EP3390634A4 - Compositions et méthodes pour le traitement de maladies oculaires - Google Patents

Compositions et méthodes pour le traitement de maladies oculaires Download PDF

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Publication number
EP3390634A4
EP3390634A4 EP16876610.3A EP16876610A EP3390634A4 EP 3390634 A4 EP3390634 A4 EP 3390634A4 EP 16876610 A EP16876610 A EP 16876610A EP 3390634 A4 EP3390634 A4 EP 3390634A4
Authority
EP
European Patent Office
Prior art keywords
compositions
treatment
methods
eye diseases
diseases
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
EP16876610.3A
Other languages
German (de)
English (en)
Other versions
EP3390634A1 (fr
Inventor
Isabel AZNAREZ
Huw M. Nash
Samuel W. HALL
Sassan Azarian
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 EP3390634A1 publication Critical patent/EP3390634A1/fr
Publication of EP3390634A4 publication Critical patent/EP3390634A4/fr
Pending 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
EP16876610.3A 2015-12-14 2016-12-14 Compositions et méthodes pour le traitement de maladies oculaires Pending EP3390634A4 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267259P 2015-12-14 2015-12-14
US201662318958P 2016-04-06 2016-04-06
PCT/US2016/066691 WO2017106370A1 (fr) 2015-12-14 2016-12-14 Compositions et méthodes pour le traitement de maladies oculaires

Publications (2)

Publication Number Publication Date
EP3390634A1 EP3390634A1 (fr) 2018-10-24
EP3390634A4 true EP3390634A4 (fr) 2019-08-14

Family

ID=59057531

Family Applications (1)

Application Number Title Priority Date Filing Date
EP16876610.3A Pending EP3390634A4 (fr) 2015-12-14 2016-12-14 Compositions et méthodes pour le traitement de maladies oculaires

Country Status (4)

Country Link
EP (1) EP3390634A4 (fr)
JP (2) JP7036723B2 (fr)
CA (1) CA3005128A1 (fr)
WO (1) WO2017106370A1 (fr)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CA2963288A1 (fr) 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Augmentation ciblee de la production de genes nucleaires
US10196639B2 (en) 2015-10-09 2019-02-05 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3390636B1 (fr) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Oligomères antisens destinés au traitement du syndrome de dravet
KR102619197B1 (ko) 2017-01-23 2024-01-03 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
WO2019040923A1 (fr) 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. Oligomères antisens pour le traitement d'états pathologiques et autres maladies
KR20240125690A (ko) 2017-10-11 2024-08-19 리제너론 파마슈티칼스 인코포레이티드 Pnpla3 i148m 변이를 발현하는 환자의 간 질환의 치료에서의 hsd17b13의 저해
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
RU177889U1 (ru) * 2017-11-30 2018-03-15 Общество с ограниченной ответственностью "Вертикаль-М" (ООО "Вертикаль-М") Дренаж для хирургического лечения глаукомы
EP3781213A4 (fr) * 2018-04-17 2022-10-19 The Trustees of the University of Pennsylvania Molécules de trans-épissage
MX2020011695A (es) 2018-05-04 2021-02-26 Stoke Therapeutics Inc Métodos y composiciones para el tratamiento de la enfermedad por almacenamiento de éster de colesterilo.
AU2019287635A1 (en) * 2018-06-14 2020-12-17 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
PE20210346A1 (es) * 2018-07-03 2021-02-25 Hoffmann La Roche Oligonucleotidos para modular la expresion de tau
JP2022523065A (ja) * 2019-01-25 2022-04-21 ナヤン セラピューティクス， インク． Ｎｒ２ｅ３発現抑制オリゴヌクレオチド、それを含む組成物、及びそれらの使用方法
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
EP4150092A1 (fr) 2020-05-11 2023-03-22 Stoke Therapeutics, Inc. Oligomères antisens opa1 pour le traitement de pathologies et de maladies
EP4263831A1 (fr) 2020-12-18 2023-10-25 F. Hoffmann-La Roche AG Oligonucléotides antisens utilisés pour le ciblage de la progranuline
EP4444882A1 (fr) * 2021-12-07 2024-10-16 F. Hoffmann-La Roche AG Oligonucléotides antisens ciblant actl6b
WO2023160454A1 (fr) * 2022-02-25 2023-08-31 北京中因科技有限公司 Combinaison de cassette d'expression et son utilisation
WO2024105673A1 (fr) * 2022-11-17 2024-05-23 Skip Therapeutics Ltd. Compositions et méthodes pour le traitement de maladies rétiniennes

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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2630241B1 (fr) * 2010-10-22 2018-10-17 CuRNA, Inc. Traitement de maladies associées à l'alpha-l-iduronidase (idua) par inhibition du transcrit antisens endogène de idua
WO2014121287A2 (fr) * 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Composés antisens sélectifs et leurs utilisations
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CA2963288A1 (fr) * 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Augmentation ciblee de la production de genes nucleaires

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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
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 *
TAKEMASA SAKAGUCHI ET AL: "Optineurin with amyotrophic lateral sclerosis-related mutations abrogates inhibition of interferon regulatory factor-3 activation", NEUROSCIENCE LETTERS, ELSEVIER, AMSTERDAM, NL, vol. 505, no. 3, 15 October 2011 (2011-10-15), pages 279 - 281, XP028114208, ISSN: 0304-3940, [retrieved on 20111021], DOI: 10.1016/J.NEULET.2011.10.040 *

Also Published As

Publication number Publication date
JP7036723B2 (ja) 2022-03-15
EP3390634A1 (fr) 2018-10-24
JP2019500348A (ja) 2019-01-10
WO2017106370A1 (fr) 2017-06-22
CA3005128A1 (fr) 2017-06-22
JP2022046724A (ja) 2022-03-23

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