmaceuticals, Inc. Composiciones y métodos para inhibir la expresión de transtiretina
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
KR102264822B1 (ko) 2008-11-10 2021-06-14 알닐람 파마슈티칼스 인코포레이티드 치료제 운반용 신규 지질 및 조성물
WO2010054384A1 (en) 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Lipids and compositions for the delivery of therapeutics
EA028860B1 (ru) 2009-06-10 2018-01-31 Арбутус Биофарма Корпорэйшн Улучшенная липидная композиция
JP5766188B2 (ja) 2009-07-01 2015-08-19 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 固形腫瘍に治療剤を送達するための脂質製剤
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
ES2579936T3 (es) 2009-08-20 2016-08-17 Sirna Therapeutics, Inc. Nuevos lípidos catiónicos con diversos grupos de cabeza para el suministro oligonucleotídico
EP2506879A4 (en) 2009-12-01 2014-03-19 Protiva Biotherapeutics Inc PREPARATIONS OF SNALP CONTAINING ANTIOXIDANTS
EP3296398A1 (en) 2009-12-07 2018-03-21 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
US20130116419A1 (en) 2010-01-22 2013-05-09 Daniel Zewge Post-synthetic chemical modification of rna at the 2'-position of the ribose ring via "click" chemistry
WO2011097480A1 (en) 2010-02-05 2011-08-11 University Of Louisville Research Foundation, Inc. Exosomal compositions and methods for the treatment of disease
JP4782232B1 (ja) 2010-04-09 2011-09-28 シャープ株式会社 光源モジュール、及びそれを備えた電子機器
EP2569276B1 (en) 2010-05-12 2021-02-24 Arbutus Biopharma Corporation 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
IL300109A (en) 2010-06-03 2023-03-01 Alnylam Pharmaceuticals Inc Biodegradable lipids for the transfer of active substances
EP2575767B1 (en) 2010-06-04 2017-01-04 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US9676810B2 (en) * 2010-07-08 2017-06-13 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
EP3542789A3 (en) 2010-08-31 2020-01-01 GlaxoSmithKline Biologicals SA Lipids suitable for liposomal delivery of protein-coding rna
BR112013004585B1 (pt) 2010-09-20 2021-09-08 Merck Sharp & Dohme Corp Lipídeo catiônico, composição de lnp, e, uso de um lipídeo catiônico
CA2811430A1 (en) 2010-09-30 2012-04-05 Merck Sharp & Dohme Corp. Low molecular weight cationic lipids for oligonucleotide delivery
CN103153347A (zh) 2010-10-21 2013-06-12 默沙东公司 用于寡核苷酸递送的新型低分子量阳离子脂质
US9617461B2 (en) 2010-12-06 2017-04-11 Schlumberger Technology Corporation Compositions and methods for well completions
AU2012207606B2 (en) 2011-01-11 2017-02-23 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
WO2012162210A1 (en) 2011-05-26 2012-11-29 Merck Sharp & Dohme Corp. Ring constrained cationic lipids for oligonucleotide delivery
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
EP3456317A1 (en) 2011-09-27 2019-03-20 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
PE20150041A1 (es) 2011-10-27 2015-01-28 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco
US20140308212A1 (en) 2011-11-07 2014-10-16 University Of Louisville Research Foundation, Inc. Edible plant-derived microvesicle compositions for diagnosis and treatment of disease
WO2013073480A1 (ja) 2011-11-18 2013-05-23 日油株式会社 細胞内動態を改善したカチオン性脂質
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
EP3988537A1 (en) 2011-12-07 2022-04-27 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
CA3170051A1 (en) 2011-12-07 2013-06-13 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
WO2013126803A1 (en) 2012-02-24 2013-08-29 Protiva Biotherapeutics Inc. 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
ES2887254T3 (es) 2013-03-08 2021-12-22 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
JP6486955B2 (ja) 2013-11-18 2019-03-20 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なカチオン性脂質
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
EP3071515A2 (en) 2013-11-18 2016-09-28 Rubius Therapeutics, Inc. Synthetic membrane-receiver complexes
ES2774968T3 (es) 2013-12-19 2020-07-23 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
WO2015095346A1 (en) 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
