EP3390666A4 - Compositions et méthodes de traitement de maladies rénales - Google Patents

Compositions et méthodes de traitement de maladies rénales Download PDF

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Publication number
EP3390666A4
EP3390666A4 EP16876548.5A EP16876548A EP3390666A4 EP 3390666 A4 EP3390666 A4 EP 3390666A4 EP 16876548 A EP16876548 A EP 16876548A EP 3390666 A4 EP3390666 A4 EP 3390666A4
Authority
EP
European Patent Office
Prior art keywords
compositions
treatment
methods
kidney diseases
kidney
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.)
Pending
Application number
EP16876548.5A
Other languages
German (de)
English (en)
Other versions
EP3390666A1 (fr
Inventor
Isabel AZNAREZ
Huw M. Nash
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 EP3390666A1 publication Critical patent/EP3390666A1/fr
Publication of EP3390666A4 publication Critical patent/EP3390666A4/fr
Pending legal-status Critical Current

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Classifications

    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Urology & Nephrology (AREA)
  • Public Health (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
EP16876548.5A 2015-12-14 2016-12-14 Compositions et méthodes de traitement de maladies rénales Pending EP3390666A4 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267242P 2015-12-14 2015-12-14
PCT/US2016/066576 WO2017106292A1 (fr) 2015-12-14 2016-12-14 Compositions et méthodes de traitement de maladies rénales

Publications (2)

Publication Number Publication Date
EP3390666A1 EP3390666A1 (fr) 2018-10-24
EP3390666A4 true EP3390666A4 (fr) 2019-08-07

Family

ID=59057520

Family Applications (1)

Application Number Title Priority Date Filing Date
EP16876548.5A Pending EP3390666A4 (fr) 2015-12-14 2016-12-14 Compositions et méthodes de traitement de maladies rénales

Country Status (4)

Country Link
EP (1) EP3390666A4 (fr)
JP (2) JP7049247B2 (fr)
CA (1) CA3005249A1 (fr)
WO (1) WO2017106292A1 (fr)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015187825A2 (fr) 2014-06-03 2015-12-10 University Of Massachusetts Compositions et méthodes de modulation de l'expression de la dysferline
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
JP6867945B2 (ja) 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強
US10196639B2 (en) 2015-10-09 2019-02-05 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
US11083745B2 (en) 2015-12-14 2021-08-10 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
IL301053A (en) 2017-01-23 2023-05-01 Regeneron Pharma HSD17B13 variants and their uses
SG11201909453RA (en) 2017-04-11 2019-11-28 Regeneron Pharma Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
GB2594767B (en) 2017-08-25 2022-06-22 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
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
US20210246443A1 (en) * 2018-06-08 2021-08-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2020154462A1 (fr) * 2019-01-23 2020-07-30 Rogcon U.R., Inc. Oligonucléotides antisens ciblant des introns de scn2a conservés
WO2021034985A1 (fr) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions et méthodes pour moduler l'épissage et l'expression de protéines
EP4126966A1 (fr) 2020-03-31 2023-02-08 Seattle Children's Hospital d/b/a Seattle Children's Research Institute Dépistage protéomique de maladies lysosomales
JP2023525799A (ja) 2020-05-11 2023-06-19 ストーク セラピューティクス，インク． 状態及び疾患の治療のためのｏｐａ１アンチセンスオリゴマー

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015035091A1 (fr) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Réduction de dégradation d'arnm à médiation non-sens

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030224514A1 (en) * 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of PPAR-delta expression
US20060134670A1 (en) * 2004-11-19 2006-06-22 Fabrice Piu Enabling tools to identify ligands for hormone nuclear receptors
US20110269735A1 (en) * 2010-04-19 2011-11-03 Celera Corporation Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
WO2014078749A1 (fr) * 2012-11-15 2014-05-22 The Regents Of The University Of California Oligonucléotides modulant l'épissage qui inhibent le cancer
JP6867945B2 (ja) * 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015035091A1 (fr) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Réduction de dégradation d'arnm à médiation non-sens

Also Published As

Publication number Publication date
JP2022062143A (ja) 2022-04-19
CA3005249A1 (fr) 2017-06-22
EP3390666A1 (fr) 2018-10-24
JP2019500346A (ja) 2019-01-10
JP7049247B2 (ja) 2022-04-06
WO2017106292A1 (fr) 2017-06-22

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