PL3448999T3 - Oligonukleotydy do leczenia choroby oka - Google Patents
Oligonukleotydy do leczenia choroby okaInfo
- Publication number
- PL3448999T3 PL3448999T3 PL17721984T PL17721984T PL3448999T3 PL 3448999 T3 PL3448999 T3 PL 3448999T3 PL 17721984 T PL17721984 T PL 17721984T PL 17721984 T PL17721984 T PL 17721984T PL 3448999 T3 PL3448999 T3 PL 3448999T3
- Authority
- PL
- Poland
- Prior art keywords
- oligonucleotides
- eye disease
- treat eye
- treat
- disease
- Prior art date
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- 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
-
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/333—Modified A
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Ophthalmology & Optometry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB201607141 | 2016-04-25 | ||
| PCT/EP2017/059830 WO2017186739A1 (en) | 2016-04-25 | 2017-04-25 | Oligonucleotides to treat eye disease |
| EP17721984.7A EP3448999B1 (en) | 2016-04-25 | 2017-04-25 | Oligonucleotides to treat eye disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3448999T3 true PL3448999T3 (pl) | 2020-11-30 |
Family
ID=58672573
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL17721984T PL3448999T3 (pl) | 2016-04-25 | 2017-04-25 | Oligonukleotydy do leczenia choroby oka |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US10617707B2 (pl) |
| EP (1) | EP3448999B1 (pl) |
| JP (1) | JP7043082B2 (pl) |
| KR (1) | KR102368920B1 (pl) |
| CN (1) | CN109072239A (pl) |
| AU (1) | AU2017257292A1 (pl) |
| CA (1) | CA3021899A1 (pl) |
| DK (1) | DK3448999T3 (pl) |
| EA (1) | EA201892431A1 (pl) |
| ES (1) | ES2801823T3 (pl) |
| IL (1) | IL262199B (pl) |
| MX (1) | MX2018013003A (pl) |
| PL (1) | PL3448999T3 (pl) |
| WO (1) | WO2017186739A1 (pl) |
| ZA (1) | ZA201806628B (pl) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2017257292A1 (en) | 2016-04-25 | 2018-12-06 | Proqr Therapeutics Ii B.V. | Oligonucleotides to treat eye disease |
| CN111836892A (zh) | 2017-12-21 | 2020-10-27 | 克里斯珀医疗股份公司 | 用于治疗2a型乌谢尔综合征的材料和方法 |
| CA3084633A1 (en) | 2017-12-21 | 2019-06-27 | Crispr Therapeutics Ag | Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp) |
| JP2022523302A (ja) | 2019-01-28 | 2022-04-22 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | アッシャー症候群の処置のためのｒｎａ編集オリゴヌクレオチド |
| EP3924494A1 (en) * | 2019-02-12 | 2021-12-22 | Università Degli Studi Di Trento | Cas12a guide rna molecules and uses thereof |
| CA3132178A1 (en) | 2019-04-02 | 2020-10-08 | Aliye Seda Yilmaz-Elis | Antisense oligonucleotides for immunotherapy |
| JP2022526455A (ja) | 2019-04-15 | 2022-05-24 | 北京大学 | Ｒｎａを編集する方法および組成物 |
| US20220213478A1 (en) | 2019-04-18 | 2022-07-07 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of usher syndrome |
| WO2020254249A1 (en) | 2019-06-21 | 2020-12-24 | Proqr Therapeutics Ii B.V. | Delivery of nucleic acids for the treatment of auditory disorders |
| CA3146771A1 (en) | 2019-07-12 | 2021-01-21 | Peking University | Targeted rna editing by leveraging endogenous adar using engineered rnas |
| AU2020321466A1 (en) | 2019-07-26 | 2022-02-17 | Proqr Therapeutics Ii B.V. | Ophthalmic compositions comprising viscosifying polymers and nucleic acids |
| US20230039928A1 (en) | 2019-12-23 | 2023-02-09 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for nucleotide deamination in the treatment of Stargardt disease |
| EP4086346A4 (en) * | 2019-12-30 | 2024-03-20 | Edigene Therapeutics Beijing Inc | METHOD FOR TREATMENT OF USHER SYNDROME AND ASSOCIATED COMPOSITION |
| CN113122577A (zh) * | 2019-12-30 | 2021-07-16 | 博雅辑因（北京）生物科技有限公司 | 一种治疗Usher综合征的方法和其组合物 |
| WO2021175904A1 (en) | 2020-03-04 | 2021-09-10 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for use in the treatment of usher syndrome |
| US20240141340A1 (en) | 2021-03-05 | 2024-05-02 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for use in the treatment of corneal dystrophies |
