CL2018001221A1 - Modular la expresión de apolipoproteína (a) - Google Patents

Modular la expresión de apolipoproteína (a)

Info

Publication number
CL2018001221A1
CL2018001221A1 CL2018001221A CL2018001221A CL2018001221A1 CL 2018001221 A1 CL2018001221 A1 CL 2018001221A1 CL 2018001221 A CL2018001221 A CL 2018001221A CL 2018001221 A CL2018001221 A CL 2018001221A CL 2018001221 A1 CL2018001221 A1 CL 2018001221A1
Authority
CL
Chile
Prior art keywords
apo
disorders
oligomeric compounds
conjugated
diseases
Prior art date
Application number
CL2018001221A
Other languages
English (en)
Inventor
Nicholas J Viney
Richard S Geary
Yanfeng Wang
Zhengrong Yu
Rudy Gunawan
Original Assignee
Ionis Pharmaceuticals Inc
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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of CL2018001221A1 publication Critical patent/CL2018001221A1/es

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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
    • A61J—CONTAINERS SPECIALLY ADAPTED FOR MEDICAL OR PHARMACEUTICAL PURPOSES; DEVICES OR METHODS SPECIALLY ADAPTED FOR BRINGING PHARMACEUTICAL PRODUCTS INTO PARTICULAR PHYSICAL OR ADMINISTERING FORMS; DEVICES FOR ADMINISTERING FOOD OR MEDICINES ORALLY; BABY COMFORTERS; DEVICES FOR RECEIVING SPITTLE
    • A61J1/00—Containers specially adapted for medical or pharmaceutical purposes
    • A61J1/05—Containers specially adapted for medical or pharmaceutical purposes for collecting, storing or administering blood, plasma or medical fluids ; Infusion or perfusion containers
    • 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
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775—Apolipopeptides
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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
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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
    • C12N2310/321—2'-O-R Modification
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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
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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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    • C12N2320/00—Applications; Uses
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    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Abstract

EN LA PRESENTE, SE PROPORCIONAN COMPUESTOS OLIGOMÉRICOS CON GRUPOS CONJUGADOS DIRIGIDOS A APOLIPOPROTEÍNA (A) [APO(A)]. EN DETERMINADAS MODALIDADES, LOS COMPUESTOS OLIGOMÉRICOS DIRIGIDOS A APO(A) ESTÁN CONJUGADOS A N-ACETILGALACTOSAMINA. ADEMÁS, EN LA PRESENTE SE DESCRIBEN COMPUESTOS OLIGOMÉRICOS CONJUGADOS DIRIGIDOS A APO(A) PARA SU USO PARA DISMINUIR LA APO(A) PARA TRATAR, PREVENIR O MEJORAR ENFERMEDADES, TRASTORNOS O AFECCIONES RELACIONADAS CON APO(A) Y/O LP(A). DETERMINADAS ENFERMEDADES, TRASTORNOS O AFECCIONES RELACIONADAS CON APO(A) Y/O LP(A) INCLUYEN ENFERMEDADES, TRASTORNOS O AFECCIONES INFLAMATORIOS, CARDIOVASCULARES Y/O METABÓLICOS. LOS COMPUESTOS OLIGOMÉRICOS CONJUGADOS DESCRITOS EN LA PRESENTE SE PUEDEN UTILIZAR PARA TRATAR DICHAS ENFERMEDADES, TRASTORNOS O AFECCIONES EN UN INDIVIDUO QUE LO NECESITA.
CL2018001221A 2015-11-06 2018-05-04 Modular la expresión de apolipoproteína (a) CL2018001221A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562252392P 2015-11-06 2015-11-06

Publications (1)

Publication Number Publication Date
CL2018001221A1 true CL2018001221A1 (es) 2018-12-28

Family

ID=58662931

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2018001221A CL2018001221A1 (es) 2015-11-06 2018-05-04 Modular la expresión de apolipoproteína (a)

Country Status (15)

Country Link
US (2) US10557137B2 (es)
EP (1) EP3370708A4 (es)
JP (3) JP2018531605A (es)
KR (1) KR20180073606A (es)
CN (2) CN108348478A (es)
AU (2) AU2016349625B2 (es)
BR (2) BR112018003291A2 (es)
CA (1) CA2999341A1 (es)
CL (1) CL2018001221A1 (es)
CO (1) CO2018002198A2 (es)
IL (2) IL257647B (es)
MX (1) MX2018005277A (es)
PE (1) PE20181180A1 (es)
WO (1) WO2017079739A1 (es)
ZA (1) ZA201801208B (es)

Families Citing this family (5)

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Publication number Priority date Publication date Assignee Title
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
IL284593B2 (en) * 2013-05-01 2023-02-01 Ionis Pharmaceuticals Inc Compositions and methods for modulation of hbv and ttr expression
US20190046555A1 (en) * 2015-11-06 2019-02-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
KR20210091703A (ko) 2018-11-09 2021-07-22 노파르티스 아게 apo(a)를 표적화하는 콘쥬게이션된 안티센스 화합물로 심혈관 사건의 위험을 감소시키는 방법
WO2023180904A1 (en) * 2022-03-21 2023-09-28 Crispr Therapeutics Ag Methods and compositions for treating lipoprotein-related diseases

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