EP3389672A4 - COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES - Google Patents

COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES 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Withdrawn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)
French (fr)
Other versions
EP3389672A1 (en
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/en
Publication of EP3389672A4 publication Critical patent/EP3389672A4/en
Withdrawn legal-status Critical Current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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
EP16876543.6A 2015-12-14 2016-12-14 COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES Withdrawn EP3389672A4 (en)

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 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of liver diseases

Publications (2)

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

Family

ID=59057460

Family Applications (1)

Application Number Title Priority Date Filing Date
EP16876543.6A Withdrawn EP3389672A4 (en) 2015-12-14 2016-12-14 COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES

Country Status (4)

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

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GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
IL308174A (en) 2015-10-09 2024-01-01 Univ Southampton Gene expression modulation and dysregulated protein expression scanning
CA3005256A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CA3047429A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hsd17b13 variants and uses thereof
AU2018250727A1 (en) 2017-04-11 2019-10-31 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (HSD17B) family
HUE064635T2 (hu) 2017-08-25 2024-04-28 Stoke Therapeutics Inc Antisensz oligomerek állapotok és betegségek kezelésére
EP3461837A1 (en) * 2017-09-28 2019-04-03 Secarna Pharmaceuticals GmbH & Co. KG Inhibitor inhibiting the expression of pprx1
RU2020115526A (ru) 2017-10-11 2021-11-12 Ридженерон Фармасьютикалз, Инк. Ингибирование hsd17b13 при лечении заболевания печени у пациентов, экспрессирующих вариацию i148m pnpla3
GB2610100B (en) 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
US11578327B2 (en) * 2018-02-14 2023-02-14 Deep Genomics Incorporated Oligonucleotide therapy for Wilson disease
CN112020559A (zh) * 2018-02-21 2020-12-01 百时美施贵宝公司 Camk2d反义寡核苷酸及其用途
EP3788169A4 (en) 2018-05-04 2022-08-10 Stoke Therapeutics, Inc. METHODS AND COMPOSITIONS FOR TREATING A CHOLESTERYL ESTER STORAGE 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
AU2020334067A1 (en) * 2019-08-19 2022-03-17 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression
PE20220604A1 (es) * 2019-09-20 2022-04-25 Ractigen Therapeutics Molecula de acido nucleico para el tratamiento de la trombocitopenia y uso de la misma
WO2021074772A1 (en) * 2019-10-14 2021-04-22 Astrazeneca Ab Modulators of pnpla3 expression
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
EP4150078A1 (en) * 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2022018187A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucleotides targeting rna binding protein sites
WO2022109030A1 (en) * 2020-11-17 2022-05-27 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases

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US4866042A (en) 1987-11-18 1989-09-12 Neuwelt Edward A Method for the delivery of genetic material across the blood brain barrier
US6294520B1 (en) 1989-03-27 2001-09-25 Albert T. Naito Material for passage through the blood-brain barrier
US5151510A (en) 1990-04-20 1992-09-29 Applied Biosystems, Inc. Method of synethesizing sulfurized oligonucleotide analogs
DE69334238D1 (de) 1992-09-25 2008-09-25 Aventis Pharma Sa Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn
US5656612A (en) 1994-05-31 1997-08-12 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
FR2727867B1 (fr) 1994-12-13 1997-01-31 Rhone Poulenc Rorer Sa Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux
US6936589B2 (en) 2001-09-28 2005-08-30 Albert T. Naito Parenteral delivery systems
WO2004001010A2 (en) * 2002-06-21 2003-12-31 Ptc Therapeutics, Inc. METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY
EP1874958B1 (en) * 2005-04-01 2019-03-27 University of Florida Research Foundation, Incorporated Biomarkers of liver injury
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
EP3141265A1 (en) 2007-12-04 2017-03-15 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
EP3449926B1 (en) 2009-06-17 2019-10-09 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
WO2012138487A2 (en) * 2011-04-07 2012-10-11 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
WO2015035091A1 (en) 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

Non-Patent Citations (3)

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Title
HOMEDAN CHADI ET AL: "Acute intermittent porphyria causes hepatic mitochondrial energetic failure in a mouse model", INTERNATIONAL JOURNAL OF BIOCHEMISTRY AND CELL BIOLOGY, PERGAMON, GB, vol. 51, 13 April 2014 (2014-04-13), pages 93 - 101, XP029026620, ISSN: 1357-2725, DOI: 10.1016/J.BIOCEL.2014.03.032 *
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
EP3389672A1 (en) 2018-10-24
CA3005090A1 (en) 2017-06-22
JP2019500345A (ja) 2019-01-10
WO2017106283A1 (en) 2017-06-22

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