AU2015241422B2 (en) 2014-04-01 2020-12-03 Rubius Therapeutics, Inc. Methods and compositions for immunomodulation
CN106795096B (zh) 2014-06-25 2020-05-29 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
WO2016115632A1 (en) * 2015-01-21 2016-07-28 Exerkine Corporation Method for treating mitochondrial disease
US20180135012A1 (en) 2015-05-13 2018-05-17 Rubius Therapeutics, Inc. Membrane-receiver complex therapeutics
JP6800410B2 (ja) 2015-06-19 2020-12-16 マサチューセッツ インスティテュート オブ テクノロジー アルケニル置換２，５−ピペラジンジオン、および、対象または細胞に剤を送達するための組成物におけるそれらの使用
DK3313829T3 (da) 2015-06-29 2024-06-17 Acuitas Therapeutics Inc Lipider og lipide nanopartikelformuleringer til levering af nukleinsyrer
US20180362974A1 (en) 2015-07-02 2018-12-20 University Of Louisville Research Foundation, Inc. EDIBLE PLANT-DERIVED MICROVESICLE COMPOSITIONS FOR DELIVERY OF miRNA AND METHODS FOR TREATMENT OF CANCER
LT3350157T (lt) 2015-09-17 2022-02-25 Modernatx, Inc. Junginiai ir kompozicijos terapinei medžiagai teikti intraceliuliniu būdu
CA3201644A1 (en) 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
AU2016366978B2 (en) 2015-12-10 2022-07-28 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
US20190022247A1 (en) 2015-12-30 2019-01-24 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
KR20180095098A (ko) 2016-01-11 2018-08-24 루비우스 테라퓨틱스, 아이엔씨. 암 적응증에 대한 다중양식 치료 세포 시스템과 관련된 조성물 및 방법
WO2017173054A1 (en) 2016-03-30 2017-10-05 Intellia Therapeutics, Inc. Lipid nanoparticle formulations for crispr/cas components
JP7080826B2 (ja) 2016-05-16 2022-06-06 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム カチオン性スルホンアミドアミノ脂質および両親媒性両性イオンアミノ脂質
US20200315967A1 (en) 2016-06-24 2020-10-08 Modernatx, Inc. Lipid nanoparticles
KR20190026819A (ko) 2016-07-07 2019-03-13 루비우스 테라퓨틱스, 아이엔씨. 외인성 rna를 발현하는 치료용 세포 시스템과 관련된 조성물 및 방법
EP3532103A1 (en) 2016-10-26 2019-09-04 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
KR20190091497A (ko) 2016-12-02 2019-08-06 루비우스 테라퓨틱스, 아이엔씨. 고형 종양으로의 침투를 위한 세포 시스템과 관련된 조성물 및 방법
MX2019009839A (es) 2017-02-17 2019-10-22 Rubius Therapeutics Inc Celulas eritroides funcionalizadas.
MX2019013312A (es) 2017-05-08 2020-08-17 Flagship Pioneering Innovations V Inc Composiciones para facilitar la fusion de membranas y usos de estas.
EP3679148A4 (en) 2017-09-08 2021-06-09 Generation Bio Co. LIPID NANOPARTICLE FORMULATIONS OF NON-VIRAL CAPSIDE-FREE DNA VECTORS
TWI833708B (zh) 2017-09-29 2024-03-01 美商英特利亞醫療公司 調配物
BR112020005323A2 (pt) 2017-09-29 2020-09-24 Intellia Therapeutics, Inc. polinucleotídeos, composições e métodos para edição de genoma
WO2019217941A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of suppressing pathogenic mutations using programmable base editor systems
US20210378980A1 (en) 2018-09-20 2021-12-09 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
BR112021005788A2 (pt) * 2018-09-26 2021-10-26 Case Western Reserve University Métodos e composições para aumentar a expressão de proteínas e/ou tratar um distúrbio de haploinsuficiência
EP3867225A1 (en) 2018-10-18 2021-08-25 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
AU2019384557A1 (en) 2018-11-21 2021-06-10 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
WO2020150608A1 (en) * 2019-01-18 2020-07-23 Flagship Pioneering, Inc. Trem compositions and uses thereof
CA3137956A1 (en) 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles
WO2020243560A1 (en) * 2019-05-31 2020-12-03 Flagship Pioneering, Inc. Uses of trem compositions to modulate trna pools
CA3160097A1 (en) * 2019-11-04 2021-05-14 Flagship Pioneering, Inc. Trem compositions for con-rare codons and related uses

Also Published As

Publication number Publication date
CN116018405A (zh) 2023-04-25
EP4158031A1 (en) 2023-04-05
US20230203510A1 (en) 2023-06-29
IL298539A (en) 2023-01-01
JP2023527413A (ja) 2023-06-28
AU2021278984A1 (en) 2022-11-17
WO2021243290A1 (en) 2021-12-02
CA3180101A1 (en) 2021-12-02
KR20230029685A (ko) 2023-03-03

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