PL2718437T3 - Sposoby leczenia wrodzonej ślepoty Lebera - Google Patents
Sposoby leczenia wrodzonej ślepoty LeberaInfo
- Publication number
- PL2718437T3 PL2718437T3 PL12729421T PL12729421T PL2718437T3 PL 2718437 T3 PL2718437 T3 PL 2718437T3 PL 12729421 T PL12729421 T PL 12729421T PL 12729421 T PL12729421 T PL 12729421T PL 2718437 T3 PL2718437 T3 PL 2718437T3
- Authority
- PL
- Poland
- Prior art keywords
- treatment
- methods
- congenital amaurosis
- leber congenital
- leber
- Prior art date
Links
- 201000003533 Leber congenital amaurosis Diseases 0.000 title 1
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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- C12N15/113—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
- C12N15/1137—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 enzymes
-
- 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
- C12N2310/32—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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
-
- 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
- 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
- C12N2320/34—Allele or polymorphism specific uses
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP11305735 | 2011-06-10 | ||
| EP12729421.3A EP2718437B1 (en) | 2011-06-10 | 2012-06-08 | Methods for the treatment of leber congenital amaurosis |
| PCT/EP2012/060906 WO2012168435A1 (en) | 2011-06-10 | 2012-06-08 | Methods for the treatment of leber congenital amaurosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2718437T3 true PL2718437T3 (pl) | 2019-01-31 |
Family
ID=44720213
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL12729421T PL2718437T3 (pl) | 2011-06-10 | 2012-06-08 | Sposoby leczenia wrodzonej ślepoty Lebera |
Country Status (6)
| Country | Link |
|---|---|
| US (5) | US9012425B2 (pl) |
| EP (2) | EP3378938A1 (pl) |
| DK (1) | DK2718437T3 (pl) |
| ES (1) | ES2683161T3 (pl) |
| PL (1) | PL2718437T3 (pl) |
| WO (1) | WO2012168435A1 (pl) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012168435A1 (en) * | 2011-06-10 | 2012-12-13 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the treatment of leber congenital amaurosis |
| EP2753694B1 (en) | 2011-09-05 | 2017-08-23 | Stichting Katholieke Universiteit | Antisense oligonucleotides for the treatment of leber congenital amaurosis |
| EP3019610B2 (en) | 2013-07-08 | 2025-03-19 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof |
| CN106459995B (zh) | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| ES2745769T3 (es) | 2014-03-10 | 2020-03-03 | Editas Medicine Inc | Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10) |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| KR102524543B1 (ko) | 2014-06-10 | 2023-04-20 | 에라스무스 유니버시티 메디컬 센터 로테르담 | 폼페병의 치료에 유용한 안티센스 올리고뉴클레오티드 |
| EP3189142B1 (en) * | 2014-09-05 | 2020-07-15 | Stichting Katholieke Universiteit | Antisense oligonucleotides for the treatment of leber congenital amaurosis |
| GB201503408D0 (en) * | 2015-02-27 | 2015-04-15 | Proqr Therapeutics N V | Oligonucleotides |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| WO2017099579A1 (en) | 2015-12-07 | 2017-06-15 | Erasmus University Medical Center Rotterdam | Enzymatic replacement therapy and antisense therapy for pompe disease |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| EP3452598A4 (en) | 2016-05-06 | 2020-04-29 | Exicure, Inc. | LIPOSOMAL SPHERICAL NUCLEIC ACID (ANS) CONSTRUCTS HAVING ANTISENSE OLIGONUCLEOTIDES (ASO) FOR THE SPECIFIC INACTIVATION OF INTERLEUKIN 17 RECEPTOR RNA |
| US20180010117A1 (en) * | 2016-07-07 | 2018-01-11 | Sangamo Therapeutics, Inc. | Targeted treatment of leber congenital amourosis |
| BR112019001887A2 (pt) | 2016-08-02 | 2019-07-09 | Editas Medicine Inc | composições e métodos para o tratamento de doença associada a cep290 |
| NL2017295B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Antisense oligomeric compound for Pompe disease |
| NL2017294B1 (en) * | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| EP3594346A4 (en) * | 2017-03-10 | 2020-12-16 | National Center For Child Health And Development | ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE |
