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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
Authority
WO
WIPO (PCT)
Prior art keywords
methods
synthetic
enhanced
modified rnas
delivery
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.)
Ceased
Application number
PCT/US2012/066055
Other languages
English (en)
Other versions
WO2013078199A2 (fr
Inventor
Derrick Rossi
Morag Stewart
Pankaj MANDAL
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Boston Childrens Hospital
Original Assignee
Boston Childrens Hospital
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 Boston Childrens Hospital filed Critical Boston Childrens Hospital
Publication of WO2013078199A2 publication Critical patent/WO2013078199A2/fr
Publication of WO2013078199A3 publication Critical patent/WO2013078199A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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Classifications

    • 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)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (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 Ceased 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

Family

ID=48470410

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/066055 Ceased 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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US12123002B2 (en) 2014-11-14 2024-10-22 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US12305189B2 (en) 2017-08-03 2025-05-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV

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US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
HRP20220250T1 (hr) 2011-10-03 2022-04-29 Modernatx, Inc. Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe
PT2791160T (pt) 2011-12-16 2022-07-04 Modernatx Inc Composições arnm modificado
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, 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
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
JP6189415B2 (ja) 2012-04-02 2017-08-30 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 細胞質および細胞骨格タンパク質の産生のための修飾ポリヌクレオチド
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US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
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EP3500585A4 (fr) 2016-08-17 2020-04-01 Factor Bioscience Inc. Produits d'acides nucléiques et leurs procédés d'administration
EP3831281A1 (fr) 2016-08-30 2021-06-09 The Regents of The University of California Procédés de ciblage et d'administration biomédicaux, et dispositifs et systèmes pour leur mise en uvre
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US11993645B2 (en) 2017-01-11 2024-05-28 The Board Of Trustees Of The Leland Stanford Junior University Compositions comprising R-Spondin (RSPO) surrogate molecules
US11958891B2 (en) 2017-01-26 2024-04-16 Surrozen Operating, Inc. Tissue-specific Wnt signal enhancing molecules and uses thereof
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12123002B2 (en) 2014-11-14 2024-10-22 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US12305189B2 (en) 2017-08-03 2025-05-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
US12116589B2 (en) 2017-10-16 2024-10-15 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)

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