JP7561129B2 - Ｐｍｐ２２の発現を低減するための化合物及び方法 - Google Patents

Ｐｍｐ２２の発現を低減するための化合物及び方法 Download PDF

Info

Publication number
JP7561129B2
JP7561129B2 JP2021535612A JP2021535612A JP7561129B2 JP 7561129 B2 JP7561129 B2 JP 7561129B2 JP 2021535612 A JP2021535612 A JP 2021535612A JP 2021535612 A JP2021535612 A JP 2021535612A JP 7561129 B2 JP7561129 B2 JP 7561129B2
Authority
JP
Japan
Prior art keywords
modified
certain embodiments
seq
modified oligonucleotide
oligonucleotide
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
JP2021535612A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022515140A5 (https=
JP2022515140A (ja
JPWO2020132558A5 (https=
Inventor
ブイ，フィン・ホア
フレアー，スーザン・エム
チャオ，ヒエン・トイ
シング，プリヤム
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.)
Ionis Pharmaceuticals Inc
Original Assignee
Ionis Pharmaceuticals 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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of JP2022515140A publication Critical patent/JP2022515140A/ja
Publication of JP2022515140A5 publication Critical patent/JP2022515140A5/ja
Publication of JPWO2020132558A5 publication Critical patent/JPWO2020132558A5/ja
Priority to JP2024163353A priority Critical patent/JP2025000717A/ja
Application granted granted Critical
Publication of JP7561129B2 publication Critical patent/JP7561129B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/341—Gapmers, i.e. of the type ===---===

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2021535612A 2018-12-21 2019-12-20 Ｐｍｐ２２の発現を低減するための化合物及び方法 Active JP7561129B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024163353A JP2025000717A (ja) 2018-12-21 2024-09-20 Ｐｍｐ２２の発現を低減するための化合物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862783997P 2018-12-21 2018-12-21
US62/783,997 2018-12-21
PCT/US2019/068040 WO2020132558A1 (en) 2018-12-21 2019-12-20 Compounds and methods for reducing pmp22 expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024163353A Division JP2025000717A (ja) 2018-12-21 2024-09-20 Ｐｍｐ２２の発現を低減するための化合物及び方法

Publications (4)

Publication Number Publication Date
JP2022515140A JP2022515140A (ja) 2022-02-17
JP2022515140A5 JP2022515140A5 (https=) 2022-12-14
JPWO2020132558A5 JPWO2020132558A5 (https=) 2022-12-14
JP7561129B2 true JP7561129B2 (ja) 2024-10-03

Family

ID=71101859

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021535612A Active JP7561129B2 (ja) 2018-12-21 2019-12-20 Ｐｍｐ２２の発現を低減するための化合物及び方法
JP2024163353A Pending JP2025000717A (ja) 2018-12-21 2024-09-20 Ｐｍｐ２２の発現を低減するための化合物及び方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024163353A Pending JP2025000717A (ja) 2018-12-21 2024-09-20 Ｐｍｐ２２の発現を低減するための化合物及び方法

Country Status (4)

Country Link
US (1) US20220112503A1 (https=)
EP (1) EP3897837A4 (https=)
JP (2) JP7561129B2 (https=)
WO (1) WO2020132558A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023530072A (ja) * 2020-06-19 2023-07-13 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｐｍｐ２２を調節するための化合物及び方法

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
BR112020013994A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
AU2021390471A1 (en) * 2020-12-01 2023-06-29 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of peripheral myelin protein-22

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017156242A1 (en) 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030198983A1 (en) * 2002-02-01 2003-10-23 Affymetrix, Inc. Methods of genetic analysis of human genes
EP1646874B1 (en) * 2003-07-11 2010-03-03 ZEILLINGER, Robert A method for predicting the metastatic potential of breast cancer
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
PT3277815T (pt) * 2015-04-03 2021-11-11 Beth Israel Deaconess Medical Ct Inc Compostos de oligonucleótidos para o tratamento de pré-eclâmpsia e outros distúrbios angiogénicos
JP7054675B2 (ja) * 2015-12-14 2022-04-14 コールド スプリング ハーバー ラボラトリー 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法
KR101889072B1 (ko) * 2017-09-15 2018-08-16 한국생명공학연구원 디지털 PCR을 이용한 유전성 강직성 대마비(Hereditary spastic paraplegia, HSP) 관련 유전자 SPG4의 거대결손 검증법

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017156242A1 (en) 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023530072A (ja) * 2020-06-19 2023-07-13 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｐｍｐ２２を調節するための化合物及び方法

Also Published As

Publication number Publication date
JP2025000717A (ja) 2025-01-07
WO2020132558A1 (en) 2020-06-25
JP2022515140A (ja) 2022-02-17
EP3897837A1 (en) 2021-10-27
EP3897837A4 (en) 2023-08-16
US20220112503A1 (en) 2022-04-14

Similar Documents

Publication Publication Date Title
JP7678033B2 (ja) Ｌｒｒｋ２発現を低減するための化合物及び方法
JP7411632B2 (ja) Ａｔｘｎ２発現を低減するための化合物及び方法
JP7646616B2 (ja) Ｓｎｃａ発現を低下させるための化合物及び方法
JP7780564B2 (ja) プリオン発現を低減するための化合物及び方法
JP6738004B2 (ja) タウ発現を低減するための化合物及び方法
JP7557378B2 (ja) Ｓｔｍｎ２発現を増加させるための化合物及び方法
JP7557469B2 (ja) Ａｐｐの発現を低減するための化合物及び方法
JP2021530241A5 (https=)
JP7550165B2 (ja) Ｕｂｅ３ａ－ａｔｓを調節するための化合物及び方法
JP7696466B2 (ja) Ｓｍｎ２を調節するための化合物及び方法
JP7561129B2 (ja) Ｐｍｐ２２の発現を低減するための化合物及び方法
JP7564817B2 (ja) ｋｃｎｔ１発現を低減するための化合物及び方法
JP2023536472A (ja) Ａｐｐの発現を低減するための化合物及び方法
JPWO2020023737A5 (https=)
JP2026012176A (ja) Ａｔｘｎ１を調節するための化合物及び方法
JP2024523363A (ja) Ｉｆｎａｒ１の発現を低減するための化合物及び方法
JP2026062868A (ja) Ｐｌｐ１を調節するための化合物及び方法
JP2023543215A (ja) Ａｐｏｅの発現を低減するための化合物及び方法
JP2023528755A (ja) Ｍｓｈ３の発現を低減するための化合物及び方法
JP2023530072A (ja) Ｐｍｐ２２を調節するための化合物及び方法
JP2023533153A (ja) Ｋｃｎｔ１の発現を低減するための化合物及び方法
EA048039B1 (ru) Соединения и способы модулирования smn2
EA044034B1 (ru) Соединения и способы для снижения экспрессии kcnt1
EA044985B1 (ru) Соединения и способы для снижения экспрессии atxn2

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221206

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20221206

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20231115

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231127

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240219

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240524

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20240524

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20240821

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240920

R150 Certificate of patent or registration of utility model

Ref document number: 7561129

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150