MA50802A - Polynucléotides codant pour des sous-unités alpha et bêta de propionyl-coa carboxylase pour le traitement de l'acidémie propionique - Google Patents

Polynucléotides codant pour des sous-unités alpha et bêta de propionyl-coa carboxylase pour le traitement de l'acidémie propionique

Info

Publication number
MA50802A
MA50802A MA050802A MA50802A MA50802A MA 50802 A MA50802 A MA 50802A MA 050802 A MA050802 A MA 050802A MA 50802 A MA50802 A MA 50802A MA 50802 A MA50802 A MA 50802A
Authority
MA
Morocco
Prior art keywords
propionyl
alpha
units
treatment
coa carboxylase
Prior art date
Application number
MA050802A
Other languages
English (en)
Inventor
Lin Tung Guey
Lei Jiang
Paolo G V Martini
Vladimir Presnyak
Original Assignee
Modernatx Inc
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 Modernatx Inc filed Critical Modernatx Inc
Publication of MA50802A publication Critical patent/MA50802A/fr

Links

Classifications

    • 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
    • A61K48/0025—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
    • A61K48/0033—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 the non-active part being non-polymeric
    • 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
    • A61K38/53—Ligases (6)
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y604/00—Ligases forming carbon-carbon bonds (6.4)
    • C12Y604/01—Ligases forming carbon-carbon bonds (6.4.1)
    • C12Y604/01003—Propionyl-CoA carboxylase (6.4.1.3)
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
MA050802A 2017-11-22 2018-11-21 Polynucléotides codant pour des sous-unités alpha et bêta de propionyl-coa carboxylase pour le traitement de l'acidémie propionique MA50802A (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762590199P 2017-11-22 2017-11-22
US201862614787P 2018-01-08 2018-01-08
US201862663024P 2018-04-26 2018-04-26
US201862693552P 2018-07-03 2018-07-03
US201862747356P 2018-10-18 2018-10-18

Publications (1)

Publication Number Publication Date
MA50802A true MA50802A (fr) 2020-09-30

Family

ID=64661518

Family Applications (1)

Application Number Title Priority Date Filing Date
MA050802A MA50802A (fr) 2017-11-22 2018-11-21 Polynucléotides codant pour des sous-unités alpha et bêta de propionyl-coa carboxylase pour le traitement de l'acidémie propionique

Country Status (6)

Country Link
US (2) US20220265856A1 (fr)
EP (1) EP3714045A1 (fr)
JP (1) JP7424976B2 (fr)
CA (1) CA3079543A1 (fr)
MA (1) MA50802A (fr)
WO (1) WO2019104195A1 (fr)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2014296278C1 (en) 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
CA2974503A1 (fr) 2015-01-21 2016-07-28 Phaserx, Inc. Procedes, compositions et systemes permettant l'administration d'agents therapeutiques et diagnostiques dans des cellules
EP3562510A4 (fr) 2016-12-30 2021-01-06 Genevant Sciences GmbH Molécules de peg ramifié, compositions et procédés associés
WO2021076797A1 (fr) * 2019-10-15 2021-04-22 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Gènes synthétiques pour le traitement de l'acidémie propionique provoquée par des mutations de la propionyl-coa carboxylase bêta
WO2022204380A1 (fr) 2021-03-24 2022-09-29 Modernatx, Inc. Nanoparticules lipidiques contenant des polynucléotides codant pour des sous-unités alpha et bêta de propionyl-coa carboxylase et leurs utilisations
WO2023287751A1 (fr) * 2021-07-12 2023-01-19 Modernatx, Inc. Polynucléotides codant pour les sous-unités alpha et bêta de la propionyl-coa carboxylase pour le traitement de l'acidémie propionique
WO2023158738A2 (fr) * 2022-02-16 2023-08-24 Trustees Of Tufts College Nanoparticules lipidiques pour administration à l'œil ou à l'oreille

