PT4008789T - Vetor aav bicistrónico para a interferênia de arn na als - Google Patents
Vetor aav bicistrónico para a interferênia de arn na alsInfo
- Publication number
- PT4008789T PT4008789T PT211976063T PT21197606T PT4008789T PT 4008789 T PT4008789 T PT 4008789T PT 211976063 T PT211976063 T PT 211976063T PT 21197606 T PT21197606 T PT 21197606T PT 4008789 T PT4008789 T PT 4008789T
- Authority
- PT
- Portugal
- Prior art keywords
- als
- rna interference
- aav vector
- bicistronic
- bicistronic aav
- Prior art date
Links
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 title 1
- 230000009368 gene silencing by RNA Effects 0.000 title 1
Classifications
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- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
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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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (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)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP15177828.9A EP3121284A1 (en) | 2015-07-22 | 2015-07-22 | Bicistronic aav vector for rna interference in als |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT4008789T true PT4008789T (pt) | 2024-07-24 |
Family
ID=53776350
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT167447044T PT3325632T (pt) | 2015-07-22 | 2016-07-22 | Vetor aav bicistrónico para a interferênia de arn na als |
| PT211976063T PT4008789T (pt) | 2015-07-22 | 2016-07-22 | Vetor aav bicistrónico para a interferênia de arn na als |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT167447044T PT3325632T (pt) | 2015-07-22 | 2016-07-22 | Vetor aav bicistrónico para a interferênia de arn na als |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US10835621B2 (pt) |
| EP (3) | EP3121284A1 (pt) |
| DK (2) | DK3325632T3 (pt) |
| ES (1) | ES2896181T3 (pt) |
| FI (1) | FI4008789T3 (pt) |
| HR (1) | HRP20240963T1 (pt) |
| HU (1) | HUE057809T2 (pt) |
| LT (2) | LT4008789T (pt) |
| PL (2) | PL4008789T3 (pt) |
| PT (2) | PT3325632T (pt) |
| RS (1) | RS65729B1 (pt) |
| SI (1) | SI3325632T1 (pt) |
| WO (1) | WO2017013252A1 (pt) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2019014760A (es) | 2017-06-06 | 2020-08-03 | Univ Massachusetts | Vectores de aav autoregulables para la expresión segura de mecp2 en el síndrome de rett. |
| EP4051324A4 (en) * | 2019-10-28 | 2023-11-29 | University Of Florida Research Foundation, Incorporated | GENE THERAPY VECTORS |
| WO2022060857A1 (en) * | 2020-09-16 | 2022-03-24 | Biogen Ma Inc. | Compositions and methods for treating amyotrophic lateral sclerosis (als) with aav-mir-sod1 |
| BR112023015761A2 (pt) | 2021-02-12 | 2023-11-07 | Alnylam Pharmaceuticals Inc | Superóxido dismutase 1 (sod1) composições de irna e métodos de uso das mesmas para tratar ou prevenir doenças neurodegenerativas associadas a superóxido dismutase 1 (sod1) |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT3039146T (lt) * | 2013-08-27 | 2020-07-27 | Research Institute at Nationwide Children`s Hospital | Produktai ir būdai, skirti amiotropinės lateralinės sklerozės gydymui |
-
2015
- 2015-07-22 EP EP15177828.9A patent/EP3121284A1/en not_active Withdrawn
-
2016
- 2016-07-22 LT LTEP21197606.3T patent/LT4008789T/lt unknown
- 2016-07-22 DK DK16744704.4T patent/DK3325632T3/da active
- 2016-07-22 PT PT167447044T patent/PT3325632T/pt unknown
- 2016-07-22 US US15/746,458 patent/US10835621B2/en active Active
- 2016-07-22 WO PCT/EP2016/067549 patent/WO2017013252A1/en active Application Filing
- 2016-07-22 RS RS20240788A patent/RS65729B1/sr unknown
- 2016-07-22 EP EP21197606.3A patent/EP4008789B1/en active Active
- 2016-07-22 SI SI201631383T patent/SI3325632T1/sl unknown
- 2016-07-22 PL PL21197606.3T patent/PL4008789T3/pl unknown
- 2016-07-22 EP EP16744704.4A patent/EP3325632B1/en active Active
- 2016-07-22 DK DK21197606.3T patent/DK4008789T3/da active
- 2016-07-22 FI FIEP21197606.3T patent/FI4008789T3/fi active
- 2016-07-22 PT PT211976063T patent/PT4008789T/pt unknown
- 2016-07-22 HU HUE16744704A patent/HUE057809T2/hu unknown
- 2016-07-22 HR HRP20240963TT patent/HRP20240963T1/hr unknown
- 2016-07-22 LT LTEPPCT/EP2016/067549T patent/LT3325632T/lt unknown
- 2016-07-22 PL PL16744704T patent/PL3325632T3/pl unknown
- 2016-07-22 ES ES16744704T patent/ES2896181T3/es active Active
-
2020
- 2020-10-26 US US17/080,205 patent/US11872290B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| PL3325632T3 (pl) | 2021-12-20 |
| US20180228919A1 (en) | 2018-08-16 |
| US10835621B2 (en) | 2020-11-17 |
| LT3325632T (lt) | 2021-12-10 |
| EP4008789A1 (en) | 2022-06-08 |
| HUE057809T2 (hu) | 2022-06-28 |
| US20210052744A1 (en) | 2021-02-25 |
| EP3325632A1 (en) | 2018-05-30 |
| EP4008789B1 (en) | 2024-07-03 |
| RS65729B1 (sr) | 2024-08-30 |
| PT3325632T (pt) | 2021-11-10 |
| LT4008789T (lt) | 2024-08-12 |
| US11872290B2 (en) | 2024-01-16 |
| DK4008789T3 (da) | 2024-07-29 |
| WO2017013252A1 (en) | 2017-01-26 |
| EP3121284A1 (en) | 2017-01-25 |
| ES2896181T3 (es) | 2022-02-24 |
| EP3325632B1 (en) | 2021-09-22 |
| SI3325632T1 (sl) | 2021-12-31 |
| DK3325632T3 (da) | 2021-11-08 |
| HRP20240963T1 (hr) | 2024-10-25 |
| FI4008789T3 (fi) | 2024-07-23 |
| PL4008789T3 (pl) | 2024-09-09 |
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