EP3389672A4 - Compositions et procédés de traitement de maladies hépatiques - Google Patents

Compositions et procédés de traitement de maladies hépatiques Download PDF

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Publication number
EP3389672A4
EP3389672A4 EP16876543.6A EP16876543A EP3389672A4 EP 3389672 A4 EP3389672 A4 EP 3389672A4 EP 16876543 A EP16876543 A EP 16876543A EP 3389672 A4 EP3389672 A4 EP 3389672A4
Authority
EP
European Patent Office
Prior art keywords
compositions
treatment
methods
liver diseases
liver
Prior art date
Legal status (The legal status 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 status listed.)
Withdrawn
Application number
EP16876543.6A
Other languages
German (de)
English (en)
Other versions
EP3389672A1 (fr
Inventor
Isabel AZNAREZ
Huw M. Nash
Samuel W. HALL
Enxuan Jing
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Cold Spring Harbor Laboratory
Stoke Therapeutics Inc
Original Assignee
Cold Spring Harbor Laboratory
Stoke Therapeutics 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 Cold Spring Harbor Laboratory, Stoke Therapeutics Inc filed Critical Cold Spring Harbor Laboratory
Publication of EP3389672A1 publication Critical patent/EP3389672A1/fr
Publication of EP3389672A4 publication Critical patent/EP3389672A4/fr
Withdrawn legal-status Critical Current

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Classifications

    • 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
    • A61K31/712—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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • 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
    • C12N2310/34—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
EP16876543.6A 2015-12-14 2016-12-14 Compositions et procédés de traitement de maladies hépatiques Withdrawn EP3389672A4 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267238P 2015-12-14 2015-12-14
US201662319015P 2016-04-06 2016-04-06
PCT/US2016/066564 WO2017106283A1 (fr) 2015-12-14 2016-12-14 Compositions et procédés de traitement de maladies hépatiques

Publications (2)

Publication Number Publication Date
EP3389672A1 EP3389672A1 (fr) 2018-10-24
EP3389672A4 true EP3389672A4 (fr) 2019-08-14

Family

ID=59057460

Family Applications (1)

Application Number Title Priority Date Filing Date
EP16876543.6A Withdrawn EP3389672A4 (fr) 2015-12-14 2016-12-14 Compositions et procédés de traitement de maladies hépatiques

Country Status (4)

Country Link
EP (1) EP3389672A4 (fr)
JP (1) JP2019500345A (fr)
CA (1) CA3005090A1 (fr)
WO (1) WO2017106283A1 (fr)

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US11083444B2 (en) * 2010-03-11 2021-08-10 Globus Medical, Inc. Tissue retractor and methods of use
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016054615A2 (fr) 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Augmentation ciblée de la production de gènes nucléaires
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JP7049248B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー
JP7458785B2 (ja) 2017-01-23 2024-04-01 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒドロキシステロイド１７－βデヒドロゲナーゼ１３（ＨＳＤ１７Ｂ１３）バリアント及びその使用
CN110325649A (zh) 2017-04-11 2019-10-11 雷杰纳荣制药公司 用于筛选羟基类固醇(17-β)脱氢酶(HSD17B)家族成员的调节剂的活性的测定法
WO2019040923A1 (fr) 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. Oligomères antisens pour le traitement d'états pathologiques et autres maladies
EP3461837A1 (fr) * 2017-09-28 2019-04-03 Secarna Pharmaceuticals GmbH & Co. KG Inhibiteur inhibant l'expression du pprx1
JP7434151B2 (ja) 2017-10-11 2024-02-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｐｎｐｌａ３ ｉ１４８ｍの変異を発現している患者の肝疾患の治療におけるｈｓｄ１７ｂ１３の阻害
AU2019222767A1 (en) * 2018-02-14 2020-08-27 Deep Genomics Incorporated Oligonucleotide therapy for Wilson disease
TW202023573A (zh) * 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
US10913951B2 (en) * 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
CN111849968A (zh) * 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
CA3148158A1 (fr) * 2019-09-20 2021-03-25 Longcheng Li Molecule d'acide nucleique pour le traitement de la thrombocytopenie immunitaire et son application
JP2022552249A (ja) * 2019-10-14 2022-12-15 アストラゼネカ・アクチエボラーグ Ｐｎｐｌａ３発現のモジュレーター
TW202208627A (zh) 2020-05-11 2022-03-01 美商斯托克治療公司 用於病症及疾病之治療的opa1反義寡聚物
WO2021231698A1 (fr) * 2020-05-15 2021-11-18 Korro Bio, Inc. Procédés et compositions pour l'édition médiée par adar d'argininosuccinate lyase (asl)
EP4185696A1 (fr) * 2020-07-23 2023-05-31 F. Hoffmann-La Roche AG Oligonucléotides ciblant des sites de protéine de liaison à l'arn
WO2022109030A1 (fr) * 2020-11-17 2022-05-27 Skyhawk Therapeutics, Inc. Modifieurs d'épissage ciblant l'arn pour le traitement de pathologies et de maladies associées à pnpla3

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WO2007047913A2 (fr) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions et procédés pour la modulation de l'expression du gène lmna
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US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
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JANA KRALOVICOVA ET AL: "Optimal antisense target reducing INS intron 1 retention is adjacent to a parallel G quadruplex", NUCLEIC ACIDS RESEARCH, vol. 42, no. 12, 8 July 2014 (2014-07-08), pages 8161 - 8173, XP055211568, ISSN: 0305-1048, DOI: 10.1093/nar/gku507 *
See also references of WO2017106283A1 *

Also Published As

Publication number Publication date
JP2019500345A (ja) 2019-01-10
CA3005090A1 (fr) 2017-06-22
EP3389672A1 (fr) 2018-10-24
WO2017106283A1 (fr) 2017-06-22

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