SG10201913786SA - Modified rna agents with reduced off-target effect - Google Patents

Modified rna agents with reduced off-target effect

Info

Publication number
SG10201913786SA
SG10201913786SA SG10201913786SA SG10201913786SA SG10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA
Authority
SG
Singapore
Prior art keywords
reduced
modified rna
target effect
rna agents
agents
Prior art date
Application number
SG10201913786SA
Inventor
Christopher Theile
Stuart Milstein
Mark K Schlegel
Maja Janas
Vasant R Jadhav
Donald Foster
Muthiah Manoharan
Kallanthottathil G Rajeev
Muthusamy Jayaraman
Alexander V Kel'in
Shigeo Matsuda
Klaus Charisse
Jayaprakash K Nair
Martin A Maier
Alfica Sehgal
Christopher Brown
Original Assignee
Alnylam Pharmaceuticals Inc
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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of SG10201913786SA publication Critical patent/SG10201913786SA/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/53Methods for regulating/modulating their activity reducing unwanted side-effects
SG10201913786SA 2016-11-23 2017-11-22 Modified rna agents with reduced off-target effect SG10201913786SA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662425907P 2016-11-23 2016-11-23
US201762548589P 2017-08-22 2017-08-22
US201762561514P 2017-09-21 2017-09-21

Publications (1)

Publication Number Publication Date
SG10201913786SA true SG10201913786SA (en) 2020-03-30

Family

ID=60888585

Family Applications (1)

Application Number Title Priority Date Filing Date
SG10201913786SA SG10201913786SA (en) 2016-11-23 2017-11-22 Modified rna agents with reduced off-target effect

Country Status (11)

Country Link
US (2) US11504391B1 (en)
EP (1) EP3544617A4 (en)
JP (2) JP7288852B2 (en)
KR (2) KR102645243B1 (en)
CN (2) CN110582283B (en)
AU (2) AU2017363892B2 (en)
CA (1) CA3044598A1 (en)
IL (1) IL266780A (en)
MX (1) MX2019005816A (en)
SG (1) SG10201913786SA (en)
WO (1) WO2018098328A1 (en)

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JP7384833B2 (en) * 2018-05-16 2023-11-21 アルニラム・ファーマシューティカルズ・インコーポレーテッド Modified RNA agents with reduced off-target effects
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AU2019300324A1 (en) 2018-07-13 2021-01-21 F. Hoffmann-La Roche Ag Oligonucleotides for modulating RTEL1 expression
MX2021001056A (en) * 2018-08-13 2021-04-12 Alnylam Pharmaceuticals Inc HEPATITIS B VIRUS (HBV) dsRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF.
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EP4000638A4 (en) * 2019-07-16 2023-12-27 The University of Tokyo Rna molecule, chimeric na molecule, double-stranded rna molecule, and double-stranded chimeric na molecule
WO2021030778A1 (en) 2019-08-15 2021-02-18 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
CN114616331A (en) * 2019-09-03 2022-06-10 阿尔尼拉姆医药品有限公司 Compositions and methods for inhibiting expression of LECT2 gene
JP2023502038A (en) * 2019-11-13 2023-01-20 アルナイラム ファーマシューティカルズ, インコーポレイテッド Methods and compositions for treating angiotensinogen (AGT) related disorders
CN115516091A (en) 2019-12-19 2022-12-23 豪夫迈·罗氏有限公司 Use of COPS3 inhibitors for the treatment of hepatitis b virus infection
CN114829601A (en) 2019-12-19 2022-07-29 豪夫迈·罗氏有限公司 Use of SBDS inhibitors for the treatment of hepatitis b virus infection
CN114901821A (en) 2019-12-19 2022-08-12 豪夫迈·罗氏有限公司 Use of SEPT9 inhibitors for treating hepatitis B virus infection
CN114867856A (en) 2019-12-19 2022-08-05 豪夫迈·罗氏有限公司 Use of SARAF inhibitors for the treatment of hepatitis B virus infection
WO2021122869A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of scamp3 inhibitors for treating hepatitis b virus infection
MX2022007908A (en) 2019-12-24 2022-07-21 Hoffmann La Roche Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv.
JP2023509872A (en) 2019-12-24 2023-03-10 エフ. ホフマン-ラ ロシュ アーゲー Pharmaceutical combinations of antiviral agents targeting HBV and/or immunomodulatory agents for the treatment of HBV
EP4150084A1 (en) 2020-05-11 2023-03-22 Genentech, Inc. Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same
EP4150085A1 (en) 2020-05-11 2023-03-22 Genentech, Inc. Complement component c1r inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
JP2023527684A (en) 2020-05-11 2023-06-30 ジェネンテック, インコーポレイテッド Complement component C1S inhibitors for treating neurological disorders and related compositions, systems and methods of using same
CN116157522A (en) 2020-08-21 2023-05-23 豪夫迈·罗氏有限公司 Use of A1CF inhibitors for the treatment of hepatitis B virus infection
AU2021345266A1 (en) * 2020-09-17 2023-04-27 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets
WO2022154123A1 (en) * 2021-01-15 2022-07-21 国立大学法人 東京大学 Rna molecule, chimeric na molecule, double-stranded rna molecule, and double-stranded chimeric na molecule
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US11912779B2 (en) 2021-09-16 2024-02-27 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
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WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
WO2023131170A1 (en) * 2022-01-05 2023-07-13 大睿生物医药科技(上海)有限公司 Double-stranded rna having nucleotide analog

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WO2011109427A2 (en) * 2010-03-01 2011-09-09 Alnylam Pharmaceuticals, Inc. Improving the biological activity of sirna through modulation of its thermodynamic profile
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RU2014106024A (en) * 2011-07-19 2015-08-27 Юниверсити Оф Айдахо OPTIONS FOR IMPLEMENTING THE PROBE AND METHODS OF DIRECTED ACTION FOR NUCLEIC ACIDS
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Also Published As

Publication number Publication date
JP2023053963A (en) 2023-04-13
CN110582283B (en) 2024-01-02
WO2018098328A1 (en) 2018-05-31
JP7288852B2 (en) 2023-06-08
CN117757791A (en) 2024-03-26
KR20240035907A (en) 2024-03-18
IL266780A (en) 2019-08-29
US20230256001A1 (en) 2023-08-17
KR102645243B1 (en) 2024-03-11
EP3544617A4 (en) 2020-08-05
CN110582283A (en) 2019-12-17
EP3544617A1 (en) 2019-10-02
CA3044598A1 (en) 2018-05-31
JP2019535288A (en) 2019-12-12
AU2017363892A1 (en) 2019-06-13
MX2019005816A (en) 2019-10-07
AU2017363892B2 (en) 2023-06-15
KR20190086001A (en) 2019-07-19
AU2023226646A1 (en) 2023-11-16
US11504391B1 (en) 2022-11-22

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