EP3390666A4 - COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES - Google Patents

COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES 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
methods
renal diseases
treating renal
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Withdrawn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
EP16876548.5A
Other languages
German (de)
English (en)
French (fr)
Other versions
EP3390666A1 (en
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/en
Publication of EP3390666A4 publication Critical patent/EP3390666A4/en
Withdrawn legal-status Critical Current

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    • 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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
EP16876548.5A 2015-12-14 2016-12-14 COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES Withdrawn EP3390666A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267242P 2015-12-14 2015-12-14
PCT/US2016/066576 WO2017106292A1 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of kidney diseases

Publications (2)

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

Family

ID=59057520

Family Applications (1)

Application Number Title Priority Date Filing Date
EP16876548.5A Withdrawn EP3390666A4 (en) 2015-12-14 2016-12-14 COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES

Country Status (4)

Country Link
EP (1) EP3390666A4 (cg-RX-API-DMAC7.html)
JP (2) JP7049247B2 (cg-RX-API-DMAC7.html)
CA (1) CA3005249A1 (cg-RX-API-DMAC7.html)
WO (1) WO2017106292A1 (cg-RX-API-DMAC7.html)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
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
KR102422625B1 (ko) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN118325899A (zh) 2017-01-23 2024-07-12 瑞泽恩制药公司 Hsd17b13变体及其应用
RU2019135845A (ru) 2017-04-11 2021-05-11 Ридженерон Фармасьютикалз, Инк. Анализы для скрининга активности модуляторов членов семейства (17-бета)гидроксистероиддегидрогеназ (hsd17b)
CN111278991B (zh) 2017-08-25 2022-04-01 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
EP4234719B1 (en) 2017-10-11 2025-11-26 Regeneron Pharmaceuticals, Inc. Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2019236995A1 (en) * 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
US12422440B2 (en) 2018-10-05 2025-09-23 Seattle Children's Hospital Newborn screening for primary immunodeficiencies, cystinosis, and Wilson disease
WO2020154462A1 (en) * 2019-01-23 2020-07-30 Rogcon U.R., Inc. Antisense oligonucleotides targeting scn2a retained introns
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression
JP2023519969A (ja) 2020-03-31 2023-05-15 シアトル・チルドレンズ・ホスピタル・ドゥーイング／ビジネス／アズ・シアトル・チルドレンズ・リサーチ・インスティテュート リソソーム蓄積症に関するプロテオミクススクリーニング
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015035091A1 (en) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

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
CA2588646A1 (en) * 2004-11-19 2006-05-26 Acadia Pharmaceuticals Inc. Methods to identify ligands of 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
US9909128B2 (en) * 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
CN107109411B (zh) * 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015035091A1 (en) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

Also Published As

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

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