HRP20231606T1 - Spojevi i metode za smanjenje ekspresije tau - Google Patents

Spojevi i metode za smanjenje ekspresije tau Download PDF

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Publication number
HRP20231606T1
HRP20231606T1 HRP20231606TT HRP20231606T HRP20231606T1 HR P20231606 T1 HRP20231606 T1 HR P20231606T1 HR P20231606T T HRP20231606T T HR P20231606TT HR P20231606 T HRP20231606 T HR P20231606T HR P20231606 T1 HRP20231606 T1 HR P20231606T1
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HR
Croatia
Prior art keywords
modified oligonucleotide
salt
compound
pharmaceutical preparation
disease
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Application number
HRP20231606TT
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English (en)
Croatian (hr)
Inventor
Holly Kordasiewicz
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Biogen Ma Inc.
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Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=61760152&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HRP20231606(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biogen Ma Inc. filed Critical Biogen Ma Inc.
Publication of HRP20231606T1 publication Critical patent/HRP20231606T1/hr

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    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/46—Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/31—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
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone 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
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Psychology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pain & Pain Management (AREA)
  • Dermatology (AREA)
  • Inorganic Chemistry (AREA)
  • Botany (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
HRP20231606TT 2016-09-29 2017-09-29 Spojevi i metode za smanjenje ekspresije tau HRP20231606T1 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662401723P 2016-09-29 2016-09-29
US201762450469P 2017-01-25 2017-01-25
EP17857565.0A EP3519572B1 (en) 2016-09-29 2017-09-29 Compounds and methods for reducing tau expression
PCT/US2017/054540 WO2018064593A1 (en) 2016-09-29 2017-09-29 Compounds and methods for reducing tau expression

Publications (1)

Publication Number Publication Date
HRP20231606T1 true HRP20231606T1 (hr) 2024-03-15

Family

ID=61760152

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20231606TT HRP20231606T1 (hr) 2016-09-29 2017-09-29 Spojevi i metode za smanjenje ekspresije tau

Country Status (32)

Country Link
US (3) US10407680B2 (cg-RX-API-DMAC7.html)
EP (2) EP4335923A3 (cg-RX-API-DMAC7.html)
JP (4) JP6738004B2 (cg-RX-API-DMAC7.html)
KR (2) KR102286223B1 (cg-RX-API-DMAC7.html)
CN (2) CN113481201A (cg-RX-API-DMAC7.html)
AU (3) AU2017336093B2 (cg-RX-API-DMAC7.html)
BR (2) BR122020019785B1 (cg-RX-API-DMAC7.html)
CA (1) CA3038891C (cg-RX-API-DMAC7.html)
CL (2) CL2019000703A1 (cg-RX-API-DMAC7.html)
CO (1) CO2019001385A2 (cg-RX-API-DMAC7.html)
CR (2) CR20190214A (cg-RX-API-DMAC7.html)
DK (1) DK3519572T5 (cg-RX-API-DMAC7.html)
ES (1) ES2963428T3 (cg-RX-API-DMAC7.html)
FI (1) FI3519572T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20231606T1 (cg-RX-API-DMAC7.html)
HU (1) HUE065170T2 (cg-RX-API-DMAC7.html)
IL (3) IL299615A (cg-RX-API-DMAC7.html)
JO (1) JOP20190065A1 (cg-RX-API-DMAC7.html)
LT (1) LT3519572T (cg-RX-API-DMAC7.html)
MD (1) MD3519572T2 (cg-RX-API-DMAC7.html)
MX (2) MX2019003633A (cg-RX-API-DMAC7.html)
MY (1) MY210293A (cg-RX-API-DMAC7.html)
NZ (1) NZ750642A (cg-RX-API-DMAC7.html)
PE (1) PE20190655A1 (cg-RX-API-DMAC7.html)
PL (1) PL3519572T3 (cg-RX-API-DMAC7.html)
PT (1) PT3519572T (cg-RX-API-DMAC7.html)
RS (1) RS64941B1 (cg-RX-API-DMAC7.html)
SG (1) SG11201901140QA (cg-RX-API-DMAC7.html)
SI (1) SI3519572T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202300441T1 (cg-RX-API-DMAC7.html)
WO (1) WO2018064593A1 (cg-RX-API-DMAC7.html)
ZA (2) ZA201900920B (cg-RX-API-DMAC7.html)

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EP2831232A4 (en) 2012-03-30 2015-11-04 Univ Washington METHODS OF MODULATING THE EXPRESSION OF TAU TO REDUCE THE STROKE AND MODIFY A NEURODEGENERATIVE SYMPTOM
WO2013159108A2 (en) * 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
KR20150130430A (ko) 2013-03-14 2015-11-23 아이시스 파마수티컬즈 인코포레이티드 타우 발현을 조절하는 조성물 및 방법
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
JOP20190065A1 (ar) * 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
US11447775B2 (en) 2018-01-12 2022-09-20 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN111819283A (zh) 2018-01-12 2020-10-23 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
SG11202012759XA (en) * 2018-07-03 2021-01-28 Hoffmann La Roche Oligonucleotides for modulating tau expression
US20220195437A1 (en) * 2020-12-11 2022-06-23 Eisai R&D Management Co., Ltd. Tau-targeting oligonucleotide gapmers
UY39713A (es) 2021-04-01 2022-10-31 Biogen Ma Inc Administracion de acido nucleico al sistema nervioso central
JP2024523364A (ja) * 2021-06-18 2024-06-28 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｉｆｎａｒ１発現を低減するための化合物及び方法
WO2023004426A2 (en) * 2021-07-23 2023-01-26 University Of Kentucky Research Foundation Inhibition of neurofibrillary tangles using oligonucleotides against circular rnas from the microtubule associated protein tau (mapt) locus
IL310332A (en) * 2021-07-23 2024-03-01 Biogen Ma Inc Methods for reducing TAU expression
WO2025043202A1 (en) * 2023-08-23 2025-02-27 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atn1 expression

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