JP2024528540A - 遺伝子操作されたガイドｒｎａ及びポリヌクレオチド - Google Patents

遺伝子操作されたガイドｒｎａ及びポリヌクレオチド Download PDF

Info

Publication number
JP2024528540A
JP2024528540A JP2023580744A JP2023580744A JP2024528540A JP 2024528540 A JP2024528540 A JP 2024528540A JP 2023580744 A JP2023580744 A JP 2023580744A JP 2023580744 A JP2023580744 A JP 2023580744A JP 2024528540 A JP2024528540 A JP 2024528540A
Authority
JP
Japan
Prior art keywords
rna
target
relative
guide
internal loop
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
JP2023580744A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024528540A5 (enExample
Inventor
リチャード トーマス サリバン，
ブライアン ジョン ブース，
エイドリアン ブリッグス，
イアニス サバ，
Original Assignee
シェイプ セラピューティクス インコーポレイテッド
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 シェイプ セラピューティクス インコーポレイテッド filed Critical シェイプ セラピューティクス インコーポレイテッド
Publication of JP2024528540A publication Critical patent/JP2024528540A/ja
Publication of JP2024528540A5 publication Critical patent/JP2024528540A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023580744A 2021-06-29 2022-06-28 遺伝子操作されたガイドｒｎａ及びポリヌクレオチド Pending JP2024528540A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163216178P 2021-06-29 2021-06-29
US63/216,178 2021-06-29
US202163277701P 2021-11-10 2021-11-10
US63/277,701 2021-11-10
US202263303680P 2022-01-27 2022-01-27
US63/303,680 2022-01-27
US202263345059P 2022-05-24 2022-05-24
US63/345,059 2022-05-24
PCT/US2022/035320 WO2023278449A1 (en) 2021-06-29 2022-06-28 Engineered guide rnas and polynucleotides

Publications (2)

Publication Number Publication Date
JP2024528540A true JP2024528540A (ja) 2024-07-30
JP2024528540A5 JP2024528540A5 (enExample) 2025-08-07

Family

ID=82701937

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023580744A Pending JP2024528540A (ja) 2021-06-29 2022-06-28 遺伝子操作されたガイドｒｎａ及びポリヌクレオチド

Country Status (6)

Country Link
US (1) US20240271139A1 (enExample)
EP (1) EP4363579A1 (enExample)
JP (1) JP2024528540A (enExample)
AU (1) AU2022301997A1 (enExample)
CA (1) CA3223109A1 (enExample)
WO (1) WO2023278449A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4423274A2 (en) * 2021-10-26 2024-09-04 Shape Therapeutics Inc. Rna-editing compositions and methods of use
AU2023407358A1 (en) * 2022-12-23 2025-06-26 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
WO2026006334A1 (en) * 2024-06-25 2026-01-02 Shape Therapeutics Inc. Dux4 vectors and engineered guide rnas
WO2026006483A1 (en) * 2024-06-25 2026-01-02 Shape Therapeutics Inc. Snca vectors and engineered guide rnas

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020395113A1 (en) * 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
CA3177380A1 (en) * 2020-05-26 2021-12-02 Shape Therapeutics Inc. Compositions and methods for modifying target rnas
JP2024500279A (ja) * 2020-11-11 2024-01-09 シェイプ セラピューティクス インコーポレイテッド Ｒｎａ編集組成物及び使用方法
EP4256050A4 (en) * 2020-12-01 2025-01-22 Shape Therapeutics Inc. High throughput rna-editing screening methods

Also Published As

Publication number Publication date
AU2022301997A1 (en) 2024-01-04
CA3223109A1 (en) 2023-01-05
EP4363579A1 (en) 2024-05-08
WO2023278449A1 (en) 2023-01-05
US20240271139A1 (en) 2024-08-15

Similar Documents

Publication Publication Date Title
JP2024528540A (ja) 遺伝子操作されたガイドｒｎａ及びポリヌクレオチド
JP7174109B2 (ja) 腫瘍溶解性ウイルスベクター及びその使用
JP2024500279A (ja) Ｒｎａ編集組成物及び使用方法
AU2021204399B2 (en) Compositions and methods for inhibiting gene expression of alpha-1 antitrypsin
JP7231935B2 (ja) アデノシン核酸塩基編集因子およびそれらの使用
JP6276763B2 (ja) 新規脂質
JP2023527354A (ja) 標的ｒｎａを修飾するための組成物及び方法
US20230116704A1 (en) Antisense oligomers for treatment of conditions and diseases
CA3091789A1 (en) Camk2d antisense oligonucleotides and uses thereof
TW201406774A (zh) 微ｒｎａ之ｍｉｒ－１５家族之抑制劑
JP2014501493A (ja) 改変ヒトＵ１ｓｎＲＮＡ分子、改変ヒトＵ１ｓｎＲＮＡ分子をコードしている遺伝子、遺伝子を含む発現ベクターおよび遺伝子治療におけるその使用
JP2017510583A (ja) 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的ｕｎａオリゴマー
US20250243493A1 (en) Compounds for treatment of conditions and diseases
CN111868245A (zh) 用于调节tmem106b表达的寡核苷酸
JP2024538097A (ja) Ｔｒｅｍ組成物及び使用方法
JP2024520429A (ja) 操作されたガイドｒｎａ及びポリヌクレオチド
US20240279656A1 (en) Engineered Guide RNAs and Polynucleotides
IL322741A (en) Antisense oligomer formulations
WO2025199503A1 (en) Methods for treating conditions and diseases
US20250066781A1 (en) Methods for treating conditions and diseases
WO2025024568A1 (en) Methods for treating conditions and diseases
AU2023329830A1 (en) Engineered constructs for increased transcription of rna payloads
CN103221540A (zh) 病毒基因组中的高度保守区序列作为设计治疗性sliRNA模板的应用
CN117980480A (zh) 工程化引导rna和多核苷酸
JP2025542405A (ja) 操作されたガイドｒｎａ及びポリヌクレオチド

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250627

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250627

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250730

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20260427