| WO2018165384A1 (en) * | 2017-03-10 | 2018-09-13 | Gentex Corporation | Imaging system and method for vehicle occupant indentification and monitoring |
| GB201706009D0 (en) | 2017-04-13 | 2017-05-31 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of stargardt disease |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| EP3744333A4 (en) | 2018-01-25 | 2021-11-03 | Osaka University | MEDICAL COMPOSITION FOR THE TREATMENT OF DISEASES ASSOCIATED WITH AN INCREASE IN THE LEVEL OF EXPRESSION OF PERIOSTIN OR A CHANGE IN THE SPLICE VARIANT THEREOF |
| KR102398295B1 (ko) * | 2018-03-09 | 2022-05-17 | 다이이찌 산쿄 가부시키가이샤 | 당원병 Ia형 치료약 |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| US12398393B2 (en) | 2019-01-28 | 2025-08-26 | Proqr Therapeutics Ii B.V. | RNA-editing oligonucleotides for the treatment of usher syndrome |
| JP2022518808A (ja) | 2019-01-28 | 2022-03-16 | 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 | レーバー先天黒内障の処置のためのアンチセンスオリゴヌクレオチド |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| CA3136172A1 (en) | 2019-04-18 | 2020-10-22 | 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 |
| CA3158528A1 (en) | 2019-12-23 | 2021-07-01 | Jim SWILDENS | Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease |
| CA3166720A1 (en) | 2020-03-04 | 2021-09-10 | Jim SWILDENS | Antisense oligonucleotides for use in the treatment of usher syndrome |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| WO2022090256A1 (en) | 2020-10-26 | 2022-05-05 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of stargardt disease |
| EP4301856A2 (en) | 2021-03-05 | 2024-01-10 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for use in the treatment of corneal dystrophies |
| EP4479535A1 (en) | 2022-02-14 | 2024-12-25 | ProQR Therapeutics II B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7517644B1 (en) | 1991-08-23 | 2009-04-14 | Larry J. Smith | Method and compositions for cellular reprogramming |
| US20040102398A1 (en) | 2002-11-23 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of B7H expression |
| JP2006500066A (ja) | 2002-09-25 | 2006-01-05 | ファルマシア・コーポレーション | ミクロソームのプロスタグランジンｅ２シンターゼ発現のアンチセンス調節 |
| US7838231B2 (en) * | 2006-04-07 | 2010-11-23 | The Regents Of The University Of Michigan | NPH6 nucleic acids and proteins |
| AU2009231171A1 (en) | 2008-04-02 | 2009-10-08 | Fondazione Telethon | Method of treating genetic disorders |
| WO2012168435A1 (en) * | 2011-06-10 | 2012-12-13 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the treatment of leber congenital amaurosis |
| EP2753694B1 (en) | 2011-09-05 | 2017-08-23 | Stichting Katholieke Universiteit | Antisense oligonucleotides for the treatment of leber congenital amaurosis |
-
2012
- 2012-06-08 WO PCT/EP2012/060906 patent/WO2012168435A1/en not_active Ceased
- 2012-06-08 US US14/125,063 patent/US9012425B2/en active Active
- 2012-06-08 ES ES12729421.3T patent/ES2683161T3/es active Active
- 2012-06-08 EP EP18168531.4A patent/EP3378938A1/en not_active Withdrawn
- 2012-06-08 PL PL12729421T patent/PL2718437T3/pl unknown
- 2012-06-08 EP EP12729421.3A patent/EP2718437B1/en active Active
- 2012-06-08 DK DK12729421.3T patent/DK2718437T3/en active
-
2014
- 2014-11-18 US US14/546,124 patent/US9487782B2/en active Active
-
2016
- 2016-09-16 US US15/267,175 patent/US9777272B2/en active Active
-
2017
- 2017-08-31 US US15/692,669 patent/US10597654B2/en active Active
-
2020
- 2020-03-03 US US16/807,919 patent/US11492621B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| US20170044533A1 (en) | 2017-02-16 |
| US20150111951A1 (en) | 2015-04-23 |
| US11492621B2 (en) | 2022-11-08 |
| US20200291402A1 (en) | 2020-09-17 |
| US10597654B2 (en) | 2020-03-24 |
| US20140179767A1 (en) | 2014-06-26 |
| US9777272B2 (en) | 2017-10-03 |
| EP2718437A1 (en) | 2014-04-16 |
| EP3378938A1 (en) | 2018-09-26 |
| US20180016579A1 (en) | 2018-01-18 |
| WO2012168435A1 (en) | 2012-12-13 |
| US9012425B2 (en) | 2015-04-21 |
| ES2683161T3 (es) | 2018-09-25 |
| EP2718437B1 (en) | 2018-05-09 |
| US9487782B2 (en) | 2016-11-08 |
| DK2718437T3 (en) | 2018-08-06 |
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