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WO2013078199A3 - Methods for enhanced in vivo delivery of synthetic, modified rnas - Google Patents

Methods for enhanced in vivo delivery of synthetic, modified rnas 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
French (fr)
Other versions
WO2013078199A2 (en
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/en
Publication of WO2013078199A3 publication Critical patent/WO2013078199A3/en
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

Landscapes

  • 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

Described herein are unit dose compositions, kits, delivery devices, and methods for enhancing delivery of a synthetic, modified RNAs encoding a therapeutic agent to a subject. These unit dose compositions, kits, delivery devices, and methods for enhancing delivery can be used to express a desired therapeutic agent without the introduction of any exogenous DNA or viral vectors, and thus, do not cause permanent modification of the genome or have the potential for unintended mutagenic effects. The RNAs provided using the dosages as provided have been shown to provide a dose-dependent response both in terms of protein expression and function.
PCT/US2012/066055 2011-11-23 2012-11-20 Methods for enhanced in vivo delivery of synthetic, modified rnas Ceased WO2013078199A2 (en)

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 (en) 2013-05-30
WO2013078199A3 true WO2013078199A3 (en) 2013-07-25

Family

ID=48470410

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/066055 Ceased WO2013078199A2 (en) 2011-11-23 2012-11-20 Methods for enhanced in vivo delivery of synthetic, modified rnas

Country Status (1)

Country Link
WO (1) WO2013078199A2 (en)

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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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WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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US20130156849A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
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US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
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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
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US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
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AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
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JP2019531787A (en) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Biomedical targeting and delivery method and apparatus and system for performing the same
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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
JP7305543B2 (en) 2017-01-26 2023-07-10 スロゼン オペレーティング， インコーポレイテッド Tissue-specific Wnt signal enhancing molecules and uses thereof
EP3576780A1 (en) 2017-02-01 2019-12-11 Modernatx, Inc. Immunomodulatory therapeutic mrna compositions encoding activating oncogene mutation peptides
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
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US11485972B2 (en) 2017-05-18 2022-11-01 Modernatx, Inc. Modified messenger RNA comprising functional RNA elements
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US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
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Non-Patent Citations (1)

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Title
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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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