JP2022552378A - パーキンソン病を治療するための顆粒球マクロファージコロニー刺激因子をコードするｍＲＮＡ - Google Patents

パーキンソン病を治療するための顆粒球マクロファージコロニー刺激因子をコードするｍＲＮＡ Download PDF

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JP2022552378A
JP2022552378A JP2022522647A JP2022522647A JP2022552378A JP 2022552378 A JP2022552378 A JP 2022552378A JP 2022522647 A JP2022522647 A JP 2022522647A JP 2022522647 A JP2022522647 A JP 2022522647A JP 2022552378 A JP2022552378 A JP 2022552378A
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mir
lnp
csf
seq
lipid
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JP2022552378A5 (enExample
Inventor
エリック イー－チュン フアン，
ジャレッド イアコヴェッリ，
ピチョット， シーモア デ
ジィー－ワー ツェ，
ローリー ケニー，
キャサリン オルソン，
ハワード ゲンデルマン，
アール． リー モズレー，
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ModernaTx Inc
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ModernaTx Inc
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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
    • A61K38/19—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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2022522647A 2019-10-15 2020-10-15 パーキンソン病を治療するための顆粒球マクロファージコロニー刺激因子をコードするｍＲＮＡ Pending JP2022552378A (ja)

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 (2)

Publication Number Publication Date
JP2022552378A true JP2022552378A (ja) 2022-12-15
JP2022552378A5 JP2022552378A5 (enExample) 2023-10-24

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JP2022522647A Pending JP2022552378A (ja) 2019-10-15 2020-10-15 パーキンソン病を治療するための顆粒球マクロファージコロニー刺激因子をコードするｍＲＮＡ

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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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008136438A1 (ja) * 2007-04-27 2008-11-13 Kyushu University, National University Corporation 遺伝子治療用ウイルスベクター
JP2010510219A (ja) * 2006-11-17 2010-04-02 バイオジェン・アイデック・エムエイ・インコーポレイテッド アミロイド関連障害を処置するためのコロニー刺激因子の全身投与
WO2019152557A1 (en) * 2018-01-30 2019-08-08 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells

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GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
GB2284210B (en) 1993-11-26 1997-11-12 British Tech Group Translational enhancer DNA from a kinesin gene
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
EP2447370B1 (en) 2001-09-28 2018-07-18 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
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics 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
CA2715078C (en) 2007-09-26 2019-07-23 Intrexon Corporation Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression
EP2288336B8 (en) 2008-04-25 2017-03-22 Northwestern University Nanostructures suitable for sequestering cholesterol
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
KR101766408B1 (ko) 2009-06-10 2017-08-10 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)
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
AU2016253972B2 (en) * 2015-04-27 2020-01-02 Acuitas Therapeutics Inc. Nucleoside-modified RNA for inducing an adaptive immune response
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
JP7194594B2 (ja) 2016-05-18 2022-12-22 モデルナティエックス インコーポレイテッド 免疫調節ポリペプチドをコードするｍＲＮＡの組み合わせ及びその使用

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010510219A (ja) * 2006-11-17 2010-04-02 バイオジェン・アイデック・エムエイ・インコーポレイテッド アミロイド関連障害を処置するためのコロニー刺激因子の全身投与
WO2008136438A1 (ja) * 2007-04-27 2008-11-13 Kyushu University, National University Corporation 遺伝子治療用ウイルスベクター
WO2019152557A1 (en) * 2018-01-30 2019-08-08 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
FRONTIERS IN AGING NEUROSCIENCE, vol. 7, JPN6024044061, 2015, pages 126 - 1, ISSN: 0005575465 *
MOLECULAR NEURODEGENERATION, vol. 13, JPN6024044060, 2018, pages 1 - 17, ISSN: 0005575466 *

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CA3158013A1 (en) 2021-04-22
AU2020366209A1 (en) 2022-05-19
EP4045076A1 (en) 2022-08-24
WO2021076811A1 (en) 2021-04-22
US20240123034A1 (en) 2024-04-18

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