JP2023504661A - 疾患の処置のための標的化トランスファーｒｎａ - Google Patents

疾患の処置のための標的化トランスファーｒｎａ Download PDF

Info

Publication number
JP2023504661A
JP2023504661A JP2022533139A JP2022533139A JP2023504661A JP 2023504661 A JP2023504661 A JP 2023504661A JP 2022533139 A JP2022533139 A JP 2022533139A JP 2022533139 A JP2022533139 A JP 2022533139A JP 2023504661 A JP2023504661 A JP 2023504661A
Authority
JP
Japan
Prior art keywords
trna
variant
engineered
substitution
engineered trna
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
JP2022533139A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023504661A5 (https=
Inventor
ハス，デイビッド
スタイン，リアーナ
サリバン，リチャード
ラクシュマナン，アヌパマ
バーレイ，スティーブン
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.)
Shape Therapeutics Inc
Original Assignee
Shape Therapeutics 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 Shape Therapeutics Inc filed Critical Shape Therapeutics Inc
Publication of JP2023504661A publication Critical patent/JP2023504661A/ja
Publication of JP2023504661A5 publication Critical patent/JP2023504661A5/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
    • 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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • 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

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022533139A 2019-12-02 2020-12-01 疾患の処置のための標的化トランスファーｒｎａ Pending JP2023504661A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962942667P 2019-12-02 2019-12-02
US201962942690P 2019-12-02 2019-12-02
US62/942,667 2019-12-02
US62/942,690 2019-12-02
US202063010856P 2020-04-16 2020-04-16
US63/010,856 2020-04-16
US202063111856P 2020-11-10 2020-11-10
US63/111,856 2020-11-10
PCT/US2020/062671 WO2021113218A1 (en) 2019-12-02 2020-12-01 Targeted transfer rnas for treatment of diseases

Publications (2)

Publication Number Publication Date
JP2023504661A true JP2023504661A (ja) 2023-02-06
JP2023504661A5 JP2023504661A5 (https=) 2023-12-08

Family

ID=74046150

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022533139A Pending JP2023504661A (ja) 2019-12-02 2020-12-01 疾患の処置のための標的化トランスファーｒｎａ

Country Status (6)

Country Link
EP (1) EP4069841A1 (https=)
JP (1) JP2023504661A (https=)
CN (1) CN115397983A (https=)
AU (1) AU2020396868A1 (https=)
CA (1) CA3163514A1 (https=)
WO (1) WO2021113218A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102930465B1 (ko) 2017-11-02 2026-02-25 더 위스타 인스티튜트 오브 아나토미 앤드 바이올로지 ACE-tRNA를 이용한 유전 재할당을 통한 정지 코돈의 구조 방법
MX2022012955A (es) 2020-04-14 2023-02-23 Flagship Pioneering Innovations Vi Llc Composiciones de trem y usos de las mismas.
WO2023097268A2 (en) * 2021-11-23 2023-06-01 Mantra Bio, Inc. Extracellular vesicles comprising non-naturally occurring modular rna hairpins and uses thereof
WO2023220342A2 (en) 2022-05-13 2023-11-16 Shape Therapeutics Inc. Engineered tranfer rnas
WO2024149415A1 (en) * 2023-01-10 2024-07-18 Biologicke Centrum Av Cr, V. V. I. Altered trnas for treatment of genetic diseases caused by premature termination codon
WO2024197081A1 (en) * 2023-03-20 2024-09-26 Hc Bioscience, Inc. Minimally-sized dna threads for trna therapy
EP4442826A1 (en) 2023-04-06 2024-10-09 Universität Hamburg Synthetic dna construct encoding transfer rna
EP4458967A1 (en) 2023-05-02 2024-11-06 Universität Hamburg Pharmaceutical composition comprising multiple suppressor transfer rnas
TW202504646A (zh) * 2023-06-16 2025-02-01 北京大學 利用環狀ｒｎａ抑制無義介導的降解的系統和方法
WO2025036281A1 (en) * 2023-08-11 2025-02-20 Shanghai Codone Biotechnology Co. Engineered suppressor trna molecules and methods
WO2026001270A1 (en) * 2024-06-24 2026-01-02 Zhuhai Codone Biotechnology Co., Ltd. Engineered trna molecules and methods

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019090169A1 (en) * 2017-11-02 2019-05-09 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with ace-trna

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012006551A2 (en) * 2010-07-08 2012-01-12 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CN110612353A (zh) * 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
US10968257B2 (en) * 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
CA3125567A1 (en) * 2019-01-18 2020-07-23 Flagship Pioneering, Inc. Trem compositions and uses thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019090169A1 (en) * 2017-11-02 2019-05-09 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with ace-trna

Also Published As

Publication number Publication date
WO2021113218A1 (en) 2021-06-10
EP4069841A1 (en) 2022-10-12
AU2020396868A1 (en) 2022-07-14
CA3163514A1 (en) 2021-06-10
CN115397983A (zh) 2022-11-25

Similar Documents

Publication Publication Date Title
JP2023504661A (ja) 疾患の処置のための標的化トランスファーｒｎａ
US20240279650A1 (en) Therapeutic Editing
TWI804518B (zh) 肌萎縮性脊髓側索硬化症(als)之治療
US11155817B2 (en) Therapeutic for treatment of diseases including the central nervous system
BR112020001940A2 (pt) modelos celulares de e terapias para doenças oculares
KR20200039617A (ko) 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
US20250049955A1 (en) Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20240035047A1 (en) Manufacturing and use of recombinant aav vectors
JP2023501897A (ja) Ｃ９ｏｒｆ７２関連疾患の治療のための三重機能アデノ随伴ウイルス（ａａｖ）ベクター
WO2022081890A1 (en) Compositions and methods for treating glycogen storage disease type 1a
JP2024515344A (ja) 補体成分３の発現を阻害するための組成物及び方法
CN121241061A (zh) 衣壳多肽及其使用方法
TW202323528A (zh) 用於治療威爾森氏病之基因療法
EP3299456A1 (en) Recombinant dgkk gene for fragile x syndrome gene therapy
CN114222818B (zh) cPLA2e诱导剂及其用途
TW202535908A (zh) 中樞神經系統靶向之ａａｖ衣殼之重定向
CA3235208A1 (en) Gene therapy for the treatment of ht1
WO2024238584A1 (en) Engineered constructs with cassette arrangements for increased transcription of rna payloads
WO2025096897A1 (en) Method for identifying and employing target site blocking oligonucleotides in the treatment of mef2c haploinsufficiency syndrome
TW202548010A (zh) 用於靶向人類運鐵蛋白受體之ａａｖ衣殼
WO2025227159A1 (en) Gene editing treatment for correcting splicing defect in familial dysautonomia
TW202545971A (zh) 殼體多肽及其使用方法
EP4665853A1 (en) Compositions for and methods of treating and/or preventing glutaric aciduria type-i
TW202528335A (zh) 衣殼多肽及其使用方法(一)
WO2022197941A2 (en) Methods of detecting pathogenic variants in sarm1 gene

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231130

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20231130

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20240502

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241210

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250603