Family Cites Families (132)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
US5563250A (en) 1987-12-02 1996-10-08 Neorx Corporation Cleavable conjugates for the delivery and release of agents in native form
US5505931A (en) 1993-03-04 1996-04-09 The Dow Chemical Company Acid cleavable compounds, their preparation and use as bifunctional acid-labile crosslinking agents
US5723332A (en) 1993-11-26 1998-03-03 British Technology Group Limited Translational enhancer DNA
US5681702A (en) 1994-08-30 1997-10-28 Chiron Corporation Reduction of nonspecific hybridization by using novel base-pairing schemes
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US6004573A (en) 1997-10-03 1999-12-21 Macromed, Inc. Biodegradable low molecular weight triblock poly(lactide-co-glycolide) polyethylene glycol copolymers having reverse thermal gelation properties
ZA9811377B (en) 1997-12-12 1999-08-27 Expression Genetics Inc Positively charged poly[alpha-(omega-aminoalkyl) glycolic acid[ for the delivery of a bioactive agent via tissue and cellular uptake.
US6517869B1 (en) 1997-12-12 2003-02-11 Expression Genetics, Inc. Positively charged poly(alpha-(omega-aminoalkyl)lycolic acid) for the delivery of a bioactive agent via tissue and cellular uptake
AU758368B2 (en) 1998-01-05 2003-03-20 University Of Massachusetts Enhanced transport using membrane disruptive agents
US6426086B1 (en) 1998-02-03 2002-07-30 The Regents Of The University Of California pH-sensitive, serum-stable liposomes
DE69939740D1 (de) 1998-05-20 2008-11-27 Expression Genetics Inc Laktose oder galaktose-polyethylen glykol-gepfropfte poly-l-lysin als genträger
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
MXPA01000271A (es) 1998-07-13 2002-10-17 Expression Genetics Inc Analogo de poliester de poli-lisina como un portador para el suministro de genes, soluble, biodegradable.
WO2000075164A1 (fr) 1999-06-07 2000-12-14 Mirus Corporation Compositions et methodes de diffusion de medicaments au moyen de molecules sensibles au ph
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
WO2001051092A2 (fr) 2000-01-07 2001-07-19 University Of Washington Transport ameliore d'agents au moyen d'agents perturbateurs de membranes
US20040142474A1 (en) 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US6696038B1 (en) 2000-09-14 2004-02-24 Expression Genetics, Inc. Cationic lipopolymer as biocompatible gene delivery agent
US6897196B1 (en) 2001-02-07 2005-05-24 The Regents Of The University Of California pH sensitive lipids based on ortho ester linkers, composition and method
US6652886B2 (en) 2001-02-16 2003-11-25 Expression Genetics Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents
US6586524B2 (en) 2001-07-19 2003-07-01 Expression Genetics, Inc. Cellular targeting poly(ethylene glycol)-grafted polymeric gene carrier
AU2002319668A1 (en) 2001-07-27 2003-02-17 President And Fellows Of Harvard College Laminar mixing apparatus and methods
JP4371812B2 (ja) 2001-09-28 2009-11-25 マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ マイクロｒｎａ分子
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
WO2003092665A2 (fr) 2002-05-02 2003-11-13 Massachusetts Eye And Ear Infirmary Systemes oculaires de distribution de medicament et utilisation associee
WO2005007819A2 (fr) 2003-07-09 2005-01-27 Wisconsin Alumni Research Foundation Polymeres a charge dynamique et transport de composes anioniques
WO2005013901A2 (fr) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Composes oligomeres et compositions utilisables pour moduler des petits arn non-codants
US7927873B2 (en) 2003-12-19 2011-04-19 University Of Cincinnati Polyamides for nucleic acid delivery
NZ550320A (en) 2004-04-15 2010-02-26 Chiasma Inc Compositions capable of facilitating penetration across a biological barrier
US8057821B2 (en) 2004-11-03 2011-11-15 Egen, Inc. Biodegradable cross-linked cationic multi-block copolymers for gene delivery and methods of making thereof
