CA3070747A1 - Chemically modified oligonucleotides - Google Patents

Chemically modified oligonucleotides Download PDF

Info

Publication number
CA3070747A1
CA3070747A1 CA3070747A CA3070747A CA3070747A1 CA 3070747 A1 CA3070747 A1 CA 3070747A1 CA 3070747 A CA3070747 A CA 3070747A CA 3070747 A CA3070747 A CA 3070747A CA 3070747 A1 CA3070747 A1 CA 3070747A1
Authority
CA
Canada
Prior art keywords
cell
nucleic acid
chemically
double stranded
rxrna
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
CA3070747A
Other languages
English (en)
French (fr)
Inventor
Alexey Eliseev
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 CA3070747A1 publication Critical patent/CA3070747A1/en
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic 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)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3070747A 2017-08-07 2018-08-07 Chemically modified oligonucleotides Pending CA3070747A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3070747A1 true CA3070747A1 (en) 2019-02-14

Family

ID=65271710

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3070747A Pending CA3070747A1 (en) 2017-08-07 2018-08-07 Chemically modified oligonucleotides

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
EP2342340A1 (en) 2008-09-22 2011-07-13 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
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
CN106061488B (zh) * 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
US10900039B2 (en) 2014-09-05 2021-01-26 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1
EP3862005A1 (en) 2015-07-06 2021-08-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
US12544344B2 (en) 2017-04-19 2026-02-10 Phio Pharmaceuticals Corp. 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
KR20230024368A (ko) 2020-06-18 2023-02-20 제넨테크, 인크. 항-tigit 항체 및 pd-1 축 결합 길항제를 사용한 치료
CN112266911B (zh) * 2020-08-27 2022-07-22 清华大学 核酸分子
WO2023010094A2 (en) 2021-07-28 2023-02-02 Genentech, Inc. Methods and compositions for treating cancer
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
WO2023015264A1 (en) * 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
WO2023056403A1 (en) 2021-09-30 2023-04-06 Genentech, Inc. Methods for treatment of hematologic cancers using anti-tigit antibodies, anti-cd38 antibodies, and pd-1 axis binding antagonists
WO2023130021A1 (en) * 2021-12-30 2023-07-06 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides with improved delivery properties
KR20250022049A (ko) 2022-06-07 2025-02-14 제넨테크, 인크. 항-pd-l1 길항제 및 항-tigit 길항제 항체를 포함하는, 폐암 치료의 효율을 결정하는 방법
US20260097120A1 (en) * 2022-09-21 2026-04-09 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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
EP2342340A1 (en) * 2008-09-22 2011-07-13 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
EP3495497B1 (en) * 2011-04-28 2021-03-24 Life Technologies Corporation Methods and compositions for multiplex pcr
CN106061488B (zh) * 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
AU2015301460B2 (en) * 2014-08-14 2021-04-08 Novartis Ag Treatment of cancer using GFR alpha-4 chimeric antigen receptor
CN107921147B (zh) * 2015-05-05 2021-12-24 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-

Also Published As

Publication number Publication date
AU2018313149B2 (en) 2024-11-14
CN111201024A (zh) 2020-05-26
EP3664817A4 (en) 2021-09-22
JP2020532955A (ja) 2020-11-19
JP7820909B2 (ja) 2026-02-26
WO2019032619A1 (en) 2019-02-14
AU2018313149A1 (en) 2020-03-05
WO2019032619A9 (en) 2020-02-13
EP3664817A1 (en) 2020-06-17
JP2024028231A (ja) 2024-03-01
US20200215113A1 (en) 2020-07-09

Similar Documents

Publication Publication Date Title
AU2018313149B2 (en) Chemically modified oligonucleotides
AU2018202014B2 (en) Rna interference in dermal and fibrotic indications
JP2018502837A (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
CA2991598A1 (en) Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
CA2932753A1 (en) Methods for treatment of wound healing utilizing chemically modified oligonucleotides
WO2017007825A1 (en) Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
US20230002766A1 (en) Chemically modified oligonucleotides targeting bromodomain containing protein 4 (brd4) for immunotherapy
WO2023130021A1 (en) Chemically modified oligonucleotides with improved delivery properties
US20240301430A1 (en) Chemically modified oligonucleotides
WO2023015264A1 (en) Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
US20230089478A1 (en) Chemically modified oligonucleotides with improved systemic delivery
US20260097120A1 (en) Induction of stem-like activated t cells

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220914

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240802

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20240802

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20240802

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20241002

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20250130

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250220

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250521

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20250521

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250801

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250829