JP2023524071A - 嚢胞性線維症を治療するための核酸及び方法 - Google Patents

嚢胞性線維症を治療するための核酸及び方法 Download PDF

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JP2023524071A
JP2023524071A JP2022566343A JP2022566343A JP2023524071A JP 2023524071 A JP2023524071 A JP 2023524071A JP 2022566343 A JP2022566343 A JP 2022566343A JP 2022566343 A JP2022566343 A JP 2022566343A JP 2023524071 A JP2023524071 A JP 2023524071A
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mrna
lipid
seq
mol
cftr
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JP2023524071A5 (https=
Inventor
ジー ペレス－ガルシア，カルロス
清 立川
大輝 松田
チブクラ，パドマナブ
プラカシュ カルマリ，プリヤ
バオ，ヤンジエ
ボイド リー ベガ，ジェレル
ムクタバーラム，ラジェシュ
サギ，アミット
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Arcturus Therapeutics Inc
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Arcturus Therapeutics Inc
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Publication of JP2023524071A publication Critical patent/JP2023524071A/ja
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    • 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
    • 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
    • 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
    • A61K9/0043—Nose
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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

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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)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pulmonology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Otolaryngology (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2022566343A 2020-05-01 2021-04-30 嚢胞性線維症を治療するための核酸及び方法 Pending JP2023524071A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063019170P 2020-05-01 2020-05-01
US63/019,170 2020-05-01
US202063082997P 2020-09-24 2020-09-24
US63/082,997 2020-09-24
PCT/US2021/030257 WO2021222801A2 (en) 2020-05-01 2021-04-30 Nucleic acids and methods of treatment for cystic fibrosis

Publications (2)

Publication Number Publication Date
JP2023524071A true JP2023524071A (ja) 2023-06-08
JP2023524071A5 JP2023524071A5 (https=) 2024-05-10

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Country Link
US (2) US12070509B2 (https=)
EP (1) EP4143217A4 (https=)
JP (1) JP2023524071A (https=)
AU (1) AU2021263590A1 (https=)
CA (1) CA3176844A1 (https=)
NZ (1) NZ794954A (https=)
WO (1) WO2021222801A2 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3458108A4 (en) 2016-05-18 2020-04-22 ModernaTX, Inc. POLYNUCLEOTIDES ENCODING A CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
CA3176844A1 (en) * 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
EP4208549A4 (en) 2020-09-04 2025-04-16 Verve Therapeutics, Inc. Compositions and methods for capping RNAs
WO2022115645A1 (en) 2020-11-25 2022-06-02 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
JP2024511463A (ja) * 2021-03-22 2024-03-13 リコード セラピューティクス，インク． 細胞への標的送達のための組成物および方法
CA3213107A1 (en) 2021-03-23 2022-09-29 Mirko HENNIG Polynucleotide compositions, related formulations, and methods of use thereof
EP4334330A1 (en) 2021-05-07 2024-03-13 Helix Nanotechnologies, Inc. Modified ribonucleic acids and uses thereof
US20240398940A1 (en) 2021-09-03 2024-12-05 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
CA3256897A1 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for the administration of nucleic acids and their methods of use
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2024089229A1 (en) 2022-10-28 2024-05-02 CureVac SE Improved formulations comprising lipid-based carriers encapsulating rna
WO2024184500A1 (en) 2023-03-08 2024-09-12 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
US20250144237A1 (en) * 2023-11-07 2025-05-08 Cila Therapeutics Inc. Agents, compositions and methods for enhanced delivery of nucleic acids to the cells within the respiratory system
WO2025103803A1 (en) 2023-11-13 2025-05-22 CureVac SE Immunotherapy against neuronal and brain tumors
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
WO2026072721A1 (en) * 2024-09-25 2026-04-02 Carbon Biosciences, Inc. Parvovirus compositions and methods for gene therapy

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018119163A1 (en) * 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US20180256741A1 (en) * 2017-02-27 2018-09-13 Translate Bio, Inc. Novel Codon-Optimized CFTR MRNA

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS58500845A (ja) 1981-06-04 1983-05-26 デラウェア・ケミカルス・コーポレーション ポンプ
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
ES2061416T3 (es) 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
KR20040051450A (ko) * 2002-12-12 2004-06-18 주식회사 인비보사이언스 알엔에이의 생체내 안정성을 증가시키는 벡터
US8304529B2 (en) 2006-07-28 2012-11-06 Life Technologies Corporation Dinucleotide MRNA cap analogs
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
KR20190039347A (ko) 2010-06-03 2019-04-10 알닐람 파마슈티칼스 인코포레이티드 활성제의 전달을 위한 생분해성 지질
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
RS57739B1 (sr) 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのｍＲＮＡ療法
AU2014348212C1 (en) * 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
AU2015338923B2 (en) 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
CN120842296A (zh) 2015-09-21 2025-10-28 垂林克生物技术有限公司 用于合成5’-加帽rna的组合物和方法
US10487105B2 (en) 2016-10-19 2019-11-26 Arcturus Therapeutics, Inc. Trinucleotide MRNA cap analogs
US11407800B2 (en) 2017-02-28 2022-08-09 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
ES3039647T3 (en) 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
CA3176844A1 (en) * 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
AU2022381750A1 (en) * 2021-11-03 2024-05-16 Arcturus Therapeutics, Inc. Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018119163A1 (en) * 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US20180256741A1 (en) * 2017-02-27 2018-09-13 Translate Bio, Inc. Novel Codon-Optimized CFTR MRNA

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US12070509B2 (en) 2024-08-27
WO2021222801A3 (en) 2021-12-09
WO2021222801A2 (en) 2021-11-04
NZ794954A (en) 2026-03-27
EP4143217A2 (en) 2023-03-08
US20250049950A1 (en) 2025-02-13
CA3176844A1 (en) 2021-11-04
AU2021263590A1 (en) 2023-01-19
US20220023442A1 (en) 2022-01-27
EP4143217A4 (en) 2024-10-02

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