JP2023535225A - 改変ヌクレオチドを含む脂質ナノ粒子 - Google Patents

改変ヌクレオチドを含む脂質ナノ粒子 Download PDF

Info

Publication number
JP2023535225A
JP2023535225A JP2023505388A JP2023505388A JP2023535225A JP 2023535225 A JP2023535225 A JP 2023535225A JP 2023505388 A JP2023505388 A JP 2023505388A JP 2023505388 A JP2023505388 A JP 2023505388A JP 2023535225 A JP2023535225 A JP 2023535225A
Authority
JP
Japan
Prior art keywords
aspects
cancer
lipid nanoparticles
lipid
guanosine
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.)
Pending
Application number
JP2023505388A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023535225A5 (https=
Inventor
ジェイコブ ベクラフト，
ライアン ソーウェル，
ジャスプリート クラナ，
輔 北田
Original Assignee
ストランド セラピューティクス インコーポレイテッド
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 ストランド セラピューティクス インコーポレイテッド filed Critical ストランド セラピューティクス インコーポレイテッド
Publication of JP2023535225A publication Critical patent/JP2023535225A/ja
Publication of JP2023535225A5 publication Critical patent/JP2023535225A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/5434—IL-12
    • 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
    • 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
    • A61K48/0041—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 polymeric
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dispersion Chemistry (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biomedical Technology (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023505388A 2020-07-24 2021-07-26 改変ヌクレオチドを含む脂質ナノ粒子 Pending JP2023535225A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063056382P 2020-07-24 2020-07-24
US63/056,382 2020-07-24
PCT/US2021/043214 WO2022020811A1 (en) 2020-07-24 2021-07-26 Lipidnanoparticle comprising modified nucleotides

Publications (2)

Publication Number Publication Date
JP2023535225A true JP2023535225A (ja) 2023-08-16
JP2023535225A5 JP2023535225A5 (https=) 2024-07-31

Family

ID=77412342

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023505388A Pending JP2023535225A (ja) 2020-07-24 2021-07-26 改変ヌクレオチドを含む脂質ナノ粒子

Country Status (5)

Country Link
US (1) US20230295257A1 (https=)
EP (1) EP4185274A1 (https=)
JP (1) JP2023535225A (https=)
CN (1) CN116710079A (https=)
WO (1) WO2022020811A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3018046A1 (en) * 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CN113966221B (zh) * 2019-03-08 2026-03-31 麻省理工学院 合成溶瘤lnp复制子rna和用于癌症免疫治疗的用途
CN119317430A (zh) * 2022-04-26 2025-01-14 斯特兰德生物科技公司 包含委内瑞拉马脑炎(vee)复制子的脂质纳米颗粒及其用途
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020509016A (ja) * 2017-02-28 2020-03-26 サノフイＳａｎｏｆｉ 治療用ｒｎａ
JP2020520640A (ja) * 2017-05-18 2020-07-16 モデルナティーエックス， インコーポレイテッド 連結したインターロイキン−１２（ｉｌ１２）ポリペプチドをコードするポリヌクレオチド及びその使用

