AU2019314529B2 - Variant RNAi against alpha-synuclein - Google Patents

Variant RNAi against alpha-synuclein

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Publication number
AU2019314529B2
AU2019314529B2 AU2019314529A AU2019314529A AU2019314529B2 AU 2019314529 B2 AU2019314529 B2 AU 2019314529B2 AU 2019314529 A AU2019314529 A AU 2019314529A AU 2019314529 A AU2019314529 A AU 2019314529A AU 2019314529 B2 AU2019314529 B2 AU 2019314529B2
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Australia
Prior art keywords
rnai
nucleic acid
strand
sequence
seq
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AU2019314529A
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AU2019314529A1 (en
Inventor
Véronique BLANCHARD
Bradford ELMER
Martine Latta-Mahieu
Maria Carmen Obinu
Brenda RICHARDS
Véronique Taupin
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Genzyme Corp
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Genzyme Corp
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    • 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)
AU2019314529A 2018-08-03 2019-08-02 Variant RNAi against alpha-synuclein Active AU2019314529B2 (en)

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

Publications (2)

Publication Number Publication Date
AU2019314529A1 AU2019314529A1 (en) 2021-03-25
AU2019314529B2 true AU2019314529B2 (en) 2025-09-11

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AU2019314529A Active AU2019314529B2 (en) 2018-08-03 2019-08-02 Variant RNAi against alpha-synuclein

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US (1) US12365895B2 (enExample)
EP (2) EP3830265A1 (enExample)
JP (2) JP2021534814A (enExample)
KR (1) KR102931619B1 (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) IL280581B1 (enExample)
MX (1) MX2021001395A (enExample)
PH (1) PH12021550254A1 (enExample)
SG (1) SG11202101072UA (enExample)
TW (2) TW202509215A (enExample)
WO (1) WO2020028816A1 (enExample)

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MX2020003095A (es) 2017-09-22 2020-10-15 Genzyme Corp Arni variante.
TW202509215A (zh) 2018-08-03 2025-03-01 美商健臻公司 針對α-突觸核蛋白的變體RNAi
JP2023518268A (ja) * 2020-03-18 2023-04-28 ユニバーシティー オブ マサチューセッツ Ｓｎｃａ調節のためのオリゴヌクレオチド
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
JP2024523445A (ja) * 2021-06-21 2024-06-28 ユニキュアー バイオファーマ ビー．ブイ． α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用
KR20240166577A (ko) * 2022-04-01 2024-11-26 알닐람 파마슈티칼스 인코포레이티드 Snca-연관된 질환을 치료하기 위한 snca-표적화 sirna 조성물
KR20240169111A (ko) * 2022-04-06 2024-12-02 젠자임 코포레이션 Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법
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
WO2025170027A1 (ja) 2024-02-08 2025-08-14 テルモ株式会社 オリゴヌクレオチド

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Also Published As

Publication number Publication date
EP4763987A3 (en) 2026-08-05
JP2021534814A (ja) 2021-12-16
TW202509215A (zh) 2025-03-01
EP3830265A1 (en) 2021-06-09
IL280581A (en) 2021-03-25
SG11202101072UA (en) 2021-03-30
MX2021001395A (es) 2021-08-11
EP4763987A2 (en) 2026-06-24
AR114540A1 (es) 2020-09-16
KR102931619B1 (ko) 2026-02-26
PH12021550254A1 (en) 2023-02-06
US20210309999A1 (en) 2021-10-07
CA3108526A1 (en) 2020-02-06
CN112805382A (zh) 2021-05-14
IL280581B1 (en) 2026-07-01
TW202022116A (zh) 2020-06-16
KR20210062627A (ko) 2021-05-31
US12365895B2 (en) 2025-07-22
CO2021002637A2 (es) 2021-07-30
AU2019314529A1 (en) 2021-03-25
JP2024156715A (ja) 2024-11-06
BR112021001980A2 (pt) 2021-05-04
WO2020028816A1 (en) 2020-02-06

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