CO2020008425A2 - Oligonucleótidos antisentido que actúan sobre alfa-sinucleína, y usos de estos - Google Patents

Oligonucleótidos antisentido que actúan sobre alfa-sinucleína, y usos de estos

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Publication number
CO2020008425A2
CO2020008425A2 CONC2020/0008425A CO2020008425A CO2020008425A2 CO 2020008425 A2 CO2020008425 A2 CO 2020008425A2 CO 2020008425 A CO2020008425 A CO 2020008425A CO 2020008425 A2 CO2020008425 A2 CO 2020008425A2
Authority
CO
Colombia
Prior art keywords
act
antisense oligonucleotides
synuclein
alpha
protein
Prior art date
Application number
CONC2020/0008425A
Other languages
English (en)
Inventor
Richard E Olson
Peter Hagedorn
Angela M Cacace
Jere E Meredith Jr
Nino Devidze
James K Loy
Carl J Baldick
Annapurna Pendri
Ivar M Mcdonald
Marianne Lerbech Jensen
Original Assignee
Bristol Myers Squibb Co
Roche Innovation Ct Copenhagen As
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 Bristol Myers Squibb Co, Roche Innovation Ct Copenhagen As filed Critical Bristol Myers Squibb Co
Publication of CO2020008425A2 publication Critical patent/CO2020008425A2/es

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    • 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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • 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
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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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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 ===---===
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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

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  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

La presente divulgación se refiere a oligonucleótidos antisentido, que actúan sobre mARN de SNCA (por ejemplo, en una unión intrón-exón) en una célula, que tiene como resultado una expresión reducida de la proteína SNCA. La reducción de la expresión de la proteína SCNA es beneficiosa para el tratamiento de determinados trastornos médicos, por ejemplo, un trastorno neurológico.
CONC2020/0008425A 2018-01-12 2020-08-05 Oligonucleótidos antisentido que actúan sobre alfa-sinucleína, y usos de estos CO2020008425A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862616937P 2018-01-12 2018-01-12
PCT/US2019/013249 WO2019140231A1 (en) 2018-01-12 2019-01-11 Antisense oligonucleotides targeting alpha-synuclein and uses thereof

Publications (1)

Publication Number Publication Date
CO2020008425A2 true CO2020008425A2 (es) 2020-08-21

Family

ID=65269095

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2020/0008425A CO2020008425A2 (es) 2018-01-12 2020-08-05 Oligonucleótidos antisentido que actúan sobre alfa-sinucleína, y usos de estos

Country Status (15)

Country Link
US (3) US11447775B2 (es)
EP (1) EP3737387A1 (es)
JP (2) JP7455746B2 (es)
KR (1) KR20200140793A (es)
CN (1) CN111836624A (es)
AU (1) AU2019208006A1 (es)
BR (1) BR112020013994A2 (es)
CA (1) CA3088112A1 (es)
CO (1) CO2020008425A2 (es)
EA (1) EA202091695A1 (es)
IL (1) IL275902A (es)
MX (1) MX2020007433A (es)
PE (1) PE20201501A1 (es)
SG (1) SG11202006526YA (es)
WO (1) WO2019140231A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
PE20201501A1 (es) 2018-01-12 2020-12-29 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
KR20200140240A (ko) * 2018-01-12 2020-12-15 브리스톨-마이어스 스큅 컴퍼니 알파-시누클레인을 표적화하는 안티센스 올리고뉴클레오티드 및 그의 용도
AU2019218987A1 (en) 2018-02-12 2020-07-23 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof

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