CA3005128A1 - Compositions and methods for treatment of eye diseases - Google Patents
Compositions and methods for treatment of eye diseases Download PDFInfo
- Publication number
- CA3005128A1 CA3005128A1 CA3005128A CA3005128A CA3005128A1 CA 3005128 A1 CA3005128 A1 CA 3005128A1 CA 3005128 A CA3005128 A CA 3005128A CA 3005128 A CA3005128 A CA 3005128A CA 3005128 A1 CA3005128 A1 CA 3005128A1
- Authority
- CA
- Canada
- Prior art keywords
- seq
- mrna
- nucleobases
- nos
- ric pre
- 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
-
- 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)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267259P | 2015-12-14 | 2015-12-14 | |
| US62/267,259 | 2015-12-14 | ||
| US201662318958P | 2016-04-06 | 2016-04-06 | |
| US62/318,958 | 2016-04-06 | ||
| PCT/US2016/066691 WO2017106370A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of eye diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3005128A1 true CA3005128A1 (en) | 2017-06-22 |
Family
ID=59057531
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3005128A Pending CA3005128A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of eye diseases |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP3390634A4 (https=) |
| JP (2) | JP7036723B2 (https=) |
| CA (1) | CA3005128A1 (https=) |
| WO (1) | WO2017106370A1 (https=) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EP3359685B1 (en) | 2015-10-09 | 2026-01-28 | University Of Southampton | Modulation of gene expression for deregulated protein expression |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| IL314915B1 (en) | 2017-01-23 | 2026-02-01 | Regeneron Pharma | Variants of 17-beta-hydroxysteroid dehydrogenase and their uses |
| 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 |
| SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
| US10961583B2 (en) | 2017-10-11 | 2021-03-30 | Regeneron Phramaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| 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 |
| JP7507093B2 (ja) | 2018-03-21 | 2024-06-27 | 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 | １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法 |
| CA3097004A1 (en) * | 2018-04-17 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Trans-splicing molecules |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| CA3103429A1 (en) * | 2018-06-14 | 2019-12-19 | Don W. Cleveland | Compounds and methods for increasing stmn2 expression |
| SG11202012759XA (en) | 2018-07-03 | 2021-01-28 | Hoffmann La Roche | Oligonucleotides for modulating tau expression |
| WO2020154686A1 (en) * | 2019-01-25 | 2020-07-30 | Nayan Therapeutics, Inc. | Nr2e3 expression reducing oligonucleotides, compositions containing the same, and methods of their use |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| WO2021023863A1 (en) * | 2019-08-08 | 2021-02-11 | Stichting Katholieke Universiteit | Antisense oligonucleotides rescue aberrant splicing of abca4 |
| EP4017979A4 (en) * | 2019-08-19 | 2024-03-27 | Stoke Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION |
| WO2021222654A1 (en) * | 2020-04-29 | 2021-11-04 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of inherited macular degeneration |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| TW202229553A (zh) | 2020-12-18 | 2022-08-01 | 瑞士商赫孚孟拉羅股份公司 | 靶向顆粒蛋白前體之反義寡核苷酸 |
| EP4444882A1 (en) * | 2021-12-07 | 2024-10-16 | F. Hoffmann-La Roche AG | Antisense oligonucleotides targeting actl6b |
| JP2025506826A (ja) * | 2022-02-25 | 2025-03-13 | 北京中因科技有限公司 | 発現カセット組み合わせ及びその使用 |
| AU2023269865A1 (en) | 2022-05-13 | 2024-11-21 | Ascidian Therapeutics, Inc. | Abca4 trans-splicing molecules |
| CN120225227A (zh) * | 2022-11-17 | 2025-06-27 | 斯基普治疗有限公司 | 用于治疗视网膜疾病的组合物和方法 |
| WO2024223696A1 (en) | 2023-04-24 | 2024-10-31 | Universiteit Gent | Compounds to treat inherited retinal disease |
| WO2025171404A1 (en) * | 2024-02-09 | 2025-08-14 | Utr Therapeutics Inc. | Destabilized are 3' utrs of tead1 and yap1 as pan cancer therapeutics |
| WO2025238644A1 (en) * | 2024-05-16 | 2025-11-20 | Skip Therapeutics Ltd. | Compositions and methods for treating retinal diseases |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9222088B2 (en) * | 2010-10-22 | 2015-12-29 | Curna, Inc. | Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA |
| ES2817050T3 (es) * | 2013-02-04 | 2021-04-06 | Ionis Pharmaceuticals Inc | Compuestos antisentido selectivos y usos de los mismos |
| PL3041958T4 (pl) * | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
| 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 |
-
2016
- 2016-12-14 EP EP16876610.3A patent/EP3390634A4/en not_active Withdrawn
- 2016-12-14 JP JP2018529249A patent/JP7036723B2/ja not_active Expired - Fee Related
- 2016-12-14 WO PCT/US2016/066691 patent/WO2017106370A1/en not_active Ceased
- 2016-12-14 CA CA3005128A patent/CA3005128A1/en active Pending
-
2022
- 2022-01-04 JP JP2022000109A patent/JP2022046724A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3390634A1 (en) | 2018-10-24 |
| JP7036723B2 (ja) | 2022-03-15 |
| JP2022046724A (ja) | 2022-03-23 |
| EP3390634A4 (en) | 2019-08-14 |
| WO2017106370A1 (en) | 2017-06-22 |
| JP2019500348A (ja) | 2019-01-10 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA3005128A1 (en) | Compositions and methods for treatment of eye diseases | |
| US11096956B2 (en) | Antisense oligomers and uses thereof | |
| JP7049249B2 (ja) | 中枢神経系疾患の処置のための組成物および方法 | |
| JP6395058B2 (ja) | ハンチンチン対する、組成物及びその使用 | |
| EP3390642B1 (en) | Compositions for treatment of retinitis pigmentosa 13 | |
| EP2900821B1 (en) | Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy | |
| JP2019500345A (ja) | 肝臓病の処置のための組成物および方法 | |
| JP2014519521A (ja) | 筋ジストロフィーの新たな治療法 | |
| CN119731328A (zh) | Abca4反式剪接分子 | |
| Chen et al. | Can gene therapy transform the treatment landscape of posterior segment eye diseases? A comprehensive review of recent advancements | |
| CN117980479A (zh) | 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体 | |
| Magaña et al. | Myotonic dystrophy type 1 (DM1): from the genetics to molecular mechanisms | |
| WO2025184574A1 (en) | Treatment of eye diseases and diagnostics | |
| JP2022530403A (ja) | エクソンスキッピング戦略による網膜ジストロフィーの処置方法 | |
| CN117866988A (zh) | 核酸片段及其作为标志物在制备回旋状视网膜脉络膜萎缩的诊断试剂中的应用 | |
| HK1262959B (en) | Compositions and methods for treatment of central nervous system diseases | |
| HK1262959A1 (en) | Compositions and methods for treatment of central nervous system diseases | |
| HK1262960B (en) | Compositions for treatment of retinitis pigmentosa 13 | |
| Venturini | Molecular genetics of ocular diseases |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20211213 |