JP7820909B2 - 化学修飾されたオリゴヌクレオチド - Google Patents

化学修飾されたオリゴヌクレオチド

Info

Publication number
JP7820909B2
JP7820909B2 JP2020506983A JP2020506983A JP7820909B2 JP 7820909 B2 JP7820909 B2 JP 7820909B2 JP 2020506983 A JP2020506983 A JP 2020506983A JP 2020506983 A JP2020506983 A JP 2020506983A JP 7820909 B2 JP7820909 B2 JP 7820909B2
Authority
JP
Japan
Prior art keywords
cells
cell
nucleic acid
rxrna
acid molecule
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
JP2020506983A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020532955A (ja
JP2020532955A5 (https=
Inventor
エリセーエフ，アレクセイ
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.)
Phio Pharmaceuticals Corp
Original Assignee
Phio Pharmaceuticals Corp
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 Phio Pharmaceuticals Corp filed Critical Phio Pharmaceuticals Corp
Publication of JP2020532955A publication Critical patent/JP2020532955A/ja
Publication of JP2020532955A5 publication Critical patent/JP2020532955A5/ja
Priority to JP2023146785A priority Critical patent/JP2024028231A/ja
Application granted granted Critical
Publication of JP7820909B2 publication Critical patent/JP7820909B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020506983A 2017-08-07 2018-08-07 化学修飾されたオリゴヌクレオチド Active JP7820909B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023146785A JP2024028231A (ja) 2017-08-07 2023-09-11 化学修飾されたオリゴヌクレオチド

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762542043P 2017-08-07 2017-08-07
US62/542,043 2017-08-07
US201762558183P 2017-09-13 2017-09-13
US62/558,183 2017-09-13
PCT/US2018/045671 WO2019032619A1 (en) 2017-08-07 2018-08-07 CHEMICALLY MODIFIED OLIGONUCLEOTIDES

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023146785A Division JP2024028231A (ja) 2017-08-07 2023-09-11 化学修飾されたオリゴヌクレオチド

Publications (3)

Publication Number Publication Date
JP2020532955A JP2020532955A (ja) 2020-11-19
JP2020532955A5 JP2020532955A5 (https=) 2021-08-26
JP7820909B2 true JP7820909B2 (ja) 2026-02-26

Family

ID=65271710

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020506983A Active JP7820909B2 (ja) 2017-08-07 2018-08-07 化学修飾されたオリゴヌクレオチド
JP2023146785A Pending JP2024028231A (ja) 2017-08-07 2023-09-11 化学修飾されたオリゴヌクレオチド

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023146785A Pending JP2024028231A (ja) 2017-08-07 2023-09-11 化学修飾されたオリゴヌクレオチド

Country Status (7)

Country Link
US (1) US20200215113A1 (https=)
EP (1) EP3664817A4 (https=)
JP (2) JP7820909B2 (https=)
CN (1) CN111201024A (https=)
AU (1) AU2018313149B2 (https=)
CA (1) CA3070747A1 (https=)
WO (1) WO2019032619A1 (https=)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2746527A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
WO2015084897A2 (en) * 2013-12-02 2015-06-11 Mirimmune, Llc Immunotherapy of cancer
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1（sod1）的核酸分子
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
WO2018195355A1 (en) 2017-04-19 2018-10-25 Rxi Pharmaceuticals Corporation Topical delivery of nucleic acid compounds
CN115135765A (zh) * 2019-11-08 2022-09-30 菲奥医药公司 用于免疫治疗的靶向含布罗莫结构域之蛋白4(brd4)的经化学修饰的寡核苷酸
CN115916348A (zh) 2020-06-18 2023-04-04 基因泰克公司 使用抗tigit抗体和pd-1轴结合拮抗剂的治疗
CN112266911B (zh) * 2020-08-27 2022-07-22 清华大学 核酸分子
CN118871463A (zh) 2021-07-28 2024-10-29 基因泰克公司 用于治疗癌症的方法和组合物
WO2023015264A1 (en) * 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
JP2024529025A (ja) 2021-08-04 2024-08-01 フィオ ファーマシューティカルズ コーポレーション 化学修飾されたオリゴヌクレオチド
TW202321308A (zh) 2021-09-30 2023-06-01 美商建南德克公司 使用抗ｔｉｇｉｔ抗體、抗ｃｄ３８抗體及ｐｄ—１軸結合拮抗劑治療血液癌症的方法
WO2023130021A1 (en) * 2021-12-30 2023-07-06 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides with improved delivery properties
EP4537107A2 (en) 2022-06-07 2025-04-16 Genentech, Inc. Method for determining the efficacy of a lung cancer treatment comprising an anti-pd-l1 antagonist and an anti-tigit antagonist antibody
WO2024064769A1 (en) * 2022-09-21 2024-03-28 Phio Pharmaceuticals Corp. Induction of stem-like activated t cells
WO2025101204A1 (en) * 2023-11-08 2025-05-15 Phio Pharmaceuticals Corp. Intratumoral targeting of pd-1
WO2025117786A1 (en) * 2023-11-28 2025-06-05 Phio Pharmaceuticals Corp. Enhanced combination immune checkpoint inhibition therapy utilizing rnai

