JP7674077B2 - 翻訳可能分子およびその合成 - Google Patents

翻訳可能分子およびその合成 Download PDF

Info

Publication number
JP7674077B2
JP7674077B2 JP2019546855A JP2019546855A JP7674077B2 JP 7674077 B2 JP7674077 B2 JP 7674077B2 JP 2019546855 A JP2019546855 A JP 2019546855A JP 2019546855 A JP2019546855 A JP 2019546855A JP 7674077 B2 JP7674077 B2 JP 7674077B2
Authority
JP
Japan
Prior art keywords
mrna
template
translatable
rna
molecules
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.)
Active
Application number
JP2019546855A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020510426A5 (enExample
JP2020510426A (ja
Inventor
リンフォン パトララニー・
キヨシ・タチカワ
パドマナブ・チブクラ
ダイキ・マツダ
アリサ・ケイル
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.)
Arcturus Therapeutics Inc
Original Assignee
Arcturus Therapeutics Inc
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 Arcturus Therapeutics Inc filed Critical Arcturus Therapeutics Inc
Publication of JP2020510426A publication Critical patent/JP2020510426A/ja
Publication of JP2020510426A5 publication Critical patent/JP2020510426A5/ja
Priority to JP2022209557A priority Critical patent/JP2023052106A/ja
Application granted granted Critical
Publication of JP7674077B2 publication Critical patent/JP7674077B2/ja
Priority to JP2025159744A priority patent/JP2026016392A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/505—Erythropoietin [EPO]
    • 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
    • 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
    • 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
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • C07K14/575—Hormones
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2019546855A 2017-02-28 2018-02-27 翻訳可能分子およびその合成 Active JP7674077B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022209557A JP2023052106A (ja) 2017-02-28 2022-12-27 翻訳可能分子およびその合成
JP2025159744A JP2026016392A (ja) 2017-02-28 2025-09-26 翻訳可能分子およびその合成

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762465073P 2017-02-28 2017-02-28
US62/465,073 2017-02-28
PCT/US2018/020018 WO2018160592A1 (en) 2017-02-28 2018-02-27 Translatable molecules and synthesis thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022209557A Division JP2023052106A (ja) 2017-02-28 2022-12-27 翻訳可能分子およびその合成

Publications (3)

Publication Number Publication Date
JP2020510426A JP2020510426A (ja) 2020-04-09
JP2020510426A5 JP2020510426A5 (enExample) 2021-04-15
JP7674077B2 true JP7674077B2 (ja) 2025-05-09

Family

ID=63370513

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019546855A Active JP7674077B2 (ja) 2017-02-28 2018-02-27 翻訳可能分子およびその合成
JP2022209557A Pending JP2023052106A (ja) 2017-02-28 2022-12-27 翻訳可能分子およびその合成
JP2025159744A Pending JP2026016392A (ja) 2017-02-28 2025-09-26 翻訳可能分子およびその合成

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022209557A Pending JP2023052106A (ja) 2017-02-28 2022-12-27 翻訳可能分子およびその合成
JP2025159744A Pending JP2026016392A (ja) 2017-02-28 2025-09-26 翻訳可能分子およびその合成

Country Status (4)

Country Link
US (2) US11407800B2 (enExample)
EP (1) EP3589290A4 (enExample)
JP (3) JP7674077B2 (enExample)
WO (1) WO2018160592A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3589290A4 (en) 2017-02-28 2020-12-30 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
CN110331147A (zh) * 2019-06-21 2019-10-15 苏州吉玛基因股份有限公司 一种mRNA的制备方法及其在肿瘤治疗中的应用
EP4114470A4 (en) 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
JP2023517644A (ja) 2020-03-09 2023-04-26 アークトゥラス・セラピューティクス・インコーポレイテッド コロナウイルスワクチン組成物及び方法
WO2021222801A2 (en) 2020-05-01 2021-11-04 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
US12178921B2 (en) 2020-08-14 2024-12-31 Arcturus Therapeutics, Inc. Method of lyophilizing lipid nanoparticles
CA3128660A1 (en) * 2020-10-09 2022-04-09 Eric G. Marcusson Compositions and methods for the prevention and/or treatment of covid-19
CN113372432A (zh) * 2021-06-15 2021-09-10 深圳市臻质医疗科技有限公司 一种基于化学修饰mRNA编码蛋白因子诱导和/或增强软骨损伤修复的方法
KR102785258B1 (ko) * 2022-09-07 2025-03-26 아이진 주식회사 변형핵산 함유 mRNA의 체내 전달용 조성물
WO2024054020A1 (ko) * 2022-09-07 2024-03-14 아이진 주식회사 변형핵산 함유 mrna의 체내 전달용 조성물