EP1856179B1 (fr) 2004-12-10 2013-05-15 Kala Pharmaceuticals, Inc. Copolymeres sequences de poly(ether-anhydride) fonctionnalises
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US8273339B2 (en) 2005-04-12 2012-09-25 Nektar Therapeutics Polymer-based compositions and conjugates of antimicrobial agents
MX2007016561A (es) 2005-06-30 2008-03-10 Archemix Corp Materiales y metodos para la generacion de transcriptos de acido nucleico completamente modificados en la posicion 2.
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
AU2006286228A1 (en) 2005-09-01 2007-03-08 Novartis Vaccines And Diagnostics Gmbh & Co Kg Multiple vaccination including serogroup C meningococcus
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
JP2009519033A (ja) 2005-12-16 2009-05-14 ディアト 核酸を細胞に送達するための細胞貫通ペプチド結合体
US20100168034A1 (en) 2006-02-20 2010-07-01 Ewha University-Industry Collaboration Foundation Peptide having cell membrane penetrating activity
US8309680B2 (en) 2006-02-21 2012-11-13 Nektar Therapeutics Segmented degradable polymers and conjugates made therefrom
CN101583379B (zh) 2006-10-05 2013-04-03 约翰斯霍普金斯大学 使用优良聚合物纳米粒子的水溶性差药物的水可分散性口服，肠胃外和局部制剂
EP2500015A1 (fr) 2006-12-05 2012-09-19 Landec Corporation Administration de médicaments
EP2120859B1 (fr) 2006-12-21 2013-11-20 Stryker Corporation Formulations à libération entretenue comprenant des cristaux de bmp-7
EP2207891B1 (fr) 2006-12-22 2012-07-25 Archemix LLC Matériaux et procédés pour la génération de transcrits comprenant des nucléotides modifiés
BRPI0720619B1 (pt) 2006-12-27 2022-04-05 Nektar Therapeutics Composto de fator ix-poli(etileno glicol) com uma ligação liberável, método para preparar o mesmo e composição
US20100015218A1 (en) 2007-02-16 2010-01-21 Vasant Jadhav Compositions and methods for potentiated activity of biologically active molecules
PT2136788E (pt) 2007-03-30 2012-02-03 Bind Biosciences Inc Direccionamento a células de cancro utilizando nanopartículas
HUE040417T2 (hu) 2007-05-04 2019-03-28 Marina Biotech Inc Aminosavlipidek és alkalmazásuk
CA2715078C (fr) 2007-09-26 2019-07-23 Intrexon Corporation Regions non traduites 5' (5'utr) synthetiques, vecteurs d'expression, et procedes pour augmenter l'expression transgenique
US20100004315A1 (en) 2008-03-14 2010-01-07 Gregory Slobodkin Biodegradable Cross-Linked Branched Poly(Alkylene Imines)
WO2010005721A2 (fr) 2008-06-16 2010-01-14 Bind Biosciences, Inc. Nanoparticules polymères pharmacologiquement chargées et leurs méthodes de fabrication et d’utilisation
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
WO2010005726A2 (fr) 2008-06-16 2010-01-14 Bind Biosciences Inc. Nanoparticules polymères thérapeutiques avec inhibiteurs de mtor et procédés de fabrication et d’utilisation associés
JP2012501966A (ja) 2008-06-16 2012-01-26 バインド バイオサイエンシズ インコーポレイテッド ビンカアルカロイド含有治療用ポリマーナノ粒子並びにその製造方法及び使用方法
ES2654533T3 (es) 2008-06-16 2018-02-14 Pfizer Inc. Procedimientos para la preparación de copolímeros dibloque funcionalizados con un agente de direccionamiento para su uso en la fabricación de nanopartículas terapéuticas
US20100087337A1 (en) 2008-09-10 2010-04-08 Bind Biosciences, Inc. High Throughput Fabrication of Nanoparticles
CN104910025B (zh) 2008-11-07 2019-07-16 麻省理工学院 氨基醇类脂质和其用途
US20100216804A1 (en) 2008-12-15 2010-08-26 Zale Stephen E Long Circulating Nanoparticles for Sustained Release of Therapeutic Agents
WO2010087791A1 (fr) 2009-01-27 2010-08-05 Utc Power Corporation Réacteur de conversion à la vapeur d'eau intégré, refroidi de manière distributive et atomiseur
CA2755963C (fr) 2009-03-20 2019-04-02 Egen, Inc. Derives de polyamine
KR20230098713A (ko) 2009-06-10 2023-07-04 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
JP5823405B2 (ja) 2009-11-04 2015-11-25 ザ ユニバーシティ オブ ブリティッシュ コロンビア 核酸含有脂質粒子および関連方法
WO2011062965A2 (fr) 2009-11-18 2011-05-26 University Of Washington Through Its Center For Commercialization Monomères de ciblage et polymère ayant des blocs de ciblage
