AU2022246786A1 - Huntingtin (htt) irna agent compositions and methods of use thereof - Google Patents

Huntingtin (htt) irna agent compositions and methods of use thereof Download PDF

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Publication number
AU2022246786A1
AU2022246786A1 AU2022246786A AU2022246786A AU2022246786A1 AU 2022246786 A1 AU2022246786 A1 AU 2022246786A1 AU 2022246786 A AU2022246786 A AU 2022246786A AU 2022246786 A AU2022246786 A AU 2022246786A AU 2022246786 A1 AU2022246786 A1 AU 2022246786A1
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Prior art keywords
nucleotide
nucleotides
seq
antisense strand
nucleotide sequence
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AU2022246786A
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Inventor
Adam CASTORENO
James D. MCININCH
Mark K. SCHLEGEL
Mangala Meenakshi SOUNDARAPANDIAN
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of AU2022246786A1 publication Critical patent/AU2022246786A1/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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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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
    • 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
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2022246786A 2021-03-29 2022-03-28 Huntingtin (htt) irna agent compositions and methods of use thereof Pending AU2022246786A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163167140P 2021-03-29 2021-03-29
US63/167,140 2021-03-29
PCT/US2022/022093 WO2022212231A2 (en) 2021-03-29 2022-03-28 Huntingtin (htt) irna agent compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
AU2022246786A1 true AU2022246786A1 (en) 2023-10-05

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ID=81388848

Family Applications (1)

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AU2022246786A Pending AU2022246786A1 (en) 2021-03-29 2022-03-28 Huntingtin (htt) irna agent compositions and methods of use thereof

Country Status (13)

Country Link
US (2) US11993774B2 (https=)
EP (1) EP4314296A2 (https=)
JP (1) JP2024512635A (https=)
KR (1) KR20230162024A (https=)
CN (1) CN117203336A (https=)
AR (1) AR125230A1 (https=)
AU (1) AU2022246786A1 (https=)
BR (1) BR112023019981A2 (https=)
CA (1) CA3214499A1 (https=)
IL (1) IL307239A (https=)
MX (1) MX2023011466A (https=)
TW (1) TW202305134A (https=)
WO (1) WO2022212231A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4051795A1 (en) * 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2024156083A1 (en) * 2023-01-27 2024-08-02 Beijing Jitai Pharmaceutical Technology Co., Ltd. Ai-assisted design of lnps for effective cns delivery of rna
TW202532088A (zh) * 2024-02-07 2025-08-16 大陸商上海舶望製藥有限公司 抑制亨廷頓蛋白（htt）表達的組合物和方法
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

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