AR114540A1 - VARIANTE DE ARNi CONTRA a-SINUCLEÍNA - Google Patents
VARIANTE DE ARNi CONTRA a-SINUCLEÍNAInfo
- Publication number
- AR114540A1 AR114540A1 ARP190102204A ARP190102204A AR114540A1 AR 114540 A1 AR114540 A1 AR 114540A1 AR P190102204 A ARP190102204 A AR P190102204A AR P190102204 A ARP190102204 A AR P190102204A AR 114540 A1 AR114540 A1 AR 114540A1
- Authority
- AR
- Argentina
- Prior art keywords
- rnai
- strand
- snca
- synuclein
- seq
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title abstract 9
- 230000009368 gene silencing by RNA Effects 0.000 title abstract 9
- 208000032859 Synucleinopathies Diseases 0.000 abstract 3
- 230000000626 neurodegenerative effect Effects 0.000 abstract 3
- 102100026882 Alpha-synuclein Human genes 0.000 abstract 2
- 102000003802 alpha-Synuclein Human genes 0.000 abstract 2
- 108090000185 alpha-Synuclein Proteins 0.000 abstract 2
- 229920001184 polypeptide Polymers 0.000 abstract 2
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 2
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 1
- 208000009829 Lewy Body Disease Diseases 0.000 abstract 1
- 201000002832 Lewy body dementia Diseases 0.000 abstract 1
- 208000001089 Multiple system atrophy Diseases 0.000 abstract 1
- 208000018737 Parkinson disease Diseases 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- 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
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (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 |
|---|---|---|---|
| US201862714616P | 2018-08-03 | 2018-08-03 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR114540A1 true AR114540A1 (es) | 2020-09-16 |
Family
ID=67587976
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190102204A AR114540A1 (es) | 2018-08-03 | 2019-08-02 | VARIANTE DE ARNi CONTRA a-SINUCLEÍNA |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12365895B2 (enExample) |
| EP (1) | EP3830265A1 (enExample) |
| JP (2) | JP2021534814A (enExample) |
| KR (1) | KR20210062627A (enExample) |
| CN (1) | CN112805382A (enExample) |
| AR (1) | AR114540A1 (enExample) |
| AU (1) | AU2019314529B2 (enExample) |
| BR (1) | BR112021001980A2 (enExample) |
| CA (1) | CA3108526A1 (enExample) |
| CO (1) | CO2021002637A2 (enExample) |
| IL (1) | IL280581A (enExample) |
| MX (1) | MX2021001395A (enExample) |
| PH (1) | PH12021550254A1 (enExample) |
| SG (1) | SG11202101072UA (enExample) |
| TW (2) | TW202509215A (enExample) |
| WO (1) | WO2020028816A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3684932A1 (en) * | 2017-09-22 | 2020-07-29 | Genzyme Corporation | Variant rnai |
| WO2020028816A1 (en) | 2018-08-03 | 2020-02-06 | Genzyme Corporation | Variant rnai against alpha-synuclein |
| CA3171757A1 (en) * | 2020-03-18 | 2021-09-23 | Anastasia Khvorova | Oligonucleotides for snca modulation |
| CA3193830A1 (en) * | 2020-10-01 | 2022-04-07 | Mangala Meenakshi Soundarapandian | Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases |
| US20250382611A1 (en) * | 2021-06-21 | 2025-12-18 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| CA3247417A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY |
| IL319688A (en) * | 2022-09-29 | 2025-05-01 | Atalanta Therapeutics Inc | siRNA compositions and methods targeting alpha-synuclein nucleic acids |
| US20240384271A1 (en) * | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2025160429A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| WO2025162214A1 (en) * | 2024-01-30 | 2025-08-07 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of synuclein alpha (snca) gene |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
