JP2024506709A - Ｕｂｅ３ａアンチセンス治療剤 - Google Patents

Ｕｂｅ３ａアンチセンス治療剤 Download PDF

Info

Publication number
JP2024506709A
JP2024506709A JP2023549575A JP2023549575A JP2024506709A JP 2024506709 A JP2024506709 A JP 2024506709A JP 2023549575 A JP2023549575 A JP 2023549575A JP 2023549575 A JP2023549575 A JP 2023549575A JP 2024506709 A JP2024506709 A JP 2024506709A
Authority
JP
Japan
Prior art keywords
ube3a
aso
composition
bases
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.)
Pending
Application number
JP2023549575A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024506709A5 (enExample
Inventor
ジェイムズ フィンク，
ルイス ウィリアムズ，
ケイトリン リワーク，
デイビッド ガーバー，
ダンカン ブラウン，
スディール アグラワル，
グラハム ティー． デンプシー，
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.)
Q State Biosciences Inc
Original Assignee
Q State Biosciences 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 Q State Biosciences Inc filed Critical Q State Biosciences Inc
Publication of JP2024506709A publication Critical patent/JP2024506709A/ja
Publication of JP2024506709A5 publication Critical patent/JP2024506709A5/ja
Pending legal-status Critical Current

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
    • C12N15/1137—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 enzymes
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • C12N2310/32—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
    • C12N2310/33—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 ===---===
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023549575A 2021-02-17 2022-02-17 Ｕｂｅ３ａアンチセンス治療剤 Pending JP2024506709A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163150188P 2021-02-17 2021-02-17
US63/150,188 2021-02-17
PCT/US2022/016842 WO2022178160A1 (en) 2021-02-17 2022-02-17 Ube3a antisense therapeutics

Publications (2)

Publication Number Publication Date
JP2024506709A true JP2024506709A (ja) 2024-02-14
JP2024506709A5 JP2024506709A5 (enExample) 2025-02-25

Family

ID=82802019

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023549575A Pending JP2024506709A (ja) 2021-02-17 2022-02-17 Ｕｂｅ３ａアンチセンス治療剤

Country Status (5)

Country Link
US (1) US20220259601A1 (enExample)
EP (1) EP4294925A1 (enExample)
JP (1) JP2024506709A (enExample)
CA (1) CA3211396A1 (enExample)
WO (1) WO2022178160A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025128816A1 (en) * 2023-12-13 2025-06-19 Kicho Inc. Methods of modulating expression of ube3a gene
WO2025128853A2 (en) 2023-12-13 2025-06-19 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with ube3a overexpression
WO2025132962A2 (en) * 2023-12-21 2025-06-26 F. Hoffmann-La Roche Ag Antisense oligonucleotide

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5858987A (en) * 1995-05-05 1999-01-12 Mitotix, Inc. E6AP antisense constructs and methods of use
JP2010507579A (ja) * 2006-10-18 2010-03-11 アイシス ファーマシューティカルズ， インコーポレーテッド アンチセンス化合物
JP2010522547A (ja) * 2007-03-26 2010-07-08 ノバルティス アーゲー ＨＰＶ感染を処置するためのｄｓＤＮＡ組成物および方法
WO2020205463A1 (en) * 2019-03-29 2020-10-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating ube3a-ats

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6617442B1 (en) * 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US20150322428A1 (en) * 2012-06-18 2015-11-12 Isis Pharmaceuticals, Inc. Compounds and methods for improved cellular uptake of antisense compounds
US11077132B2 (en) * 2015-02-04 2021-08-03 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5858987A (en) * 1995-05-05 1999-01-12 Mitotix, Inc. E6AP antisense constructs and methods of use
JP2010507579A (ja) * 2006-10-18 2010-03-11 アイシス ファーマシューティカルズ， インコーポレーテッド アンチセンス化合物
JP2010522547A (ja) * 2007-03-26 2010-07-08 ノバルティス アーゲー ＨＰＶ感染を処置するためのｄｓＤＮＡ組成物および方法
WO2020205463A1 (en) * 2019-03-29 2020-10-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating ube3a-ats

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CANCERS, vol. Vol.12, No.8, Article No.2108, JPN6025049729, 2020, pages 1 - 19, ISSN: 0005745983 *

Also Published As

Publication number Publication date
EP4294925A1 (en) 2023-12-27
US20220259601A1 (en) 2022-08-18
WO2022178160A1 (en) 2022-08-25
CA3211396A1 (en) 2022-08-25

Similar Documents

Publication Publication Date Title
US20250092403A1 (en) Compositions and methods for inhibiting ketohexokinase (khk)
JP2024506709A (ja) Ｕｂｅ３ａアンチセンス治療剤
US20250163419A1 (en) Therapeutic compositions for treating pain via multiple targets
EP4463553B1 (en) Advanced rna targeting (arnatar)
US20240209377A1 (en) Therapeutic compositions for treating pain via multiple targets
US20250304968A1 (en) Ube3a antisense therapeutics
CN116670276A (zh) 用于通过多个靶标治疗疼痛的治疗组合物
JP7288052B2 (ja) Ｓｃｎ９ａ発現を阻害するための増強されたオリゴヌクレオチド
US20230313189A1 (en) Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same
US20250277222A1 (en) Therapeutic compositions for treating pain via multiple targets
US20220370487A1 (en) Compositions targeting sodium channel 1.6
EP4149486A1 (en) Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
WO2025190276A1 (zh) 抑制dmpk表达的多核苷酸分子及其用途
HK40128552A (en) Advanced rna targeting (arnatar)
HK40084486A (en) Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
HK40085370A (en) Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same
JP2023527693A (ja) 神経疾患を治療するための補体成分ｃ１ｒ阻害剤、並びに関連する組成物、システム、及びそれを使用する方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231027

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250214

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250214

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251203

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260303