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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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) KR20240035907A (en)
CN (2) CN117757791A (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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EP3649243A1 (en) * 2017-07-07 2020-05-13 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing contact-activation pathway-associated diseases using irna compositions targeting factor xii (hageman factor) (f12)
EP3790557A4 (en) * 2018-05-07 2022-03-02 Alnylam Pharmaceuticals Inc. Compositions and methods for improving strand biased
WO2019222479A1 (en) * 2018-05-16 2019-11-21 Alnylam Pharmaceuticals, Inc. Modified rna agents with reduced off-target effect
EP3802824A1 (en) 2018-06-05 2021-04-14 F. Hoffmann-La Roche AG Oligonucleotides for modulating atxn2 expression
BR112021000538A2 (en) 2018-07-13 2021-04-06 F. Hoffmann-La Roche Ag OLIGONUCLEOTIDS TO MODULATE RTEL1 EXPRESSION
EA202190528A1 (en) * 2018-08-13 2021-04-23 Элнилэм Фармасьютикалз, Инк. COMPOSITIONS OF HEPATITIS B VIRUS (HBV) dsRNA AGENTS AND METHODS OF THEIR APPLICATION
EA202192733A1 (en) 2019-04-03 2022-03-14 Бристол-Маерс Сквибб Компани ANGPTL2 ANTISENSE OLIGONUCLEOTIDES AND THEIR APPLICATIONS
CN114402074A (en) * 2019-07-16 2022-04-26 国立大学法人东京大学 RNA molecule, chimeric NA molecule, double-stranded RNA molecule, and double-stranded chimeric NA molecule
KR20220062517A (en) 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 Linkage-modified oligomeric compounds and uses thereof
MX2022002689A (en) * 2019-09-03 2022-04-07 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of the lect2 gene.
EP4058577A1 (en) * 2019-11-13 2022-09-21 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
WO2021122735A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sept9 inhibitors for treating hepatitis b virus infection
EP4077669A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of sbds inhibitors for treating hepatitis b virus infection
EP4077671A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of saraf inhibitors for treating hepatitis b virus infection
EP4077668A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of scamp3 inhibitors for treating hepatitis b virus infection
EP4077670A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of cops3 inhibitors for treating hepatitis b virus infection
WO2021130270A1 (en) 2019-12-24 2021-07-01 F. Hoffmann-La Roche Ag Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
CA3163490A1 (en) 2019-12-24 2021-07-01 F. Hoffman-La Roche Ag Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
WO2021231211A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
JP2023525770A (en) 2020-05-11 2023-06-19 ジェネンテック, インコーポレイテッド COMPLEMENT C4 INHIBITORS AND RELATED COMPOSITIONS FOR TREATMENT OF NEUROLOGICAL DISEASES, AND SYSTEMS AND METHODS USING THE SAME
JP2023527693A (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 C1R Inhibitors and Related Compositions, Systems, and Methods of Using The Same for Treating Neurological Disorders
WO2022038211A2 (en) 2020-08-21 2022-02-24 F. Hoffmann-La Roche Ag Use of a1cf inhibitors for treating hepatitis b virus infection
EP4214319A2 (en) * 2020-09-17 2023-07-26 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets
EP4279591A1 (en) * 2021-01-15 2023-11-22 The University of Tokyo Rna molecule, chimeric na molecule, double-stranded rna molecule, and double-stranded chimeric na molecule
EP4291654A2 (en) 2021-02-12 2023-12-20 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
AU2022345098A1 (en) 2021-09-16 2024-04-04 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2023083906A2 (en) 2021-11-11 2023-05-19 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
CN116003494A (en) * 2022-01-05 2023-04-25 大睿生物医药科技(上海)有限公司 Double stranded RNA with nucleotide analogs
WO2024098061A2 (en) 2022-11-04 2024-05-10 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure

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Also Published As

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

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