JP2021534814A - アルファ−シヌクレインに対するバリアントＲＮＡｉ - Google Patents

アルファ−シヌクレインに対するバリアントＲＮＡｉ Download PDF

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Publication number
JP2021534814A
JP2021534814A JP2021529252A JP2021529252A JP2021534814A JP 2021534814 A JP2021534814 A JP 2021534814A JP 2021529252 A JP2021529252 A JP 2021529252A JP 2021529252 A JP2021529252 A JP 2021529252A JP 2021534814 A JP2021534814 A JP 2021534814A
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nucleic acid
rnai
promoter
seq
strand
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JP2021529252A
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JP2021534814A5 (https=
Inventor
ブラッドフォード・エルマー
ブレンダ・リチャーズ
マルティーヌ・ラッタ−マユー
マリア・カルメン・オビヌ
ヴェロニク・トウパン
ヴェロニク・ブランシャール
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Genzyme Corp
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Genzyme Corp
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Publication of JP2021534814A publication Critical patent/JP2021534814A/ja
Publication of JP2021534814A5 publication Critical patent/JP2021534814A5/ja
Priority to JP2024115452A priority Critical patent/JP2024156715A/ja
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Psychiatry (AREA)
  • Epidemiology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021529252A 2018-08-03 2019-08-02 アルファ−シヌクレインに対するバリアントＲＮＡｉ Pending JP2021534814A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024115452A JP2024156715A (ja) 2018-08-03 2024-07-19 アルファ－シヌクレインに対するバリアントＲＮＡｉ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862714616P 2018-08-03 2018-08-03
US62/714,616 2018-08-03
PCT/US2019/044924 WO2020028816A1 (en) 2018-08-03 2019-08-02 Variant rnai against alpha-synuclein

Related Child Applications (1)

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JP2024115452A Division JP2024156715A (ja) 2018-08-03 2024-07-19 アルファ－シヌクレインに対するバリアントＲＮＡｉ

Publications (2)

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JP2021534814A true JP2021534814A (ja) 2021-12-16
JP2021534814A5 JP2021534814A5 (https=) 2022-08-04

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JP2021529252A Pending JP2021534814A (ja) 2018-08-03 2019-08-02 アルファ−シヌクレインに対するバリアントＲＮＡｉ
JP2024115452A Pending JP2024156715A (ja) 2018-08-03 2024-07-19 アルファ－シヌクレインに対するバリアントＲＮＡｉ

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Country Status (16)

Country Link
US (1) US12365895B2 (https=)
EP (1) EP3830265A1 (https=)
JP (2) JP2021534814A (https=)
KR (1) KR102931619B1 (https=)
CN (1) CN112805382A (https=)
AR (1) AR114540A1 (https=)
AU (1) AU2019314529B2 (https=)
BR (1) BR112021001980A2 (https=)
CA (1) CA3108526A1 (https=)
CO (1) CO2021002637A2 (https=)
IL (1) IL280581A (https=)
MX (1) MX2021001395A (https=)
PH (1) PH12021550254A1 (https=)
SG (1) SG11202101072UA (https=)
TW (2) TW202509215A (https=)
WO (1) WO2020028816A1 (https=)

Families Citing this family (10)

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Publication number Priority date Publication date Assignee Title
CN111356763B (zh) * 2017-09-22 2024-03-12 建新公司 变体RNAi
KR102931619B1 (ko) 2018-08-03 2026-02-26 젠자임 코포레이션 알파-시뉴클레인에 대한 변이형 RNAi
US20210363524A1 (en) * 2020-03-18 2021-11-25 University Of Massachusetts Oligonucleotides for snca modulation
EP4221838A4 (en) * 2020-10-01 2025-04-16 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
WO2022268835A1 (en) * 2021-06-21 2022-12-29 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
WO2023196862A1 (en) * 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
US20260103703A1 (en) * 2022-09-29 2026-04-16 Atalanta Therapeutics, Inc. siRNA COMPOSITIONS AND METHODS TARGETING ALPHA-SYNUCLEIN NUCLEIC ACIDS
US20240384271A1 (en) * 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2025160429A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025162214A1 (en) * 2024-01-30 2025-08-07 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of synuclein alpha (snca) gene

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
JP2014501507A (ja) * 2010-11-17 2014-01-23 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 α−シヌクレイン発現の調節
JP2018506304A (ja) * 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ

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US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
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Publication number Priority date Publication date Assignee Title
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
JP2014501507A (ja) * 2010-11-17 2014-01-23 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 α−シヌクレイン発現の調節
JP2018506304A (ja) * 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ

Also Published As

Publication number Publication date
AU2019314529B2 (en) 2025-09-11
WO2020028816A1 (en) 2020-02-06
EP3830265A1 (en) 2021-06-09
TW202022116A (zh) 2020-06-16
KR102931619B1 (ko) 2026-02-26
PH12021550254A1 (en) 2023-02-06
IL280581A (en) 2021-03-25
JP2024156715A (ja) 2024-11-06
AU2019314529A1 (en) 2021-03-25
CN112805382A (zh) 2021-05-14
BR112021001980A2 (pt) 2021-05-04
AR114540A1 (es) 2020-09-16
CO2021002637A2 (es) 2021-07-30
US12365895B2 (en) 2025-07-22
CA3108526A1 (en) 2020-02-06
KR20210062627A (ko) 2021-05-31
TW202509215A (zh) 2025-03-01
US20210309999A1 (en) 2021-10-07
MX2021001395A (es) 2021-08-11
SG11202101072UA (en) 2021-03-30

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