AU2019350765A1 - Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases - Google Patents

Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases Download PDF

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Publication number
AU2019350765A1
AU2019350765A1 AU2019350765A AU2019350765A AU2019350765A1 AU 2019350765 A1 AU2019350765 A1 AU 2019350765A1 AU 2019350765 A AU2019350765 A AU 2019350765A AU 2019350765 A AU2019350765 A AU 2019350765A AU 2019350765 A1 AU2019350765 A1 AU 2019350765A1
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Prior art keywords
double stranded
seq
rnai agent
phosphate
strand
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Abandoned
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Inventor
Kirk Brown
Kevin Fitzgerald
Vasant R. Jadhav
Mark Keating
Martin A. Maier
Muthiah Manoharan
Stuart Milstein
Jayaprakash K. Nair
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of AU2019350765A1 publication Critical patent/AU2019350765A1/en
Priority to AU2026201659A priority Critical patent/AU2026201659A1/en
Abandoned legal-status Critical Current

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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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—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
    • C12N2310/315—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
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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
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2019350765A 2018-09-28 2019-09-26 Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases Abandoned AU2019350765A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2026201659A AU2026201659A1 (en) 2018-09-28 2026-03-04 Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862738256P 2018-09-28 2018-09-28
US62/738,256 2018-09-28
US201962844174P 2019-05-07 2019-05-07
US62/844,174 2019-05-07
PCT/US2019/053050 WO2020069055A1 (en) 2018-09-28 2019-09-26 Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases

Related Child Applications (1)

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AU2026201659A Division AU2026201659A1 (en) 2018-09-28 2026-03-04 Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases

Publications (1)

Publication Number Publication Date
AU2019350765A1 true AU2019350765A1 (en) 2021-05-27

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AU2019350765A Abandoned AU2019350765A1 (en) 2018-09-28 2019-09-26 Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases
AU2026201659A Pending AU2026201659A1 (en) 2018-09-28 2026-03-04 Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases

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US (2) US20220333104A1 (https=)
EP (1) EP3856907A1 (https=)
JP (1) JP7470107B2 (https=)
AU (2) AU2019350765A1 (https=)
CA (1) CA3114396A1 (https=)
TW (1) TW202028465A (https=)
WO (1) WO2020069055A1 (https=)

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PT3329002T (pt) 2015-07-31 2021-01-12 Alnylam Pharmaceuticals Inc Composições de iarn de transtirretina (ttr) e métodos de seu uso para tratamento ou prevenção de doenças associadas à ttr
BR112020005230A2 (pt) 2017-09-19 2020-09-24 Alnylam Pharmaceuticals, Inc. composições e métodos para o tratamento da amiloidose mediada por transtiretina (ttr)
EP4133076A1 (en) * 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
CR20230308A (es) * 2020-12-11 2023-09-08 Civi Biopharma Inc Entrega oral de conjugados antisentido que tienen por blanco a pcsk9
WO2022192519A1 (en) * 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
WO2022232343A1 (en) * 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
AR126070A1 (es) 2021-06-08 2023-09-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para tratar o prevenir la enfermedad de stargardt y/o trastornos asociados con la proteína transportadora de retinol 4 (rbp4)
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
JP2025541245A (ja) * 2022-12-16 2025-12-18 アムジェン インコーポレイテッド Ｔｔｒ発現を阻害するためのｒｎａｉコンストラクト及びその使用方法
EP4673453A1 (en) * 2023-03-01 2026-01-07 Wave Life Sciences Ltd. Double stranded oligonucleotide compositions for rna interference and methods relating thereto
US20260009029A1 (en) * 2023-04-14 2026-01-08 Rigerna Therapeutics (Suzhou) Co., Ltd. Compound for inhibiting expression of lpa gene, pharmaceutical composition and use thereof
WO2025113470A1 (en) * 2023-11-27 2025-06-05 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of transthyretin (ttr)

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