| AU6972301A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Duplexed parvovirus vectors |
| US20050137155A1 (en) * | 2001-05-18 | 2005-06-23 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA) |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| CA2691617C (en) | 2007-05-31 | 2020-07-21 | University Of Iowa Research Foundation | Reduction of off-target rna interference toxicity |
| GB0816778D0 (en) * | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| US9102949B2 (en) | 2010-04-23 | 2015-08-11 | University Of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| WO2012027713A2 (en) * | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| EP2640853B1 (en) * | 2010-11-17 | 2018-12-26 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| AU2012219380B2 (en) | 2011-02-17 | 2017-04-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for altering tissue specificity and improving AAV9-mediated gene transfer |
| EP2820159B1 (en) | 2012-02-29 | 2019-10-23 | Sangamo Therapeutics, Inc. | Methods and compositions for treating huntington's disease |
| PE20170260A1 (es) | 2014-05-02 | 2017-04-12 | Genzyme Corp | Vectores de aav para la terapia genica de la retina y el snc |
| JP2018506304A (ja) | 2015-02-10 | 2018-03-08 | ジェンザイム・コーポレーション | バリアントＲＮＡｉ |
| WO2020028816A1 (en) | 2018-08-03 | 2020-02-06 | Genzyme Corporation | Variant rnai against alpha-synuclein |
-
2019
- 2019-08-02 WO PCT/US2019/044924 patent/WO2020028816A1/en not_active Ceased
- 2019-08-02 JP JP2021529252A patent/JP2021534814A/ja active Pending
- 2019-08-02 AU AU2019314529A patent/AU2019314529B2/en active Active
- 2019-08-02 BR BR112021001980-4A patent/BR112021001980A2/pt unknown
- 2019-08-02 CA CA3108526A patent/CA3108526A1/en active Pending
- 2019-08-02 TW TW113117876A patent/TW202509215A/zh unknown
- 2019-08-02 TW TW108127564A patent/TW202022116A/zh unknown
- 2019-08-02 MX MX2021001395A patent/MX2021001395A/es unknown
- 2019-08-02 PH PH1/2021/550254A patent/PH12021550254A1/en unknown
- 2019-08-02 AR ARP190102204A patent/AR114540A1/es unknown
- 2019-08-02 EP EP19752626.2A patent/EP3830265A1/en active Pending
- 2019-08-02 CN CN201980063972.6A patent/CN112805382A/zh active Pending
- 2019-08-02 KR KR1020217006255A patent/KR20210062627A/ko active Pending
- 2019-08-02 SG SG11202101072UA patent/SG11202101072UA/en unknown
- 2019-08-02 US US17/265,489 patent/US12365895B2/en active Active
-
2021
- 2021-02-02 IL IL280581A patent/IL280581A/en unknown
- 2021-02-26 CO CONC2021/0002637A patent/CO2021002637A2/es unknown
-
2024
- 2024-07-19 JP JP2024115452A patent/JP2024156715A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2021534814A (ja) | 2021-12-16 |
| US12365895B2 (en) | 2025-07-22 |
| SG11202101072UA (en) | 2021-03-30 |
| KR20210062627A (ko) | 2021-05-31 |
| US20210309999A1 (en) | 2021-10-07 |
| CO2021002637A2 (es) | 2021-07-30 |
| AU2019314529A1 (en) | 2021-03-25 |
| TW202022116A (zh) | 2020-06-16 |
| EP3830265A1 (en) | 2021-06-09 |
| AU2019314529B2 (en) | 2025-09-11 |
| TW202509215A (zh) | 2025-03-01 |
| WO2020028816A1 (en) | 2020-02-06 |
| CA3108526A1 (en) | 2020-02-06 |
| IL280581A (en) | 2021-03-25 |
| PH12021550254A1 (en) | 2023-02-06 |
| BR112021001980A2 (pt) | 2021-05-04 |
| MX2021001395A (es) | 2021-08-11 |
| CN112805382A (zh) | 2021-05-14 |
| JP2024156715A (ja) | 2024-11-06 |
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