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016540511A (ja) 2013-12-02 2016-12-28 ミルイミューン インコーポレイティッド 癌の免疫療法

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
CA2746527A1 (en) * 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
US8691971B2 (en) * 2008-09-23 2014-04-08 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference
US9745574B2 (en) * 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US9957558B2 (en) * 2011-04-28 2018-05-01 Life Technologies Corporation Methods and compositions for multiplex PCR
CA2958200A1 (en) * 2014-08-14 2016-02-18 Novartis Ag Treatment of cancer using a gfr alpha-4 chimeric antigen receptor
WO2016177343A1 (zh) * 2015-05-05 2016-11-10 江苏命码生物科技有限公司 一种新的前体miRNA及其在肿瘤治疗中的应用
EP3349792A1 (en) * 2015-09-14 2018-07-25 Compass Therapeutics LLC Compositions and methods for treating cancer via antagonism of the cd155/tigit pathway and tgf-

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016540511A (ja) 2013-12-02 2016-12-28 ミルイミューン インコーポレイティッド 癌の免疫療法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Cancer Res, 2017年07月01日, 77(13_Supplement):5650
The Journal of Clinical Investigation, 2015, Vol.125, No.5, pp.2046-2058

Also Published As

Publication number Publication date
EP3664817A1 (en) 2020-06-17
AU2018313149B2 (en) 2024-11-14
JP2024028231A (ja) 2024-03-01
WO2019032619A1 (en) 2019-02-14
AU2018313149A1 (en) 2020-03-05
CN111201024A (zh) 2020-05-26
JP2020532955A (ja) 2020-11-19
CA3070747A1 (en) 2019-02-14
EP3664817A4 (en) 2021-09-22
WO2019032619A9 (en) 2020-02-13
US20200215113A1 (en) 2020-07-09

Similar Documents

Publication Publication Date Title
JP7820909B2 (ja) 化学修飾されたオリゴヌクレオチド
JP6983752B2 (ja) スーパーオキシドディスムターゼ１（ｓｏｄ１）を標的とする核酸分子
JP6553001B2 (ja) 皮膚および線維症適用におけるｒｎａ干渉
JP2020114866A (ja) 遺伝子調節アプローチを用いた円形脱毛症の処置方法
US10808247B2 (en) Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
US20160304875A1 (en) Methods for treatment of wound healing utilizing chemically modified oligonucleotides
JP2023501445A (ja) 免疫治療のためのブロモドメイン含有タンパク質４（ｂｒｄ４）を標的とする化学修飾されたオリゴヌクレオチド
WO2023130021A1 (en) Chemically modified oligonucleotides with improved delivery properties
US20240301430A1 (en) Chemically modified oligonucleotides
US20230089478A1 (en) Chemically modified oligonucleotides with improved systemic delivery
WO2023015264A1 (en) Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
US20260097120A1 (en) Induction of stem-like activated t cells
WO2025117786A1 (en) Enhanced combination immune checkpoint inhibition therapy utilizing rnai
WO2025101204A1 (en) Intratumoral targeting of pd-1

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210714

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210714

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220812

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221111

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230213

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230511

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230911

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230913

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20231016

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20231215

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251127

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20260213

R150 Certificate of patent or registration of utility model

Ref document number: 7820909

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150