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3773919A (en) 1969-10-23 1973-11-20 Du Pont Polylactide-drug mixtures
US4485045A (en) 1981-07-06 1984-11-27 Research Corporation Synthetic phosphatidyl cholines useful in forming liposomes
US4544545A (en) 1983-06-20 1985-10-01 Trustees University Of Massachusetts Liposomes containing modified cholesterol for organ targeting
CA1327358C (en) 1987-11-17 1994-03-01 Morio Fujiu Fluoro cytidine derivatives
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
CA2309535A1 (en) 1997-11-12 1999-05-20 Brigham And Women's Hospital, Inc. The translation enhancer element of the human amyloid precursor protein gene
US7468275B2 (en) 2000-01-28 2008-12-23 The Scripps Research Institute Synthetic internal ribosome entry sites and methods of identifying same
CA2398790A1 (en) 2000-01-28 2001-08-02 The Scripps Research Institute Methods of identifying synthetic transcriptional and translational regulatory elements, and compositions relating to same
EP2447370B1 (en) 2001-09-28 2018-07-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
WO2004041203A2 (en) 2002-11-04 2004-05-21 Xenoport, Inc. Gemcitabine prodrugs, pharmaceutical compositions and uses thereof
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
EP2479254A1 (en) 2005-08-24 2012-07-25 The Scripps Research Institute Translation enhancer-element dependent vector systems
US7670840B2 (en) 2006-01-05 2010-03-02 The Ohio State University Research Foundation Micro-RNA expression abnormalities of pancreatic, endocrine and acinar tumors
EP1969147B1 (en) 2006-01-05 2014-07-30 The Ohio State University Research Foundation microRNA-based methods for the diagnosis of stomach cancer
EP2522746B1 (en) 2006-03-02 2014-11-12 The Ohio State University Research Foundation MicroRNA expression profile associated with pancreatic cancer
US20090131348A1 (en) 2006-09-19 2009-05-21 Emmanuel Labourier Micrornas differentially expressed in pancreatic diseases and uses thereof
US8252538B2 (en) 2006-11-01 2012-08-28 The Ohio State University MicroRNA expression signature for predicting survival and metastases in hepatocellular carcinoma
WO2008073915A2 (en) 2006-12-08 2008-06-19 Asuragen, Inc. Micrornas differentially expressed in leukemia and uses thereof
WO2008147974A1 (en) 2007-05-23 2008-12-04 University Of South Florida Micro-rnas modulating immunity and inflammation
US20090099034A1 (en) 2007-06-07 2009-04-16 Wisconsin Alumni Research Foundation Reagents and Methods for miRNA Expression Analysis and Identification of Cancer Biomarkers
CN101918594A (zh) 2007-11-30 2010-12-15 俄亥俄州立大学研究基金会 肺癌中外周血中的微rna表达特征谱和靶向
SG10201408162PA (en) 2007-12-11 2015-01-29 Scripps Research Inst Compositions and methods related to mrna translational enhancer elements
EP2260110B1 (en) 2008-02-08 2014-11-12 Asuragen, INC. miRNAs DIFFERENTIALLY EXPRESSED IN LYMPH NODES FROM CANCER PATIENTS
CA2717026A1 (en) 2008-02-28 2009-09-03 The Ohio State University Research Foundation Microrna signatures associated with human chronic lymphocytic leukemia (ccl) and uses thereof
EP2112235A1 (en) 2008-04-24 2009-10-28 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Compositions and methods for microRNA expression profiling of nasopharyngeal carcinoma
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
WO2010018563A2 (en) 2008-08-12 2010-02-18 Rosetta Genomics Ltd. Compositions and methods for the prognosis of lymphoma
EP2358902A1 (en) 2008-12-10 2011-08-24 Universität Regensburg Compositions and methods for micro-rna expression profiling of cancer stem cells
EP2427574A2 (en) 2009-05-08 2012-03-14 The Ohio State University Research Foundation Microrna expression profiling and targeting in chronic obstructive pulmonary disease (copd) lung tissue and methods of use thereof
WO2011076142A1 (en) 2009-12-24 2011-06-30 Fudan University Compositions and methods for microrna expession profiling in plasma of colorectal cancer
WO2011076143A1 (en) 2009-12-24 2011-06-30 Fudan University Compositions and methods for microrna expression profiling of lung cancer
EP2341145A1 (en) 2009-12-30 2011-07-06 febit holding GmbH miRNA fingerprint in the diagnosis of diseases
WO2011094683A2 (en) 2010-01-29 2011-08-04 H. Lee Moffitt Cancer Center And Research Institute, Inc. Method of identifying myelodysplastic syndromes
EP2354246A1 (en) 2010-02-05 2011-08-10 febit holding GmbH miRNA in the diagnosis of ovarian cancer
US20130059015A1 (en) 2010-03-11 2013-03-07 H. Lee Moffitt Cancer Center & Research Institute Human Cancer micro-RNA Expression Profiles Predictive of Chemo-Response
WO2011157294A1 (en) 2010-06-16 2011-12-22 Universita' Degli Studi Di Padova Compositions for use in treating or preventing cancer, breast cancer, lung cancer, ovarian cancer, metastasis, heart failure, cardiac remodelling, dilated cardiomyopathy, autoimmune diseases, or diseases or disorders related thereto
US20130230884A1 (en) 2010-07-16 2013-09-05 John Chaput Methods to Identify Synthetic and Natural RNA Elements that Enhance Protein Translation
KR20120066952A (ko) 2010-12-15 2012-06-25 주식회사 롤팩 7층 구조의 공압출 항균 진공포장 필름 및 그 제조방법
WO2012151212A1 (en) 2011-05-01 2012-11-08 University Of Rochester Multifocal hepatocellular carcinoma microrna expression patterns and uses thereof
EP2705370A2 (en) 2011-05-06 2014-03-12 Xentech Markers for cancer prognosis and therapy and methods of use
DK2732052T3 (en) 2011-07-15 2017-02-20 Leo Pharma As Diagnostic microRNA profiling in cutaneous T cell lymphoma (CTCL)
EP2751292A4 (en) 2011-09-01 2015-05-20 Allegro Diagnostics Corp METHOD AND COMPOSITIONS FOR DETECTING CANCER BASED ON MIRNA EXPRESSION PROFILES
US20140243240A1 (en) 2011-10-26 2014-08-28 Georgetown University microRNA EXPRESSION PROFILING OF THYROID CANCER
WO2013103659A1 (en) 2012-01-04 2013-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Stabilizing rna by incorporating chain-terminating nucleosides at the 3'-terminus
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
EP3053585A1 (en) 2013-12-13 2016-08-10 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
WO2015196118A1 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
ES2880103T3 (es) * 2015-05-21 2021-11-23 Ohio State Innovation Foundation Derivados de benceno-1, 3, 5-tricarboxamida y usos de los mismos
SI3458083T1 (sl) 2016-05-18 2023-03-31 Modernatx, Inc. Polinukleotidi, ki kodirajo interlevkin-12 (IL12) in njihova uporaba

