DK3448999T3 - Oligonukleotider til behandling af øjensygdom - Google Patents
Oligonukleotider til behandling af øjensygdom Download PDFInfo
- Publication number
- DK3448999T3 DK3448999T3 DK17721984.7T DK17721984T DK3448999T3 DK 3448999 T3 DK3448999 T3 DK 3448999T3 DK 17721984 T DK17721984 T DK 17721984T DK 3448999 T3 DK3448999 T3 DK 3448999T3
- Authority
- DK
- Denmark
- Prior art keywords
- oligonucleotides
- treatment
- eye disease
- eye
- 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
Applications Claiming Priority (2)
| 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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3448999T3 true DK3448999T3 (da) | 2020-06-08 |
Family
ID=58672573
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK17721984.7T DK3448999T3 (da) | 2016-04-25 | 2017-04-25 | Oligonukleotider til behandling af øjensygdom |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US10617707B2 (da) |
| EP (1) | EP3448999B1 (da) |
| JP (1) | JP7043082B2 (da) |
| KR (1) | KR102368920B1 (da) |
| CN (1) | CN109072239A (da) |
| AU (1) | AU2017257292A1 (da) |
| CA (1) | CA3021899A1 (da) |
| DK (1) | DK3448999T3 (da) |
| EA (1) | EA201892431A1 (da) |
| ES (1) | ES2801823T3 (da) |
| IL (1) | IL262199B (da) |
| MX (1) | MX2018013003A (da) |
| PL (1) | PL3448999T3 (da) |
| WO (1) | WO2017186739A1 (da) |
| ZA (1) | ZA201806628B (da) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109072239A (zh) | 2016-04-25 | 2018-12-21 | ProQR治疗上市公司Ⅱ | 治疗眼病的寡核苷酸 |
| WO2019123430A1 (en) | 2017-12-21 | 2019-06-27 | Casebia Therapeutics Llp | Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp) |
| 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 |
| AU2020215232A1 (en) | 2019-01-28 | 2021-08-26 | Proqr Therapeutics Ii B.V. | RNA-editing oligonucleotides for the treatment of usher syndrome |
| MX2021009750A (es) * | 2019-02-12 | 2021-09-08 | Univ Degli Studi Di Trento | Moleculas del acido ribonucleico guia de cas12a y usos de las mismas. |
| WO2020201144A1 (en) | 2019-04-02 | 2020-10-08 | Proqr Therapeutics Ii B.V. | 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 |
| US20220243194A1 (en) | 2019-07-12 | 2022-08-04 | Peking University | Targeted rna editing by leveraging endogenous adar using engineered rnas |
| EP4003291A1 (en) | 2019-07-26 | 2022-06-01 | ProQR Therapeutics II B.V. | Ophthalmic compositions comprising viscosifying polymers and nucleic acids |
| CA3158528A1 (en) | 2019-12-23 | 2021-07-01 | Jim SWILDENS | Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease |
| 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 |
| CR20220366A (es) * | 2019-12-30 | 2022-11-28 | Edigene Therapeutics Beijing Inc | Método para el tratamiento del síndrome de usher y composición del mismo |
| WO2021175904A1 (en) | 2020-03-04 | 2021-09-10 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for use in the treatment of usher syndrome |
| EP4301856A2 (en) | 2021-03-05 | 2024-01-10 | 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 |
| WO2024074668A1 (en) * | 2022-10-06 | 2024-04-11 | Stichting Radboud Universitair Medisch Centrum | Antisense oligonucleotides for treatment of usher 2a. exons 30-31 |
| WO2024074670A1 (en) * | 2022-10-06 | 2024-04-11 | Stichting Radboud Universitair Medisch Centrum | Antisense oligonucleotides for treatment of usher 2a. exon 68 |
| 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 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001025188A1 (en) | 1999-10-04 | 2001-04-12 | University Of Medicine And Dentistry Of New Jersey | Novel carbamates and ureas |
| 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 |
| PL2425814T3 (pl) | 2010-09-03 | 2013-11-29 | Santen Sas | Emulsja typu woda-w-oleju do leczenia choroby oka |
| CA2952289A1 (en) * | 2014-07-10 | 2016-01-14 | 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 |
| CN108291225B (zh) | 2015-10-05 | 2022-07-12 | ProQR治疗上市公司Ⅱ | 单链反义寡核苷酸在预防或治疗涉及三核苷酸重复扩增的遗传疾病中的用途 |
| CN109072239A (zh) | 2016-04-25 | 2018-12-21 | ProQR治疗上市公司Ⅱ | 治疗眼病的寡核苷酸 |
-
2017
- 2017-04-25 CN CN201780025611.3A patent/CN109072239A/zh active Pending
- 2017-04-25 US US16/096,038 patent/US10617707B2/en active Active
- 2017-04-25 DK DK17721984.7T patent/DK3448999T3/da active
- 2017-04-25 EA EA201892431A patent/EA201892431A1/ru unknown
- 2017-04-25 MX MX2018013003A patent/MX2018013003A/es unknown
- 2017-04-25 WO PCT/EP2017/059830 patent/WO2017186739A1/en active Application Filing
- 2017-04-25 EP EP17721984.7A patent/EP3448999B1/en active Active
- 2017-04-25 ES ES17721984T patent/ES2801823T3/es active Active
- 2017-04-25 JP JP2019506546A patent/JP7043082B2/ja active Active
- 2017-04-25 AU AU2017257292A patent/AU2017257292A1/en not_active Abandoned
- 2017-04-25 PL PL17721984T patent/PL3448999T3/pl unknown
- 2017-04-25 CA CA3021899A patent/CA3021899A1/en active Pending
- 2017-04-25 KR KR1020187031243A patent/KR102368920B1/ko active IP Right Grant
-
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 |
|---|---|
| EA201892431A1 (ru) | 2019-04-30 |
| KR20180134931A (ko) | 2018-12-19 |
| JP7043082B2 (ja) | 2022-03-29 |
| CA3021899A1 (en) | 2017-11-02 |
| JP2019515688A (ja) | 2019-06-13 |
| CN109072239A (zh) | 2018-12-21 |
| IL262199B (en) | 2022-03-01 |
| KR102368920B1 (ko) | 2022-02-28 |
| ZA201806628B (en) | 2019-07-31 |
| US10617707B2 (en) | 2020-04-14 |
| PL3448999T3 (pl) | 2020-11-30 |
| ES2801823T3 (es) | 2021-01-14 |
| IL262199A (en) | 2018-12-31 |
| EP3448999B1 (en) | 2020-05-13 |
| WO2017186739A1 (en) | 2017-11-02 |
| MX2018013003A (es) | 2019-01-28 |
| US20190381089A1 (en) | 2019-12-19 |
| US11123360B2 (en) | 2021-09-21 |
| US20200237802A1 (en) | 2020-07-30 |
| US20210401871A1 (en) | 2021-12-30 |
| EP3448999A1 (en) | 2019-03-06 |
| AU2017257292A1 (en) | 2018-12-06 |
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