| TR2022001648A2 (tr) * | 2022-02-09 | 2022-02-21 | T C Ueskuedar Ueniversitesi | Ush2a kaynaklı retinitis pigmentosa hastalığının genetik tedavisi için ekzon 13 atlama işlevi gören kseno nükleik asit antisens-oligonükleotit (xna-aso) dizileri. |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2024074670A1 (en) * | 2022-10-06 | 2024-04-11 | Stichting Radboud Universitair Medisch Centrum | Antisense oligonucleotides for treatment of usher 2a. exon 68 |
| WO2024074668A1 (en) * | 2022-10-06 | 2024-04-11 | Stichting Radboud Universitair Medisch Centrum | Antisense oligonucleotides for treatment of usher 2a. exons 30-31 |
| WO2024078345A1 (zh) * | 2022-10-11 | 2024-04-18 | 广州瑞风生物科技有限公司 | snRNA核酸分子及其应用 |
| WO2024105063A1 (en) * | 2022-11-15 | 2024-05-23 | Stichting Radboud Universitair Medisch Centrum | Antisense oligonucleotides for treatment of usher 2a. exons 39-40 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2386239A1 (en) | 1999-10-04 | 2001-04-12 | University Of Medicine And Dentistry Of New Jersey | Methods for identifying rna binding compounds |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| US20100196889A1 (en) | 2006-11-13 | 2010-08-05 | Bankaitis-Davis Danute M | Gene Expression Profiling for Identification, Monitoring and Treatment of Colorectal Cancer |
| US8093000B2 (en) | 2008-05-09 | 2012-01-10 | The Regents Of The University Of California | Methods for predicting and treating tumors resistant to drug, immunotherapy, and radiation |
| US20110070582A1 (en) | 2008-11-03 | 2011-03-24 | Source Precision Medicine, Inc. d/b/d Source MDX | Gene Expression Profiling for Predicting the Response to Immunotherapy and/or the Survivability of Melanoma Subjects |
| SI2425814T1 (sl) | 2010-09-03 | 2013-10-30 | Novagali Pharma S.A. | Emulzija vrste voda-v-olju za zdravljenje bolezni očesa |
| US10131910B2 (en) * | 2014-07-10 | 2018-11-20 | Stichting Katholieke Universiteit | Antisense oligonucleotides for the treatment of Usher syndrome type 2 |
| US9927034B2 (en) * | 2015-08-25 | 2018-03-27 | Mueller International, Llc | Valve seat stiffener |
| EP3359667A1 (en) | 2015-10-05 | 2018-08-15 | ProQR Therapeutics II B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
| AU2017257292A1 (en) | 2016-04-25 | 2018-12-06 | Proqr Therapeutics Ii B.V. | Oligonucleotides to treat eye disease |
-
2017
- 2017-04-25 AU AU2017257292A patent/AU2017257292A1/en not_active Abandoned
- 2017-04-25 CN CN201780025611.3A patent/CN109072239A/zh active Pending
- 2017-04-25 PL PL17721984T patent/PL3448999T3/pl unknown
- 2017-04-25 US US16/096,038 patent/US10617707B2/en active Active
- 2017-04-25 JP JP2019506546A patent/JP7043082B2/ja active Active
- 2017-04-25 EP EP17721984.7A patent/EP3448999B1/en active Active
- 2017-04-25 DK DK17721984.7T patent/DK3448999T3/da active
- 2017-04-25 ES ES17721984T patent/ES2801823T3/es active Active
- 2017-04-25 KR KR1020187031243A patent/KR102368920B1/ko active IP Right Grant
- 2017-04-25 WO PCT/EP2017/059830 patent/WO2017186739A1/en active Application Filing
- 2017-04-25 CA CA3021899A patent/CA3021899A1/en active Pending
- 2017-04-25 EA EA201892431A patent/EA201892431A1/ru unknown
- 2017-04-25 MX MX2018013003A patent/MX2018013003A/es unknown
-
2018
- 2018-10-05 ZA ZA2018/06628A patent/ZA201806628B/en unknown
- 2018-10-08 IL IL262199A patent/IL262199B/en unknown
-
2020
- 2020-03-06 US US16/811,848 patent/US11123360B2/en active Active
-
2021
- 2021-09-15 US US17/475,795 patent/US20210401871A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| JP2019515688A (ja) | 2019-06-13 |
| EP3448999A1 (en) | 2019-03-06 |
| ZA201806628B (en) | 2019-07-31 |
| MX2018013003A (es) | 2019-01-28 |
| JP7043082B2 (ja) | 2022-03-29 |
| EA201892431A1 (ru) | 2019-04-30 |
| EP3448999B1 (en) | 2020-05-13 |
| US20190381089A1 (en) | 2019-12-19 |
| DK3448999T3 (da) | 2020-06-08 |
| US20200237802A1 (en) | 2020-07-30 |
| US10617707B2 (en) | 2020-04-14 |
| ES2801823T3 (es) | 2021-01-14 |
| CN109072239A (zh) | 2018-12-21 |
| KR20180134931A (ko) | 2018-12-19 |
| US11123360B2 (en) | 2021-09-21 |
| IL262199B (en) | 2022-03-01 |
| US20210401871A1 (en) | 2021-12-30 |
| KR102368920B1 (ko) | 2022-02-28 |
| CA3021899A1 (en) | 2017-11-02 |
| AU2017257292A1 (en) | 2018-12-06 |
| WO2017186739A1 (en) | 2017-11-02 |
| IL262199A (en) | 2018-12-31 |
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