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020509016A (ja) * 2017-02-28 2020-03-26 サノフイＳａｎｏｆｉ 治療用ｒｎａ
JP2020520640A (ja) * 2017-05-18 2020-07-16 モデルナティーエックス， インコーポレイテッド 連結したインターロイキン−１２（ｉｌ１２）ポリペプチドをコードするポリヌクレオチド及びその使用

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NANO LETTERS, vol. Vol.15, pp.8099-8107, JPN6025039261, 2015, ISSN: 0005694471 *

Also Published As

Publication number Publication date
CN116710079A (zh) 2023-09-05
US20230295257A1 (en) 2023-09-21
WO2022020811A1 (en) 2022-01-27
EP4185274A1 (en) 2023-05-31

Similar Documents

Publication Publication Date Title
JP7803621B2 (ja) ヒトサイトメガロウイルスワクチン
US20230295257A1 (en) Lipid nanoparticle comprising modified nucleotides
JP6980780B2 (ja) ヒトサイトメガロウイルスワクチン
JP2024160243A (ja) がんの治療のためのｍＲＮＡ併用療法
US20240166707A1 (en) Expression constructs and uses thereof
JP2018537521A (ja) 広域インフルエンザウイルスワクチン
CN117731769A (zh) 用于水痘带状疱疹病毒(vzv)的核酸疫苗
CA2923029A1 (en) Chimeric polynucleotides
CN114788876A (zh) 治疗糖尿病的mRNA药物制剂及其制备方法与应用
Wei et al. Technological breakthroughs and advancements in the application of mRNA vaccines: a comprehensive exploration and future prospects
US20250152737A1 (en) Lipid nanoparticles comprising venezuelan equine encephalitis (vee) replicon and uses thereof
US20240350621A1 (en) Double stranded mrna vaccines
EA052099B1 (ru) Экспрессионные конструкции и их применения для лечения рака
KR20260046366A (ko) 마이크로 rna 비표적화 센서를 포함하는 폴리뉴클레오타이드 및 이의 용도
JP2026513742A (ja) 合成回路及びその使用
KR20260003656A (ko) 합성 회로 및 이의 용도
AU2024299528A1 (en) Rna-based synthetic circuit for producing engineered immune cells for an extracorporeal cell therapy
WO2026055329A1 (en) Methods and compositions for treating mesothelioma

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240723

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240723

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250925

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20251222

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260324