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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
Application number
PCT/US2012/066055
Other languages
French (fr)
Other versions
WO2013078199A2 (en
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.)
Filing date
Publication date
Application filed by Children's Medical Center Corporation filed Critical Children's Medical Center Corporation
Publication of WO2013078199A2 publication Critical patent/WO2013078199A2/en
Publication of WO2013078199A3 publication Critical patent/WO2013078199A3/en

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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 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 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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US12305189B2 (en) 2022-10-20 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
HUE057725T2 (en) 2011-10-03 2022-06-28 Modernatx Inc Modified nucleosides, nucleotides and nucleic acids and their uses
LT2791160T (en) 2011-12-16 2022-06-10 Modernatx, Inc. MODIFIED MRNR COMPOSITIONS
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
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
PT2922554T (en) 2012-11-26 2022-06-28 Modernatx Inc Terminally modified rna
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
AU2014329452B2 (en) 2013-10-03 2019-06-20 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
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US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
RU2716422C2 (en) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112410339A (en) 2014-11-14 2021-02-26 沃雅戈治疗公司 Regulatory polynucleotides
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
JP7199809B2 (en) * 2015-02-13 2023-01-06 ファクター バイオサイエンス インコーポレイテッド Nucleic acid product and its administration method
WO2017015463A2 (en) 2015-07-21 2017-01-26 Modernatx, Inc. Infectious disease vaccines
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EA201891000A1 (en) 2015-10-22 2018-12-28 МОДЕРНАТиЭкс, ИНК. VACCINE AGAINST RESPIRATORY-SYNCTIAL VIRUS
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
RU2764587C2 (en) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Methods and compositions for treatment of huntington's chorea
BR112018073683A2 (en) 2016-05-18 2019-02-26 Modernatx, Inc. relaxin encoding polynucleotides
RU2758488C2 (en) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Modulating polynucleotides
CN116115629A (en) 2016-08-17 2023-05-16 菲克特生物科学股份有限公司 Nucleic acid products and methods of administration thereof
CN110650673B (en) 2016-08-30 2024-04-09 加利福尼亚大学董事会 Methods for biomedical targeting and delivery and devices and systems for practicing the methods
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WO2018132572A1 (en) 2017-01-11 2018-07-19 The Board Of Trustees Of The Leland Stanford Junior University 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
KR20190110612A (en) 2017-02-01 2019-09-30 모더나티엑스, 인크. Immunomodulatory Therapeutic MRNA Compositions Encoding Activating Oncogene Mutant Peptides
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MX2019013172A (en) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease.
ES2952779T3 (en) 2017-05-18 2023-11-06 Modernatx Inc Modified messenger RNA comprising functional RNA elements
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Cited By (2)

* 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
US12305189B2 (en) 2022-10-20 2025-05-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV

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