AU2020366209A1 - mRNA encoding granulocyte-macrophage colony stimulating factor for treating Parkinson's disease - Google Patents

mRNA encoding granulocyte-macrophage colony stimulating factor for treating Parkinson's disease Download PDF

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Publication number
AU2020366209A1
AU2020366209A1 AU2020366209A AU2020366209A AU2020366209A1 AU 2020366209 A1 AU2020366209 A1 AU 2020366209A1 AU 2020366209 A AU2020366209 A AU 2020366209A AU 2020366209 A AU2020366209 A AU 2020366209A AU 2020366209 A1 AU2020366209 A1 AU 2020366209A1
Authority
AU
Australia
Prior art keywords
mir
seq
lnp
lipid
utr
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.)
Abandoned
Application number
AU2020366209A
Other languages
English (en)
Inventor
Seymour DE PICCIOTTO
Howard Gendelman
Eric Yi-Chun Huang
Jared IACOVELLI
Laurie KENNEY
R. Lee Mosley
Katherine OLSON
Sze-Wah TSE
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.)
ModernaTx Inc
University of Nebraska System
Original Assignee
University of Nebraska–Lincoln
ModernaTx Inc
University of Nebraska System
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 University of Nebraska–Lincoln, ModernaTx Inc, University of Nebraska System filed Critical University of Nebraska–Lincoln
Publication of AU2020366209A1 publication Critical patent/AU2020366209A1/en
Abandoned 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
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/193—Colony stimulating factors [CSF]
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Psychology (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
AU2020366209A 2019-10-15 2020-10-15 mRNA encoding granulocyte-macrophage colony stimulating factor for treating Parkinson's disease Abandoned AU2020366209A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962915317P 2019-10-15 2019-10-15
US62/915,317 2019-10-15
US202063013139P 2020-04-21 2020-04-21
US63/013,139 2020-04-21
PCT/US2020/055852 WO2021076811A1 (en) 2019-10-15 2020-10-15 Mrnas encoding granulocyte-macrophage colony stimulating factor for treating parkinson's disease

Publications (1)

Publication Number Publication Date
AU2020366209A1 true AU2020366209A1 (en) 2022-05-19

Family

ID=73198473

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020366209A Abandoned AU2020366209A1 (en) 2019-10-15 2020-10-15 mRNA encoding granulocyte-macrophage colony stimulating factor for treating Parkinson's disease

Country Status (6)

Country Link
US (1) US20240123034A1 (enExample)
EP (1) EP4045076A1 (enExample)
JP (1) JP2022552378A (enExample)
AU (1) AU2020366209A1 (enExample)
CA (1) CA3158013A1 (enExample)
WO (1) WO2021076811A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202214445D0 (en) * 2022-09-30 2022-11-16 Imp College Innovations Ltd Gene therapy
WO2024240962A1 (en) * 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof

Family Cites Families (22)

* 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
US5723332A (en) 1993-11-26 1998-03-03 British Technology Group Limited Translational enhancer DNA
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
EP2428570B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
US20040197312A1 (en) * 2003-04-02 2004-10-07 Marina Moskalenko Cytokine-expressing cellular vaccine combinations
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
EP2089049A4 (en) * 2006-11-17 2011-11-16 Biogen Idec Inc SYSTEMIC ADMINISTRATION OF COLONIAL STIMULATING FACTORS TO TREAT AMYLOID ASSOCIATED DISEASES
EP2143794A1 (en) * 2007-04-27 2010-01-13 Kyushu University, National University Corporation Viral vector for gene therapy
DK2205618T3 (en) 2007-09-26 2017-02-20 Intrexon Corp SYNTHETIC 5 'NON-TRANSLATED REGIONS, EXPRESSION VECTORS AND PROCEDURES FOR INCREASING TRANSGENIC EXPRESSION
JP5539962B2 (ja) 2008-04-25 2014-07-02 ノースウェスタン、ユニバーシティ コレステロールを隔離するのに適したナノ構造体
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
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US9567398B2 (en) * 2010-11-18 2017-02-14 Merck Serono S.A. Antibody with specificity for GM-CSF (II)
US9999673B2 (en) 2011-01-11 2018-06-19 Alnylam Pharmaceuticals, Inc. PEGylated lipids and their use for drug delivery
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
WO2014164253A1 (en) 2013-03-09 2014-10-09 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
WO2016176330A1 (en) * 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response
DK3386484T3 (da) 2015-12-10 2022-07-04 Modernatx Inc Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler
RS63625B1 (sr) 2016-05-18 2022-10-31 Modernatx Inc Kombinacije irnk koje kodiraju imunomodulirajuće polipeptide i njihova upotreba
US20190314291A1 (en) * 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells

Also Published As

Publication number Publication date
CA3158013A1 (en) 2021-04-22
US20240123034A1 (en) 2024-04-18
WO2021076811A1 (en) 2021-04-22
EP4045076A1 (en) 2022-08-24
JP2022552378A (ja) 2022-12-15

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Legal Events

Date Code Title Description
MK1 Application lapsed section 142(2)(a) - no request for examination in relevant period