CN112805382A - 针对α-突触核蛋白的变体RNAi - Google Patents

针对α-突触核蛋白的变体RNAi Download PDF

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Publication number
CN112805382A
CN112805382A CN201980063972.6A CN201980063972A CN112805382A CN 112805382 A CN112805382 A CN 112805382A CN 201980063972 A CN201980063972 A CN 201980063972A CN 112805382 A CN112805382 A CN 112805382A
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nucleic acid
rnai
promoter
sequence
aav
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Pending
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CN201980063972.6A
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English (en)
Chinese (zh)
Inventor
B·埃尔默
B·理查兹
M·拉塔·马希欧
M·G·奥比努
V·陶平
V·布兰查德
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Genzyme Corp
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Genzyme Corp
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    • 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
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    • 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
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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
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    • C12N2320/00—Applications; Uses
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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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  • Genetics & Genomics (AREA)
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  • 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)
CN201980063972.6A 2018-08-03 2019-08-02 针对α-突触核蛋白的变体RNAi Pending CN112805382A (zh)

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 (1)

Publication Number Publication Date
CN112805382A true CN112805382A (zh) 2021-05-14

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CN201980063972.6A Pending CN112805382A (zh) 2018-08-03 2019-08-02 针对α-突触核蛋白的变体RNAi

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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)

Cited By (1)

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CN111356763A (zh) * 2017-09-22 2020-06-30 建新公司 变体RNAi

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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

Citations (5)

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US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
WO2010029303A1 (en) * 2008-09-12 2010-03-18 Isis Innovation Limited Gene silencing
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
WO2012068405A2 (en) * 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN107438671A (zh) * 2015-02-10 2017-12-05 建新公司 变体RNAi

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Publication number Priority date Publication date Assignee Title
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
WO2010029303A1 (en) * 2008-09-12 2010-03-18 Isis Innovation Limited Gene silencing
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
WO2012068405A2 (en) * 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN107438671A (zh) * 2015-02-10 2017-12-05 建新公司 变体RNAi

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111356763A (zh) * 2017-09-22 2020-06-30 建新公司 变体RNAi
CN111356763B (zh) * 2017-09-22 2024-03-12 建新公司 变体RNAi
US12331296B2 (en) 2017-09-22 2025-06-17 Genzyme Corporation Variant RNAi

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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
AU2019314529B2 (en) 2025-09-11
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
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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