WO2013078199A3 - Méthodes pour une administration in vivo améliorée d'arn synthétiques modifiés - Google Patents

Méthodes pour une administration in vivo améliorée d'arn synthétiques modifiés Download PDF

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Publication number
WO2013078199A3
WO2013078199A3 PCT/US2012/066055 US2012066055W WO2013078199A3 WO 2013078199 A3 WO2013078199 A3 WO 2013078199A3 US 2012066055 W US2012066055 W US 2012066055W WO 2013078199 A3 WO2013078199 A3 WO 2013078199A3
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WO
WIPO (PCT)
Prior art keywords
methods
synthetic
enhanced
modified rnas
delivery
Prior art date
Application number
PCT/US2012/066055
Other languages
English (en)
Other versions
WO2013078199A2 (fr
Inventor
Derrick Rossi
Morag Stewart
Pankaj MANDAL
Original Assignee
Children's Medical Center Corporation
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.)
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Publication date
Application filed by Children's Medical Center Corporation filed Critical Children's Medical Center Corporation
Publication of WO2013078199A2 publication Critical patent/WO2013078199A2/fr
Publication of WO2013078199A3 publication Critical patent/WO2013078199A3/fr

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    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7084—Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

L'invention concerne des compositions monodoses, des kits, des dispositifs d'administration, et des méthodes pour améliorer l'administration d'ARN synthétiques modifiés codant pour un agent thérapeutique à un patient. Ces compositions monodoses, kits, dispositifs d'administration, et méthodes pour améliorer l'administration peuvent être utilisés pour exprimer un agent thérapeutique désiré sans introduire d'ADN exogène ou des vecteurs viraux, et ainsi, ne provoquent pas de modifications permanentes du génome et n'ont pas un potentiel pour produire des effets mutagènes inattendus. Les ARN fournis au moyen des dosages de l'invention se sont avérés pouvoir produire une réaction dépendante de la dose en termes d'expression et de fonction de protéine.
PCT/US2012/066055 2011-11-23 2012-11-20 Méthodes pour une administration in vivo améliorée d'arn synthétiques modifiés WO2013078199A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161563171P 2011-11-23 2011-11-23
US61/563,171 2011-11-23

Publications (2)

Publication Number Publication Date
WO2013078199A2 WO2013078199A2 (fr) 2013-05-30
WO2013078199A3 true WO2013078199A3 (fr) 2013-07-25

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/066055 WO2013078199A2 (fr) 2011-11-23 2012-11-20 Méthodes pour une administration in vivo améliorée d'arn synthétiques modifiés

Country Status (1)

Country Link
WO (1) WO2013078199A2 (fr)

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US9149506B2 (en) 2012-04-02 2015-10-06 Moderna Therapeutics, Inc. Modified polynucleotides encoding septin-4
US12116589B2 (en) 2017-10-16 2024-10-15 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US12123002B2 (en) 2022-12-20 2024-10-22 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)

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CA2831613A1 (fr) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Administration et formulation d'acides nucleiques genetiquement modifies
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP3492109B1 (fr) 2011-10-03 2020-03-04 ModernaTX, Inc. Nucléosides, nucléotides et acides nucléiques modifiés et leurs utilisations
RS63244B1 (sr) 2011-12-16 2022-06-30 Modernatx Inc Kompozicije modifikovane mrna
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EP3052106A4 (fr) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucléotides codant des polypeptides de modulation immunitaire
SG11201602503TA (en) 2013-10-03 2016-04-28 Moderna Therapeutics Inc Polynucleotides encoding low density lipoprotein receptor
BR112016024644A2 (pt) 2014-04-23 2017-10-10 Modernatx Inc vacinas de ácido nucleico
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3169693B1 (fr) 2014-07-16 2022-03-09 ModernaTX, Inc. Polynucléotides chimériques
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
RU2749882C2 (ru) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
EP3218484A4 (fr) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la sclérose latérale amyotrophique (sla)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3543339A1 (fr) * 2015-02-13 2019-09-25 Factor Bioscience Inc. Produits d'acides nucléiques et leurs procédés d'administration
EP3324979B1 (fr) 2015-07-21 2022-10-12 ModernaTX, Inc. Vaccins contre une maladie infectieuse
BR112018008090A2 (pt) 2015-10-22 2018-11-13 Modernatx Inc vacina de vírus do herpes simplex.
WO2017070620A2 (fr) 2015-10-22 2017-04-27 Modernatx, Inc. Vaccin contre le virus de la grippe à large spectre
AU2016342045A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Human cytomegalovirus vaccine
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CA3002912A1 (fr) 2015-10-22 2017-04-27 Modernatx, Inc. Vaccins a base d'acide nucleique contre le virus varicelle-zona (vzv)
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JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
MA45051A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant la relaxine
CN116115629A (zh) 2016-08-17 2023-05-16 菲克特生物科学股份有限公司 核酸产品及其施用方法
EP3506817A4 (fr) 2016-08-30 2020-07-22 The Regents of The University of California Procédés de ciblage et d'administration biomédicaux, et dispositifs et systèmes pour la mise en oeuvre de ceux-ci
EP3528827A4 (fr) 2016-10-21 2020-11-04 Merck Sharp & Dohme Corp. Vaccins contre la grippe à base de protéine hémagglutinine
WO2018132572A1 (fr) 2017-01-11 2018-07-19 The Board Of Trustees Of The Leland Stanford Junior University Molécules de substitution de r-spondine (rspo)
AU2018211985A1 (en) 2017-01-26 2019-07-18 Surrozen Operating, Inc. Tissue-specific Wnt signal enhancing molecules and uses thereof
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EP3999093A4 (fr) 2019-07-19 2023-11-22 Merck Sharp & Dohme LLC Polypeptides de glycoprotéines e antigéniques, compositions et leurs procédés d'utilisation
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TW202424187A (zh) 2022-10-27 2024-06-16 美商輝瑞大藥廠 Rna分子
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9149506B2 (en) 2012-04-02 2015-10-06 Moderna Therapeutics, Inc. Modified polynucleotides encoding septin-4
US12116589B2 (en) 2017-10-16 2024-10-15 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US12123002B2 (en) 2022-12-20 2024-10-22 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)

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