DK3044315T3 - Nukleinsyrer og fremgangsmåder til behandling af pompes sygdom - Google Patents
Nukleinsyrer og fremgangsmåder til behandling af pompes sygdom Download PDFInfo
- Publication number
- DK3044315T3 DK3044315T3 DK14761668.4T DK14761668T DK3044315T3 DK 3044315 T3 DK3044315 T3 DK 3044315T3 DK 14761668 T DK14761668 T DK 14761668T DK 3044315 T3 DK3044315 T3 DK 3044315T3
- Authority
- DK
- Denmark
- Prior art keywords
- pumps
- procedures
- disease
- treatment
- nuclear insurance
- Prior art date
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
- 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
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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
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- 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
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP13184013 | 2013-09-11 | ||
| PCT/EP2014/069325 WO2015036451A1 (en) | 2013-09-11 | 2014-09-10 | Nucleic acids and methods for the treatment of pompe disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3044315T3 true DK3044315T3 (da) | 2019-05-20 |
Family
ID=49162020
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK14761668.4T DK3044315T3 (da) | 2013-09-11 | 2014-09-10 | Nukleinsyrer og fremgangsmåder til behandling af pompes sygdom |
Country Status (5)
| Country | Link |
|---|---|
| US (3) | US10059947B2 (da) |
| EP (2) | EP3044315B1 (da) |
| DK (1) | DK3044315T3 (da) |
| ES (1) | ES2739850T3 (da) |
| WO (1) | WO2015036451A1 (da) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10059947B2 (en) * | 2013-09-11 | 2018-08-28 | Synthena Ag | Nucleic acids and methods for the treatment of Pompe disease |
| WO2015190922A1 (en) | 2014-06-10 | 2015-12-17 | Erasmus University Medical Center Rotterdam | Antisense oligonucleotides useful in treatment of pompe disease |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| US9976143B2 (en) | 2014-10-03 | 2018-05-22 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| AU2016233135B2 (en) | 2015-03-19 | 2021-07-08 | Translate Bio, Inc. | mRNA therapy for pompe disease |
| EP3359685A1 (en) | 2015-10-09 | 2018-08-15 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| WO2017099579A1 (en) * | 2015-12-07 | 2017-06-15 | Erasmus University Medical Center Rotterdam | Enzymatic replacement therapy and antisense therapy for pompe disease |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| 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 |
| NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| CN111278991B (zh) | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| CA3173647A1 (en) | 2020-05-11 | 2021-11-18 | Isabel AZNAREZ | Opa1 antisense oligomers for treatment of conditions and diseases |
| WO2023064833A1 (en) * | 2021-10-14 | 2023-04-20 | The University Of North Carolina At Chapel Hill | Correction of splicing mutations causing primary ciliary dyskinesia using oligonucleotides |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0215942B1 (en) | 1985-03-15 | 1995-07-12 | Antivirals Inc. | Polynucleotide assay reagent and method |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5646265A (en) * | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
| US6582908B2 (en) * | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
| US20040214255A1 (en) * | 1999-04-22 | 2004-10-28 | Myriad Genetics, Incorporated | Compositions and methods for treating diabetes |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| JP2010505741A (ja) | 2006-05-10 | 2010-02-25 | エイブイアイ バイオファーマ， インコーポレイテッド | カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| JP2012523225A (ja) | 2009-04-10 | 2012-10-04 | アソシアシオン・アンスティテュ・ドゥ・ミオロジー | 疾患の処置のためのトリシクロ−ｄｎａアンチセンスオリゴヌクレオチド、組成物及び方法 |
| ES2566553T3 (es) * | 2010-03-17 | 2016-04-13 | Association Institut De Myologie | ARNnp U7 modificados para el tratamiento de las enfermedades neuromusculares |
| DK2581448T3 (da) | 2011-10-13 | 2015-04-27 | Ass Inst De Myologie | Tricyclo-phosphorothioat-DNA |
| CN104203289B (zh) | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| US20150197534A1 (en) | 2013-09-05 | 2015-07-16 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| US10059947B2 (en) * | 2013-09-11 | 2018-08-28 | Synthena Ag | Nucleic acids and methods for the treatment of Pompe disease |
| WO2015190922A1 (en) | 2014-06-10 | 2015-12-17 | Erasmus University Medical Center Rotterdam | Antisense oligonucleotides useful in treatment of pompe disease |
| AU2015272127A1 (en) | 2014-06-10 | 2017-01-19 | Erasmus University Medical Center Rotterdam | Methods for characterizing alternatively or aberrantly spliced mRNA isoforms |
-
2014
- 2014-09-10 US US14/917,118 patent/US10059947B2/en active Active
- 2014-09-10 ES ES14761668T patent/ES2739850T3/es active Active
- 2014-09-10 EP EP14761668.4A patent/EP3044315B1/en active Active
- 2014-09-10 WO PCT/EP2014/069325 patent/WO2015036451A1/en active Application Filing
- 2014-09-10 EP EP18197095.5A patent/EP3480312A1/en active Pending
- 2014-09-10 DK DK14761668.4T patent/DK3044315T3/da active
-
2018
- 2018-07-23 US US16/042,714 patent/US11104903B2/en active Active
-
2021
- 2021-08-03 US US17/393,289 patent/US20220213485A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2015036451A1 (en) | 2015-03-19 |
| EP3044315B1 (en) | 2019-02-20 |
| US20220213485A1 (en) | 2022-07-07 |
| ES2739850T3 (es) | 2020-02-04 |
| US20190169618A1 (en) | 2019-06-06 |
| US20160215291A1 (en) | 2016-07-28 |
| US11104903B2 (en) | 2021-08-31 |
| US10059947B2 (en) | 2018-08-28 |
| EP3480312A1 (en) | 2019-05-08 |
| EP3044315A1 (en) | 2016-07-20 |
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