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015534817A (ja) 2012-11-01 2015-12-07 ファクター バイオサイエンス インコーポレイテッド 細胞中でタンパク質を発現するための方法および生成物
JP2015535430A (ja) 2012-11-26 2015-12-14 モデルナ セラピューティクス インコーポレイテッドＭｏ 末端修飾ｒｎａ
US20160237134A1 (en) 2014-11-10 2016-08-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
JP2016527908A (ja) 2013-08-21 2016-09-15 キュアバック アーゲー Ｒｎａコードされたタンパク質の発現を促進するための方法

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE291925T1 (de) 2001-06-05 2005-04-15 Curevac Gmbh Stabilisierte mrna mit erhöhtem g/c-gehalt und optimierter codon usage für die gentherapie
US7659391B2 (en) 2001-11-05 2010-02-09 Janssen Pharmaceutica N.V. Method for the in vitro synthesis of short double stranded RNAs
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
EP2049665A2 (en) 2006-07-28 2009-04-22 Applera Corporation Dinucleotide mrna cap analogs
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
US20090226906A1 (en) * 2008-03-05 2009-09-10 Helicos Biosciences Corporation Methods and compositions for reducing nucleotide impurities
PT3682905T (pt) * 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9283287B2 (en) * 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
GB201403096D0 (en) 2014-02-21 2014-04-09 Oxford Nanopore Tech Ltd Sample preparation method
WO2016070166A2 (en) * 2014-11-02 2016-05-06 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
EP4324473A3 (en) * 2014-11-10 2024-05-29 ModernaTX, Inc. Multiparametric nucleic acid optimization
US20190382774A1 (en) * 2016-05-18 2019-12-19 Modernatx, Inc. Polyribonucleotides containing reduced uracil content and uses thereof
EP3589290A4 (en) 2017-02-28 2020-12-30 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015534817A (ja) 2012-11-01 2015-12-07 ファクター バイオサイエンス インコーポレイテッド 細胞中でタンパク質を発現するための方法および生成物
JP2015535430A (ja) 2012-11-26 2015-12-14 モデルナ セラピューティクス インコーポレイテッドＭｏ 末端修飾ｒｎａ
JP2016527908A (ja) 2013-08-21 2016-09-15 キュアバック アーゲー Ｒｎａコードされたタンパク質の発現を促進するための方法
US20160237134A1 (en) 2014-11-10 2016-08-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Bioconjugate Chemistry, 2016年, 発行日, Vol.27, pp.849-853

Also Published As

Publication number Publication date
WO2018160592A1 (en) 2018-09-07
JP2026016392A (ja) 2026-02-03
JP2020510426A (ja) 2020-04-09
JP2023052106A (ja) 2023-04-11
US11407800B2 (en) 2022-08-09
EP3589290A1 (en) 2020-01-08
US20230059111A1 (en) 2023-02-23
US11939363B2 (en) 2024-03-26
US20180327471A1 (en) 2018-11-15
EP3589290A4 (en) 2020-12-30

Similar Documents

Publication Publication Date Title
JP6728156B2 (ja) メッセンジャーｕｎａ分子およびその使用
US11939363B2 (en) Translatable molecules and synthesis thereof
ES3039647T3 (en) Synthesis and structure of high potency rna therapeutics
ES2542015T3 (es) Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias
ES2576126T3 (es) Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
US20230235364A1 (en) Methods and means for genetic alteration of genomes utilizing designer dna recombining enzymes
US20230265461A1 (en) Compositions and methods for targeting, editing, or modifying genes
US20240011054A1 (en) Constructs and uses thereof for efficient and specific genome editing
US20180353622A1 (en) Gene deletion and rescue by crispr-mediated elimination of exon splicing enhancers
WO2020097360A1 (en) Methods and compositions for genome-wide analysis and use of genome cutting and repair
US20240100136A1 (en) Chimeric adaptor proteins and methods of regulating gene expression
WO2024166035A1 (en) Regulating gene expression in gamma delta t cell receptors expressing cells
EP4665361A1 (en) Regulating gene expression in gamma delta t cell receptors expressing cells
US20180179502A1 (en) Nucleic Acid Analogue-Guided Chemical Nuclease System, Methods and Compositions

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210225

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210225

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220215

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220511

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20220830

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20221227

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20221227

C116 Written invitation by the chief administrative judge to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C116

Effective date: 20230124

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20230124

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20240502

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240926

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20241028

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241226

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20250217

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250424

R150 Certificate of patent or registration of utility model

Ref document number: 7674077

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150