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
methods
hepatic diseases
treating hepatic
treating
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Withdrawn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)
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Withdrawn legal-status Critical Current

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    • 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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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 (en)
JP (1) JP2019500345A (en)
CA (1) CA3005090A1 (en)
WO (1) WO2017106283A1 (en)

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GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
KR102604132B1 (en) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 Antisense oligomers for the 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
KR102712656B1 (en) 2017-01-23 2024-10-04 리제너론 파마슈티칼스 인코포레이티드 HSD17B13 Variants And Uses Thereof
US11479802B2 (en) 2017-04-11 2022-10-25 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family
SI3673080T1 (en) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
EP3461837A1 (en) * 2017-09-28 2019-04-03 Secarna Pharmaceuticals GmbH & Co. KG Inhibitor inhibiting the expression of pprx1
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
HRP20250322T1 (en) * 2017-10-23 2025-06-06 Stoke Therapeutics, Inc. ANTI-SENSE OLIGOMERS, INTENDED FOR THE TREATMENT OF CONDITIONS AND DISEASES CAUSED BY RNA DEGRADATION MEDIATED BY NONSENSE MUTATIONS
AU2019222767A1 (en) 2018-02-14 2020-08-27 Deep Genomics Incorporated Oligonucleotide therapy for Wilson disease
IL320427A (en) * 2018-02-21 2025-06-01 Bristol Myers Squibb Co Camk2d antisense oligonucleotides and uses thereof
BR112020018758A2 (en) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. double-stranded ribonucleic acid agent, cell, vector, pharmaceutical composition, and, methods for inhibiting the expression of 17¿-hydroxysteroid dehydrogenases type 13, for treatment of an individual, for prevention of a symptom in an individual, for risk reduction to develop chronic liver disease, to inhibit steatosis progression, to inhibit the accumulation of lipid droplets
BR112020022512A2 (en) 2018-05-04 2021-05-04 Stoke Therapeutics, Inc. methods and compositions for treating cholesteryl ester storage disease
TWI869213B (en) * 2018-09-19 2025-01-01 美商Ionis製藥公司 Modulators of pnpla3 expression
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 (en) * 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 Oligonucleotide molecule and application thereof in acute intermittent porphyria treatment
CN114746550A (en) * 2019-08-19 2022-07-12 斯托克制药公司 Compositions and methods for modulating splicing and protein expression
CN114585738A (en) * 2019-09-20 2022-06-03 中美瑞康核酸技术(南通)研究院有限公司 Nucleic acid molecules for treating thrombocytopenia and their applications
JP7757277B2 (en) * 2019-10-14 2025-10-21 アストラゼネカ・アクチエボラーグ Modulators of PNPLA3 Expression
WO2021113773A2 (en) 2019-12-04 2021-06-10 Pai Athma A Identifying non-productive splice sites
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
WO2021231698A1 (en) * 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
JP2023534557A (en) * 2020-07-23 2023-08-09 エフ． ホフマン－ラ ロシュ アーゲー 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
FI951404A7 (en) 1992-09-25 1995-03-24 Rhone Poulenc Rorer Sa Adenovirus vectors for transferring foreign genes into cells of the central nervous system, especially the brain
US5656612A (en) 1994-05-31 1997-08-12 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
FR2727867B1 (en) 1994-12-13 1997-01-31 Rhone Poulenc Rorer Sa GENE TRANSFER IN MEDULLAR MOTONURONES USING ADENOVIRAL VECTORS
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
WO2006107846A2 (en) * 2005-04-01 2006-10-12 University Of Florida Research Foundation, Inc. Biomakers of liver injury
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
EP3705125B1 (en) 2007-12-04 2023-07-05 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
SI3449926T1 (en) 2009-06-17 2020-04-30 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
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
CA2930859C (en) 2013-09-04 2022-05-03 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

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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
JP2019500345A (en) 2019-01-10
WO2017106283A1 (en) 2017-06-22
CA3005090A1 (en) 2017-06-22
EP3389672A1 (en) 2018-10-24

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