WO2011069529A1 (fr) 2009-12-09 2011-06-16 Curevac Gmbh Solution contenant du mannose pour la lyophilisation, la transfection et/ou l'injection d'acides nucléiques
EP2512487A4 (fr) 2009-12-15 2013-08-07 Nanoparticules polymères thérapeutiques comprenant de corticostéroïdes, et procédés pour les fabriquer et les utiliser
EA201290498A1 (ru) 2009-12-15 2013-01-30 Байнд Байосайенсиз, Инк. Терапевтические полимерные наночастицы, включающие эпотилон, и способы их получения и применения
JP5965844B2 (ja) 2009-12-15 2016-08-10 バインド セラピューティックス インコーポレイテッド 高いガラス転移温度または高分子量のコポリマーを有する治療用ポリマーナノ粒子組成物
WO2011106086A1 (fr) 2010-02-25 2011-09-01 Albert Einstein College Of Medicine Of Yeshiva University Polymères d'albumine pégylés et leurs utilisations
US20130037977A1 (en) 2010-04-08 2013-02-14 Paul A. Burke Preparation of Lipid Nanoparticles
CN103002878B (zh) 2010-04-09 2015-07-01 帕西拉制药有限公司 用于配制大直径合成膜囊泡的方法
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
NZ704191A (en) 2010-06-14 2016-05-27 Hoffmann La Roche Cell-penetrating peptides and uses therof
US20130196948A1 (en) 2010-06-25 2013-08-01 Massachusetts Insitute Of Technology Polymers for biomaterials and therapeutics
MX2013000164A (es) 2010-07-06 2013-03-05 Novartis Ag Liposomas con lipidos que tienen valor de pka ventajoso para suministro de arn.
JP6025721B2 (ja) 2010-07-06 2016-11-16 ノバルティス アーゲー カチオン性水中油型エマルジョン
WO2012006376A2 (fr) 2010-07-06 2012-01-12 Novartis Ag Particules d'administration de type virion pour des molécules d'arn auto-répliquant
US20130211249A1 (en) 2010-07-22 2013-08-15 The Johns Hopkins University Drug eluting hydrogels for catheter delivery
CA2801523C (fr) 2010-07-30 2021-08-03 Curevac Gmbh Complexation d'acides nucleiques avec des composants cationiques reticules par un pont disulfure pour une transfection et une immunostimulation
WO2012021516A2 (fr) 2010-08-09 2012-02-16 The Trustees Of The University Of Pennsylvania Structures hybrides nanoparticulaires-oligonucléotidiques et leurs méthodes d'utilisation
EP2606072A4 (fr) 2010-08-19 2016-04-20 Peg Biosciences Inc Conjugués synergiques biomolécule-polymère
KR101451840B1 (ko) 2010-08-24 2014-10-16 게케엔 드리펠린 인터나쇼날 게엠베하 전이 영역을 가진 조인트용, 특히 등속 조인트용 부트
RS63984B1 (sr) 2010-08-31 2023-03-31 Glaxosmithkline Biologicals Sa Mali lipozomi za isporuku rnk koja kodira imunogen
HRP20220695T1 (hr) 2010-08-31 2022-07-08 Glaxosmithkline Biologicals Sa Pegilirani liposomi za isporuku rnk kodirane za imunogen
BR112013004865A2 (pt) 2010-08-31 2016-06-07 Novartis Ag lípidos adequados para entrega lipossomal de codificadores de proteínas rna
WO2012039979A2 (fr) 2010-09-10 2012-03-29 The Johns Hopkins University Diffusion rapide de grosses nanoparticules polymères dans le cerveau de mammifères
CA2811601A1 (fr) 2010-09-24 2012-03-29 Mallinckrodt Llc Conjugues d'aptameres pour ciblage de nanovecteurs therapeutiques et/ou diagnostiques
JP2013543844A (ja) 2010-10-22 2013-12-09 バインド セラピューティックス インコーポレイテッド 高分子コポリマーを含む治療用ナノ粒子
DK3202760T3 (da) 2011-01-11 2019-11-25 Alnylam Pharmaceuticals Inc Pegylerede lipider og deres anvendelse til lægemiddelfremføring
US20140066363A1 (en) 2011-02-07 2014-03-06 Arun K. Bhunia Carbohydrate nanoparticles for prolonged efficacy of antimicrobial peptide
EP2489371A1 (fr) 2011-02-18 2012-08-22 Instituto Nacional de Investigacion y Tecnologia Agraria y Alimentaria Support peptidique pour l'administration de médicaments
US8846850B2 (en) 2011-02-22 2014-09-30 Rutgers, The State University Of New Jersey Amphiphilic macromolecules for nucleic acid delivery
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
AU2012267578B2 (en) 2011-06-08 2017-04-20 Translate Bio, Inc. Cleavable lipids
BR112013031553A2 (pt) 2011-06-08 2020-11-10 Shire Human Genetic Therapies, Inc. composições, mrna que codifica para uma hgla e seu uso, uso de pelo menos uma molécula de mrna e um veículo de transferência e uso de um mrna que codifica para proteína exógena
