CA3227144A1 - Modified small interfering rna molecules with reduced off-target effects - Google Patents

Modified small interfering rna molecules with reduced off-target effects Download PDF

Info

Publication number
CA3227144A1
CA3227144A1 CA3227144A CA3227144A CA3227144A1 CA 3227144 A1 CA3227144 A1 CA 3227144A1 CA 3227144 A CA3227144 A CA 3227144A CA 3227144 A CA3227144 A CA 3227144A CA 3227144 A1 CA3227144 A1 CA 3227144A1
Authority
CA
Canada
Prior art keywords
nucleotides
disease
interfering rna
modified sirna
positions
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3227144A
Other languages
English (en)
French (fr)
Inventor
Yi-Chung Chang
Chi-Fan Yang
Hui-Yu Chen
Chia-Chun Yang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Microbio Shanghai Co Ltd
Original Assignee
Microbio Shanghai Co Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Microbio Shanghai Co Ltd filed Critical Microbio Shanghai Co Ltd
Publication of CA3227144A1 publication Critical patent/CA3227144A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
CA3227144A 2021-07-22 2022-07-21 Modified small interfering rna molecules with reduced off-target effects Pending CA3227144A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2021107862 2021-07-22
CNPCT/CN2021/107862 2021-07-22
PCT/CN2022/107028 WO2023001234A1 (en) 2021-07-22 2022-07-21 Modified small interfering rna molecules with reduced off-target effects

Publications (1)

Publication Number Publication Date
CA3227144A1 true CA3227144A1 (en) 2023-01-26

Family

ID=84979733

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3227144A Pending CA3227144A1 (en) 2021-07-22 2022-07-21 Modified small interfering rna molecules with reduced off-target effects

Country Status (7)

Country Link
US (1) US20240425853A1 (https=)
EP (1) EP4373932A1 (https=)
JP (1) JP2024529930A (https=)
KR (1) KR20240042457A (https=)
CN (1) CN118202046A (https=)
CA (1) CA3227144A1 (https=)
WO (1) WO2023001234A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080188430A1 (en) * 2001-05-18 2008-08-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
WO2005035759A2 (en) * 2003-08-20 2005-04-21 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2005032486A2 (en) * 2003-10-02 2005-04-14 Duke University A NOVEL SIRNA-BASED APPROACH TO TARGET THE HIF-α FACTOR FOR GENE THERAPY
EP3889261A1 (en) * 2007-08-27 2021-10-06 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
CN106117289B (zh) * 2016-06-24 2019-02-12 郑州大学 2’-o-moe-3’-h-硫代磷酸酯核苷单体及其合成方法
CN110540990B (zh) * 2019-07-16 2024-08-20 武汉泽智生物医药有限公司 沉默小眼畸形转录因子mRNA表达的siRNA分子

Also Published As

Publication number Publication date
KR20240042457A (ko) 2024-04-02
EP4373932A1 (en) 2024-05-29
CN118202046A (zh) 2024-06-14
US20240425853A1 (en) 2024-12-26
WO2023001234A9 (en) 2023-03-02
WO2023001234A1 (en) 2023-01-26
JP2024529930A (ja) 2024-08-14

Similar Documents

Publication Publication Date Title
CN111107853B (zh) 用于抑制载脂蛋白C-III (APOC3)的表达的RNAi试剂和组合物
EP3236974B9 (en) Rna interference agents for gst-pi gene modulation
WO2023098908A1 (en) Modification patterns for small interfering rna molecules with high stability and gene silencing activities
EP3386519B1 (en) Sirna structures for high activity and reduced off target
TW202321452A (zh) 用於抑制血管生成素樣３（ａｎｇｐｔｌ３）蛋白表達的組合物和方法
US20170081667A1 (en) Nucleic acid that inhibits expression of irf5
JP2011524745A (ja) 修飾オリゴヌクレオチドを使用するｈｒｐ−３の阻害
TW201730341A (zh) 抑制補體b因子之表現之反義寡核苷酸
WO2023001234A1 (en) Modified small interfering rna molecules with reduced off-target effects
WO2026026776A1 (zh) 抑制APP基因表达的siRNA及其缀合物和应用
CN113508175B (zh) 用于治疗癌症的组合物和方法
HK1231403A1 (en) Nucleic acid capable of inhibiting expression of beta2gpi
WO2026067447A1 (en) Small Interfering RNAs Targeting ANT2 and Uses Thereof for Disease Treatment
EP4678747A2 (en) Double-stranded rnai agents for targeting and regulating hbv gene expression, and a use thereof
CN112654706B (zh) 靶向淋巴细胞活化基因3(lag-3)的核酸适体及其用途
JP6751185B2 (ja) ＧＳＴ−π遺伝子を調節するためのＲＮＡ干渉剤
EP4653531A1 (en) Antisense oligomers targeting gfral and use thereof
TW202527968A (zh) 用於抑制跨膜絲氨酸蛋白酶6（tmprss6）表達的組合物和方法
TW202532088A (zh) 抑制亨廷頓蛋白（htt）表達的組合物和方法
TW202521133A (zh) 抑制甲狀腺素運載蛋白(ttr)表達的組合物和方法
HK40062991A (en) Compositions and methods for treating cancer
CN120359038A (zh) 抑制性核酸及其使用方法
HK1260156B (en) Sirna structures for high activity and reduced off target
HK1260156A1 (en) Sirna structures for high activity and reduced off target
HK1245674B (en) Rna interference agents for gst-pi gene modulation

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD

Year of fee payment: 2

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240719

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20240719

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250721

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250721