CL2020002955A1 - Composiciones y métodos para reducir la empalmopatía y para tratar trastornos de dominancia del arn. - Google Patents
Composiciones y métodos para reducir la empalmopatía y para tratar trastornos de dominancia del arn.Info
- Publication number
- CL2020002955A1 CL2020002955A1 CL2020002955A CL2020002955A CL2020002955A1 CL 2020002955 A1 CL2020002955 A1 CL 2020002955A1 CL 2020002955 A CL2020002955 A CL 2020002955A CL 2020002955 A CL2020002955 A CL 2020002955A CL 2020002955 A1 CL2020002955 A1 CL 2020002955A1
- Authority
- CL
- Chile
- Prior art keywords
- rna
- splicing
- compositions
- methods
- patient
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 229920002477 rna polymer Polymers 0.000 abstract 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 4
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 abstract 3
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 abstract 3
- 208000035475 disorder Diseases 0.000 abstract 3
- 230000002452 interceptive effect Effects 0.000 abstract 3
- 239000013603 viral vector Substances 0.000 abstract 2
- 206010068871 Myotonic dystrophy Diseases 0.000 abstract 1
- 102000015097 RNA Splicing Factors Human genes 0.000 abstract 1
- 108010039259 RNA Splicing Factors Proteins 0.000 abstract 1
- 108091027967 Small hairpin RNA Proteins 0.000 abstract 1
- 108020004459 Small interfering RNA Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 230000014759 maintenance of location Effects 0.000 abstract 1
- 108091070501 miRNA Proteins 0.000 abstract 1
- 239000002679 microRNA Substances 0.000 abstract 1
- 239000002924 silencing RNA Substances 0.000 abstract 1
- 239000004055 small Interfering RNA Substances 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
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
- C12N15/63—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
-
- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- 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
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
-
- 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 nucleic acids [NA]
-
- 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 nucleic acids [NA]
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862671769P | 2018-05-15 | 2018-05-15 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2020002955A1 true CL2020002955A1 (es) | 2021-04-23 |
Family
ID=68540673
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2020002955A CL2020002955A1 (es) | 2018-05-15 | 2020-11-13 | Composiciones y métodos para reducir la empalmopatía y para tratar trastornos de dominancia del arn. |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20210269825A1 (https=) |
| EP (1) | EP3793566A4 (https=) |
| JP (1) | JP2021522836A (https=) |
| KR (1) | KR20210010549A (https=) |
| CN (1) | CN112469421A (https=) |
| AU (1) | AU2019268346A1 (https=) |
| BR (1) | BR112020023298A2 (https=) |
| CA (1) | CA3098249A1 (https=) |
| CL (1) | CL2020002955A1 (https=) |
| CO (1) | CO2020015239A2 (https=) |
| MA (1) | MA51938B1 (https=) |
| MX (1) | MX2020012269A (https=) |
| PH (1) | PH12020551913A1 (https=) |
| SG (1) | SG11202011151VA (https=) |
| WO (1) | WO2019222354A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| WO2023034870A2 (en) | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2023196862A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| US20250352672A1 (en) * | 2022-05-13 | 2025-11-20 | University Of Washington | Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting |
| TW202409289A (zh) * | 2022-06-27 | 2024-03-01 | 美商安斯泰來基因治療股份有限公司 | 用於治療肌強直性營養不良之組合物及方法 |
| US20240384271A1 (en) * | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2024229164A2 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025038795A1 (en) * | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025122536A1 (en) * | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025259747A2 (en) * | 2024-06-12 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5977333A (en) * | 1992-02-06 | 1999-11-02 | Massachusetts Institute Of Technology | DNA sequence encoding the myotonic dystrophy gene and uses thereof |
| US20050042646A1 (en) * | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2595663A4 (en) * | 2010-07-19 | 2014-03-05 | Isis Pharmaceuticals Inc | MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION |
| HRP20190992T1 (hr) * | 2014-12-24 | 2019-09-20 | Uniqure Ip B.V. | Suprimiranje gena za huntingtin inducirano s rnai |
| WO2017079291A1 (en) * | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| CN115896106A (zh) * | 2016-03-01 | 2023-04-04 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
| CN109862785B (zh) * | 2016-09-30 | 2022-09-06 | 瑞泽恩制药公司 | C9orf72基因座中具有六核苷酸重复扩增的非人类动物 |
| CN112292895B (zh) * | 2018-08-10 | 2024-06-14 | 富士通株式会社 | 一种通信方法、装置和通信系统 |
-
2019
- 2019-05-15 BR BR112020023298-0A patent/BR112020023298A2/pt not_active Application Discontinuation
- 2019-05-15 WO PCT/US2019/032423 patent/WO2019222354A1/en not_active Ceased
- 2019-05-15 KR KR1020207036197A patent/KR20210010549A/ko not_active Abandoned
- 2019-05-15 US US17/054,474 patent/US20210269825A1/en not_active Abandoned
- 2019-05-15 MX MX2020012269A patent/MX2020012269A/es unknown
- 2019-05-15 MA MA51938A patent/MA51938B1/fr unknown
- 2019-05-15 JP JP2020563947A patent/JP2021522836A/ja active Pending
- 2019-05-15 AU AU2019268346A patent/AU2019268346A1/en not_active Abandoned
- 2019-05-15 CN CN201980047374.XA patent/CN112469421A/zh active Pending
- 2019-05-15 EP EP19803882.0A patent/EP3793566A4/en not_active Withdrawn
- 2019-05-15 SG SG11202011151VA patent/SG11202011151VA/en unknown
- 2019-05-15 CA CA3098249A patent/CA3098249A1/en active Pending
-
2020
- 2020-11-10 PH PH12020551913A patent/PH12020551913A1/en unknown
- 2020-11-13 CL CL2020002955A patent/CL2020002955A1/es unknown
- 2020-12-03 CO CONC2020/0015239A patent/CO2020015239A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| PH12020551913A1 (en) | 2021-06-14 |
| BR112020023298A2 (pt) | 2021-03-09 |
| EP3793566A4 (en) | 2022-03-16 |
| MA51938A1 (fr) | 2021-11-30 |
| CN112469421A (zh) | 2021-03-09 |
| MA51938B1 (fr) | 2022-10-31 |
| WO2019222354A1 (en) | 2019-11-21 |
| MX2020012269A (es) | 2021-04-28 |
| KR20210010549A (ko) | 2021-01-27 |
| US20210269825A1 (en) | 2021-09-02 |
| JP2021522836A (ja) | 2021-09-02 |
| SG11202011151VA (en) | 2020-12-30 |
| EP3793566A1 (en) | 2021-03-24 |
| AU2019268346A1 (en) | 2020-12-24 |
| CO2020015239A2 (es) | 2021-03-08 |
| CA3098249A1 (en) | 2019-11-21 |
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