KR20140067070A (ko) 2011-08-31 2014-06-03 말린크로트 엘엘씨 Ｈ-포스포네이트에 의한 나노입자 ｐｅｇ 개질
WO2013039857A1 (fr) 2011-09-12 2013-03-21 modeRNA Therapeutics Acides nucléiques modifiés et leurs procédés d'utilisation
US9428535B2 (en) 2011-10-03 2016-08-30 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
WO2013082111A2 (fr) 2011-11-29 2013-06-06 The University Of North Carolina At Chapel Hill Particules manipulées de façon géométrique et procédés de modulation de réponses des macrophages ou immunitaires
US9463247B2 (en) 2011-12-07 2016-10-11 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
CA2856742A1 (fr) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipides biodegradables pour l'administration d'agents actifs
EP2787977A4 (fr) 2011-12-09 2015-05-06 Univ California Encapsulation liposomale de médicaments
WO2013106073A1 (fr) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprenant des polymères réticulés de liaison à des cations et utilisations associées
WO2013106086A1 (fr) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprenant des polymères réticulés de liaison à des cations et utilisations associées
WO2013106072A1 (fr) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprenant des polymères réticulés de liaison à des cations et utilisations associées
WO2013105101A1 (fr) 2012-01-13 2013-07-18 Department Of Biotechnology Nanoparticules lipidiques solides encapsulant un médicament hydrophile/amphiphile et leur procédé de préparation
CN104936620B (zh) 2012-01-19 2019-08-09 约翰霍普金斯大学 增强粘膜渗透的纳米粒子调配物
WO2013116126A1 (fr) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Nouveaux lipides cationiques biodégradables de faible masse moléculaire pour la délivrance d'oligonucléotides
JP6356614B2 (ja) 2012-02-17 2018-07-11 ユニバーシティ・オブ・ジョージア・リサーチ・ファウンデイション・インコーポレイテッド 薬剤のミトコンドリア輸送のためのナノ粒子
CN104394853B (zh) 2012-02-19 2017-10-10 纳维基因股份有限公司 多孔性纳米结构在递送中的用途
WO2013124867A1 (fr) 2012-02-21 2013-08-29 Amrita Vishwa Vidyapeetham University Polymer - polymer or polymer - protein core - shell nano medicine loaded with multiple drug molecules
CA2865506C (fr) 2012-03-15 2021-07-06 Omeros Corporation Composition et procede de diversification de sequences cible
AU2013243946A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
EP2885419A4 (fr) 2012-08-14 2016-05-25 Moderna Therapeutics Inc Enzymes et polymérases destinées à la synthèse d'arn
US20150307542A1 (en) 2012-10-03 2015-10-29 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
JP2016500254A (ja) * 2012-12-05 2016-01-12 サンガモ バイオサイエンシーズ， インコーポレイテッド 代謝疾患の調節のための方法および組成物
EP2964234A4 (fr) 2013-03-09 2016-12-07 Moderna Therapeutics Inc Régions non traduites hétérologues pour arnm
AU2014296278C1 (en) * 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
CA2926218A1 (fr) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides codant pour un recepteur de lipoproteines de faible densite
EP3110401A4 (fr) 2014-02-25 2017-10-25 Merck Sharp & Dohme Corp. Adjuvants de vaccins sous forme de nanoparticules lipidiques et systèmes d'administration d'antigènes
EP3736261B1 (fr) 2015-09-17 2023-10-11 ModernaTX, Inc. Composés et compositions pour l'administration intracellulaire d'agents thérapeutiques
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
EP3364982A4 (fr) * 2015-10-22 2019-04-17 ModernaTX, Inc. Vaccins contre les maladies sexuellement transmissibles
SI3718565T1 (sl) * 2015-10-22 2022-08-31 Modernatx, Inc. Cepiva za respiratorni virus
US11389546B2 (en) * 2015-12-09 2022-07-19 Modernatx, Inc. Heterologous UTR sequences for enhanced mRNA expression
EP3452101A2 (fr) * 2016-05-04 2019-03-13 CureVac AG Arn codant pour une protéine thérapeutique
EP3458081A1 (fr) * 2016-05-18 2019-03-27 Modernatx, Inc. Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
US11786607B2 (en) * 2017-06-15 2023-10-17 Modernatx, Inc. RNA formulations
MA49634A (fr) * 2017-07-21 2020-05-27 Modernatx Inc Arnm modifié codant pour une propionyl-coa-carboxylase et utilisations associées

