JP2023524061A - 操作された中枢神経系用組成物 - Google Patents

操作された中枢神経系用組成物 Download PDF

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Publication number
JP2023524061A
JP2023524061A JP2022566315A JP2022566315A JP2023524061A JP 2023524061 A JP2023524061 A JP 2023524061A JP 2022566315 A JP2022566315 A JP 2022566315A JP 2022566315 A JP2022566315 A JP 2022566315A JP 2023524061 A JP2023524061 A JP 2023524061A
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JP
Japan
Prior art keywords
seq
polypeptide
mutation
protein
vector system
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JP2022566315A
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English (en)
Japanese (ja)
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JP2023524061A5 (https=
JPWO2021222831A5 (https=
Inventor
パルディス サベッティ
ムハンマドシャリフ タブボードバー
サイモン イー
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Massachusetts Institute of Technology
Broad Institute Inc
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Massachusetts Institute of Technology
Broad Institute Inc
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Application filed by Massachusetts Institute of Technology, Broad Institute Inc filed Critical Massachusetts Institute of Technology
Publication of JP2023524061A publication Critical patent/JP2023524061A/ja
Publication of JP2023524061A5 publication Critical patent/JP2023524061A5/ja
Publication of JPWO2021222831A5 publication Critical patent/JPWO2021222831A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022566315A 2020-05-01 2021-04-30 操作された中枢神経系用組成物 Withdrawn JP2023524061A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063019221P 2020-05-01 2020-05-01
US63/019,221 2020-05-01
US202063061517P 2020-08-05 2020-08-05
US63/061,517 2020-08-05
PCT/US2021/030298 WO2021222831A2 (en) 2020-05-01 2021-04-30 Engineered central nervous system compositions

Publications (3)

Publication Number Publication Date
JP2023524061A true JP2023524061A (ja) 2023-06-08
JP2023524061A5 JP2023524061A5 (https=) 2024-05-13
JPWO2021222831A5 JPWO2021222831A5 (https=) 2024-05-13

Family

ID=78373993

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022566315A Withdrawn JP2023524061A (ja) 2020-05-01 2021-04-30 操作された中枢神経系用組成物

Country Status (7)

Country Link
US (1) US20230193316A1 (https=)
EP (1) EP4143305A4 (https=)
JP (1) JP2023524061A (https=)
CN (1) CN115867646A (https=)
AU (1) AU2021264061A1 (https=)
CA (1) CA3176506A1 (https=)
WO (1) WO2021222831A2 (https=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3861010A1 (en) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
WO2022173847A2 (en) * 2021-02-09 2022-08-18 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
WO2022226375A1 (en) * 2021-04-23 2022-10-27 Locanabio, Inc. Tissue-targeted modified aav capsids and methods of use thereof
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
US20260085284A1 (en) * 2022-08-30 2026-03-26 Shanghai Genemagic Biosciences Co., Ltd. Method for trans-differentiating non-neuronal cells into neurons and use thereof
WO2024086747A1 (en) 2022-10-19 2024-04-25 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
AR133098A1 (es) * 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
CN117165677A (zh) * 2023-11-03 2023-12-05 首都儿科研究所 检测生物标志物在制备神经管畸形疾病的产品的应用
WO2025160155A1 (en) 2024-01-22 2025-07-31 The Broad Institute, Inc. Epigenetic targeting of prion diseases
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014139182A1 (en) * 2013-03-15 2014-09-18 Shenzhen Hightide Biopharmaceutical, Ltd. Compositions and methods of using islet neogenesis peptides and analogs thereof
WO2016081811A1 (en) * 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
CA2988388C (en) * 2015-04-23 2022-11-22 Nantomics, Llc Cancer neoepitopes
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
NZ763617A (en) * 2017-10-16 2026-01-30 Vigeneron Gmbh Aav vectors
IL278598B2 (en) * 2018-05-11 2025-11-01 Massachusetts Eye & Ear Infirmary Altering tissue tropism of adeno-associated viruses
US20210277418A1 (en) * 2018-08-03 2021-09-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP3861010A1 (en) * 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2023039480A2 (en) * 2021-09-08 2023-03-16 The Broad Institute, Inc. Engineered central nervous system compositions

Also Published As

Publication number Publication date
US20230193316A1 (en) 2023-06-22
WO2021222831A3 (en) 2021-12-09
WO2021222831A2 (en) 2021-11-04
EP4143305A4 (en) 2024-09-11
AU2021264061A1 (en) 2022-12-01
CA3176506A1 (en) 2021-11-04
CN115867646A (zh) 2023-03-28
EP4143305A2 (en) 2023-03-08

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