JP2023526059A - ｍＲＮＡ治療薬及びエフェクター分子を含むＬＮＰ組成物 - Google Patents

ｍＲＮＡ治療薬及びエフェクター分子を含むＬＮＰ組成物 Download PDF

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Publication number
JP2023526059A
JP2023526059A JP2022568830A JP2022568830A JP2023526059A JP 2023526059 A JP2023526059 A JP 2023526059A JP 2022568830 A JP2022568830 A JP 2022568830A JP 2022568830 A JP2022568830 A JP 2022568830A JP 2023526059 A JP2023526059 A JP 2023526059A
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Prior art keywords
lnp
polynucleotide
lnp composition
cell
composition
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Japanese (ja)
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JP2023526059A5 (https=
JPWO2021231854A5 (https=
Inventor
ジャイン，ルチ
アン ビックネル，アリシア
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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
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1273—Polymersomes; Liposomes with polymerisable or polymerised bilayer-forming substances
    • 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
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Virology (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
JP2022568830A 2020-05-14 2021-05-14 ｍＲＮＡ治療薬及びエフェクター分子を含むＬＮＰ組成物 Pending JP2023526059A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063024862P 2020-05-14 2020-05-14
US63/024,862 2020-05-14
US202163183119P 2021-05-03 2021-05-03
US63/183,119 2021-05-03
PCT/US2021/032438 WO2021231854A1 (en) 2020-05-14 2021-05-14 Lnp compositions comprising an mrna therapeutic and an effector molecule

Publications (3)

Publication Number Publication Date
JP2023526059A true JP2023526059A (ja) 2023-06-20
JP2023526059A5 JP2023526059A5 (https=) 2024-05-24
JPWO2021231854A5 JPWO2021231854A5 (https=) 2024-05-24

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JP2022568830A Pending JP2023526059A (ja) 2020-05-14 2021-05-14 ｍＲＮＡ治療薬及びエフェクター分子を含むＬＮＰ組成物

Country Status (6)

Country Link
US (1) US20230173104A1 (https=)
EP (1) EP4149435A1 (https=)
JP (1) JP2023526059A (https=)
AU (1) AU2021270587A1 (https=)
CA (1) CA3182920A1 (https=)
WO (1) WO2021231854A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20260035707A1 (en) * 2022-07-26 2026-02-05 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
CN115869332A (zh) * 2022-10-27 2023-03-31 北京新合睿恩生物医疗科技有限公司 递送至体内后在肝脏少表达的mRNA药物及其制备方法
EP4637786A2 (en) * 2022-12-22 2025-10-29 ModernaTX, Inc. Small nuclear ribonucleoprotein 13 polypeptides, polynucleotides, and uses thereof
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025116441A1 (ko) * 2023-11-30 2025-06-05 주식회사 레나임 골관절염의 예방 또는 치료용 약제학적 조성물
WO2025255510A1 (en) * 2024-06-07 2025-12-11 The University Of Chicago Systems and methods for modulating rna

Citations (4)

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Publication number Priority date Publication date Assignee Title
WO2013013013A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for producing modified glycoproteins
WO2019092145A1 (en) * 2017-11-08 2019-05-16 Evox Therapeutics Ltd Exosomes comprising rna therapeutics
JP2019519517A (ja) * 2016-05-18 2019-07-11 モデルナティーエックス， 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
WO2020056304A1 (en) * 2018-09-14 2020-03-19 Modernatx, Inc. Methods and compositions for treating cancer using mrna therapeutics

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GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
JPH09505474A (ja) 1993-11-26 1997-06-03 ブリテイツシユ・テクノロジー・グループ・リミテツド 翻訳エンハンサーｄｎａ
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
EP2390331B1 (en) 2001-09-28 2018-04-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
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
DK2205618T3 (en) 2007-09-26 2017-02-20 Intrexon Corp SYNTHETIC 5 'NON-TRANSLATED REGIONS, EXPRESSION VECTORS AND PROCEDURES FOR INCREASING TRANSGENIC EXPRESSION
CA2722183C (en) 2008-04-25 2018-09-18 Northwestern University Nanostructures suitable for sequestering cholesterol and other molecules
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
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
MX342785B (es) 2009-06-10 2016-10-12 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
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WO2013013013A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for producing modified glycoproteins
JP2019519517A (ja) * 2016-05-18 2019-07-11 モデルナティーエックス， 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
WO2019092145A1 (en) * 2017-11-08 2019-05-16 Evox Therapeutics Ltd Exosomes comprising rna therapeutics
WO2020056304A1 (en) * 2018-09-14 2020-03-19 Modernatx, Inc. Methods and compositions for treating cancer using mrna therapeutics

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CA3182920A1 (en) 2021-11-18
EP4149435A1 (en) 2023-03-22
AU2021270587A1 (en) 2022-12-08
US20230173104A1 (en) 2023-06-08
WO2021231854A1 (en) 2021-11-18

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