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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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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JPWO2020028816A5 (enExample
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 JPWO2020028816A5 publication Critical patent/JPWO2020028816A5/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
    • 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
    • C12N2310/14—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)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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

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JP2021534814A true JP2021534814A (ja) 2021-12-16
JPWO2020028816A5 JPWO2020028816A5 (enExample) 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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US (1) US12365895B2 (enExample)
EP (1) EP3830265A1 (enExample)
JP (2) JP2021534814A (enExample)
KR (1) KR20210062627A (enExample)
CN (1) CN112805382A (enExample)
AR (1) AR114540A1 (enExample)
AU (1) AU2019314529B2 (enExample)
BR (1) BR112021001980A2 (enExample)
CA (1) CA3108526A1 (enExample)
CO (1) CO2021002637A2 (enExample)
IL (1) IL280581A (enExample)
MX (1) MX2021001395A (enExample)
PH (1) PH12021550254A1 (enExample)
SG (1) SG11202101072UA (enExample)
TW (2) TW202509215A (enExample)
WO (1) WO2020028816A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3684932A1 (en) 2017-09-22 2020-07-29 Genzyme Corporation Variant rnai
PH12021550254A1 (en) 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein
CN115777021A (zh) * 2020-03-18 2023-03-10 马萨诸塞大学 用于snca调节的寡核苷酸
WO2022072447A1 (en) * 2020-10-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
EP4359525A1 (en) * 2021-06-21 2024-05-01 uniQure biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
AU2023248463A1 (en) * 2022-04-06 2024-11-28 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
JP2025531512A (ja) * 2022-09-29 2025-09-19 アタランタ セラピューティクス，インコーポレーテッド アルファシヌクレイン核酸を標的化するｓｉＲＮＡ組成物及び方法
WO2024211480A2 (en) * 2023-04-05 2024-10-10 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2025160429A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
TW202529779A (zh) * 2024-01-30 2025-08-01 大陸商上海舶望製藥有限公司 用於抑制突觸核蛋白α（SNCA）基因表達的組合物和方法

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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
US20050137155A1 (en) 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
GB0816778D0 (en) 2008-09-12 2008-10-22 Isis Innovation Gene silencing
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
JP6224459B2 (ja) 2011-02-17 2017-11-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
BR112014021104B1 (pt) 2012-02-29 2023-03-28 Sangamo Biosciences, Inc Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington
EP4600255A3 (en) 2014-05-02 2025-10-22 Genzyme Corporation Aav vectors for retinal and cns gene therapy
PH12021550254A1 (en) 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein

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

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