AU2021232029A1 - Compositions and methods for inhibiting expression of transthyretin (TTR) - Google Patents

Compositions and methods for inhibiting expression of transthyretin (TTR) Download PDF

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Publication number
AU2021232029A1
AU2021232029A1 AU2021232029A AU2021232029A AU2021232029A1 AU 2021232029 A1 AU2021232029 A1 AU 2021232029A1 AU 2021232029 A AU2021232029 A AU 2021232029A AU 2021232029 A AU2021232029 A AU 2021232029A AU 2021232029 A1 AU2021232029 A1 AU 2021232029A1
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Australia
Prior art keywords
rnai agent
ttr
fixed dose
double stranded
phosphate
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Pending
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AU2021232029A
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English (en)
Inventor
Amy Chan
Maja JANAS
Robin D. MCDOUGALL
Diane RAMSDEN
Mark K. SCHLEGEL
Jessica E. SUTHERLAND
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of AU2021232029A1 publication Critical patent/AU2021232029A1/en
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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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 N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2021232029A 2020-03-06 2021-03-05 Compositions and methods for inhibiting expression of transthyretin (TTR) Pending AU2021232029A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062985950P 2020-03-06 2020-03-06
US62/985,950 2020-03-06
PCT/US2021/021049 WO2021178778A1 (en) 2020-03-06 2021-03-05 Compositions and methods for inhibiting expression of transthyretin (ttr)

Publications (1)

Publication Number Publication Date
AU2021232029A1 true AU2021232029A1 (en) 2022-10-06

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ID=75252841

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AU2021232029A Pending AU2021232029A1 (en) 2020-03-06 2021-03-05 Compositions and methods for inhibiting expression of transthyretin (TTR)

Country Status (8)

Country Link
EP (1) EP4114949A1 (ja)
JP (1) JP2023516748A (ja)
KR (1) KR20220152270A (ja)
CN (1) CN115461460A (ja)
AU (1) AU2021232029A1 (ja)
CA (1) CA3174473A1 (ja)
IL (1) IL296106A (ja)
WO (1) WO2021178778A1 (ja)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI3329002T1 (sl) 2015-07-31 2021-02-26 Alnylam Pharmaceuticals, Inc. Sestavki transtiretin (TTR) IRNA in sestavki in postopki njihove uporabe za zdravljenje ali preprečevanje s TTR povezanih bolezni
WO2019060442A1 (en) 2017-09-19 2019-03-28 Alnylam Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF TRANSTHYRETIN MEDIATED AMYLOSIS (TTR)
KR20240042004A (ko) 2021-08-03 2024-04-01 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물 및 이의 사용 방법
WO2024130142A2 (en) * 2022-12-16 2024-06-20 Amgen Inc. Rnai constructs for inhibiting ttr expression and methods of use thereof

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EP4114949A1 (en) 2023-01-11
WO2021178778A1 (en) 2021-09-10
CA3174473A1 (en) 2021-09-10
CN115461460A (zh) 2022-12-09
IL296106A (en) 2022-11-01
KR20220152270A (ko) 2022-11-15
JP2023516748A (ja) 2023-04-20

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