Also Published As

Publication number Publication date
US20220265856A1 (en) 2022-08-25
CA3079543A1 (fr) 2019-05-31
EP3714045A1 (fr) 2020-09-30
US20240024506A1 (en) 2024-01-25
JP7424976B2 (ja) 2024-01-30
WO2019104195A1 (fr) 2019-05-31
JP2021504331A (ja) 2021-02-15

Similar Documents

Publication Publication Date Title
MA50802A (fr) Polynucléotides codant pour des sous-unités alpha et bêta de propionyl-coa carboxylase pour le traitement de l'acidémie propionique
MA49402A (fr) Polynucléotides aadc pour le traitement de la maladie de parkinson
MA45481A (fr) Utilisation d'exosomes pour le traitement de maladies
MA47310A (fr) Combinaisons de cabozantinib et d'atzolizumab pour traiter le cancer
MA45089A (fr) Utilisation d'indazoles 2-substitués pour le traitement et la prophylaxie de maladies auto-immunes
MA45192A (fr) Traitement d'association
MA41028A (fr) Méthodes et formulations pour le traitement de pathologies oculaires vasculaires
MA47719A (fr) Esketamine pour le traitement de la dépression
MA50849A (fr) Substances et procédés pour le traitement d'hémoglobinopathies
MA50803A (fr) Polynucléotides codant pour l'ornithine transcarbamylase pour le traitement de troubles du cycle de l'urée
MA50155A (fr) Acides aminés à chaîne ramifiée pour le traitement d'une lésion neuronale
MA52631A (fr) Compositions et méthodes pour le traitement de la maladie de parkinson
FR3032353B1 (fr) Composition pharmaceutique et dispositif pour le traitement de la douleur
MA42657A (fr) Anticorps inhibiteurs anti-pcsk9 destinés au traitement des patients atteints d'hyperlipidémie subissant une aphérèse de lipoprotéines
MA43170A (fr) Association d'un inhibiteur de bcl-2 et d'un inhibiteur de mek pour le traitement du cancer
MA53329A (fr) Méthodes de traitement de l'épilepsie
MA49131A (fr) Utilisation d'antagonistes de klk5 pour le traitement d'une maladie
IL276896A (en) Methods for treating Crohn's disease with a specific anti-IL23 antibody
MA44700A (fr) Combinaison d'un anticorps anti-il-10 et d'un oligonucléotide de type cpg-c pour le traitement du cancer
MA44864A (fr) Trithérapie pour le traitement d'une maladie intestinale inflammatoire
MA54792A (fr) Compositions et méthodes pour le traitement de la maladie de parkinson
MA41629A (fr) Compositions et méthodes d'utilisation de l'hormone anti-müllérienne pour le traitement de l'infertilité
FR3044227B1 (fr) Derives aminophosphiniques pour la prevention et le traitement des douleurs oculaires
FR3020951B1 (fr) Association d'un tetrapeptide et d'un glyceryl ester pour le traitement de l'alopecie androgenique.
IL275349A (en) Use of hM4Di in the treatment of seizure disorders