EP3390634A4 - Compositions et méthodes pour le traitement de maladies oculaires - Google Patents
Compositions et méthodes pour le traitement de maladies oculaires Download PDFInfo
- 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
Links
Classifications
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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
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- 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
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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
- C12N15/111—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
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- 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
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 (20)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
SG11201702682PA (en) | 2014-10-03 | 2017-04-27 | Cold Spring Harbor Lab | Targeted augmentation of nuclear gene output |
CA3000971A1 (fr) | 2015-10-09 | 2017-04-13 | University Of Southampton | Modulation de l'expression genique et criblage de l'expression de proteines deregulee |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
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 |
SG11201906735RA (en) | 2017-01-23 | 2019-08-27 | Regeneron Pharma | Hydroxysteroid 17-beta dehydrogenase 13 (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 |
GB201706009D0 (en) * | 2017-04-13 | 2017-05-31 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of stargardt disease |
FI3673080T3 (fi) | 2017-08-25 | 2023-11-23 | Stoke Therapeutics Inc | Antisense-oligomeerejä häiriötilojen ja sairauksien hoitoon |
WO2019075181A1 (fr) | 2017-10-11 | 2019-04-18 | Regeneron Pharmaceuticals, Inc. | Inhibition de hsd17b13 dans le traitement de la maladie hépatique chez des patients exprimant la variation pnpla3 i148m |
KR20200070367A (ko) * | 2017-10-23 | 2020-06-17 | 스톡 테라퓨틱스, 인크. | 넌센스 매개 rna 분해 기반 병태 및 질환 치료를 위한 안티센스 올리고머 |
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 |
CA3103429A1 (fr) * | 2018-06-14 | 2019-12-19 | Don W. Cleveland | Composes et procedes permettant d'augmenter l'expression de stmn2 |
SG11202012759XA (en) | 2018-07-03 | 2021-01-28 | Hoffmann La Roche | Oligonucleotides for modulating tau expression |
CA3127483A1 (fr) * | 2019-01-25 | 2020-07-30 | Nayan Therapeutics, Inc. | Oligonucleotides reducteurs d'expression de nr2e3, compositions les contenant, et leurs procedes d'utilisation |
MX2022002198A (es) * | 2019-08-19 | 2022-05-24 | Stoke Therapeutics Inc | Composiciones y métodos para modular el empalme y la expresión de proteínas. |
US20230287410A1 (en) | 2020-05-11 | 2023-09-14 | Stoke Therapeutics, Inc. | Opa1 antisense oligomers for treatment of conditions and diseases |
WO2023104693A1 (fr) * | 2021-12-07 | 2023-06-15 | 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 |
Citations (1)
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)
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 |
SG11201702682PA (en) * | 2014-10-03 | 2017-04-27 | Cold Spring Harbor Lab | Targeted augmentation of nuclear gene output |
-
2016
- 2016-12-14 JP JP2018529249A patent/JP7036723B2/ja active Active
- 2016-12-14 WO PCT/US2016/066691 patent/WO2017106370A1/fr active Application Filing
- 2016-12-14 CA CA3005128A patent/CA3005128A1/fr active Pending
- 2016-12-14 EP EP16876610.3A patent/EP3390634A4/fr active Pending
-
2022
- 2022-01-04 JP JP2022000109A patent/JP2022046724A/ja active Pending
Patent Citations (1)
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)
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 |
---|---|
CA3005128A1 (fr) | 2017-06-22 |
JP2019500348A (ja) | 2019-01-10 |
JP2022046724A (ja) | 2022-03-23 |
EP3390634A1 (fr) | 2018-10-24 |
WO2017106370A1 (fr) | 2017-06-22 |
JP7036723B2 (ja) | 2022-03-15 |
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RIN1 | Information on inventor provided before grant (corrected) |
Inventor name: AZNAREZ, ISABEL Inventor name: NASH, HUW M. Inventor name: HALL, SAMUEL W. Inventor name: AZARIAN, SASSAN |
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DAX | Request for extension of the european patent (deleted) | ||
A4 | Supplementary search report drawn up and despatched |
Effective date: 20190711 |
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RIC1 | Information provided on ipc code assigned before grant |
Ipc: C12N 15/11 20060101ALI20190705BHEP Ipc: C12N 15/113 20100101AFI20190705BHEP |
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Inventor name: KRAINER,ADRIAN Inventor name: AZNAREZ, ISABEL Inventor name: AZARIAN, SASSAN Inventor name: NASH, HUW M. Inventor name: HALL, SAMUEL W. |
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