CO2021002637A2 - Arni variante frente a alfa-sinucleína - Google Patents
Arni variante frente a alfa-sinucleínaInfo
- Publication number
- CO2021002637A2 CO2021002637A2 CONC2021/0002637A CO2021002637A CO2021002637A2 CO 2021002637 A2 CO2021002637 A2 CO 2021002637A2 CO 2021002637 A CO2021002637 A CO 2021002637A CO 2021002637 A2 CO2021002637 A2 CO 2021002637A2
- Authority
- CO
- Colombia
- Prior art keywords
- synuclein
- rnai
- arni
- alpha
- variant versus
- Prior art date
Links
- 108090000185 alpha-Synuclein Proteins 0.000 title abstract 2
- 241000320892 Clerodendrum phlomidis Species 0.000 title 1
- 102000003802 alpha-Synuclein Human genes 0.000 title 1
- 108091030071 RNAI Proteins 0.000 abstract 4
- 230000009368 gene silencing by RNA Effects 0.000 abstract 4
- 102100026882 Alpha-synuclein Human genes 0.000 abstract 2
- 208000032859 Synucleinopathies Diseases 0.000 abstract 2
- 230000000626 neurodegenerative effect Effects 0.000 abstract 2
- 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
- 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)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Hospice & Palliative Care (AREA)
- Virology (AREA)
- Psychiatry (AREA)
- Epidemiology (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)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862714616P | 2018-08-03 | 2018-08-03 | |
| PCT/US2019/044924 WO2020028816A1 (en) | 2018-08-03 | 2019-08-02 | Variant rnai against alpha-synuclein |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2021002637A2 true CO2021002637A2 (es) | 2021-07-30 |
Family
ID=67587976
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2021/0002637A CO2021002637A2 (es) | 2018-08-03 | 2021-02-26 | Arni variante frente a alfa-sinucleína |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12365895B2 (https=) |
| EP (1) | EP3830265A1 (https=) |
| JP (2) | JP2021534814A (https=) |
| KR (1) | KR102931619B1 (https=) |
| CN (1) | CN112805382A (https=) |
| AR (1) | AR114540A1 (https=) |
| AU (1) | AU2019314529B2 (https=) |
| BR (1) | BR112021001980A2 (https=) |
| CA (1) | CA3108526A1 (https=) |
| CO (1) | CO2021002637A2 (https=) |
| IL (1) | IL280581A (https=) |
| MX (1) | MX2021001395A (https=) |
| PH (1) | PH12021550254A1 (https=) |
| SG (1) | SG11202101072UA (https=) |
| TW (2) | TW202509215A (https=) |
| WO (1) | WO2020028816A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111356763B (zh) * | 2017-09-22 | 2024-03-12 | 建新公司 | 变体RNAi |
| KR102931619B1 (ko) | 2018-08-03 | 2026-02-26 | 젠자임 코포레이션 | 알파-시뉴클레인에 대한 변이형 RNAi |
| US20210363524A1 (en) * | 2020-03-18 | 2021-11-25 | University Of Massachusetts | Oligonucleotides for snca modulation |
| EP4221838A4 (en) * | 2020-10-01 | 2025-04-16 | Alnylam Pharmaceuticals, Inc. | Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases |
| WO2022268835A1 (en) * | 2021-06-21 | 2022-12-29 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| WO2023196862A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| US20260103703A1 (en) * | 2022-09-29 | 2026-04-16 | 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 |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| DK2164967T3 (en) | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
| GB0816778D0 (en) | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | 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 |
| CA2817960C (en) | 2010-11-17 | 2020-06-09 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| WO2013130824A1 (en) | 2012-02-29 | 2013-09-06 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| CR20170406A (es) | 2015-02-10 | 2017-11-14 | Genzyme Corp | Arni variante |
| KR102931619B1 (ko) | 2018-08-03 | 2026-02-26 | 젠자임 코포레이션 | 알파-시뉴클레인에 대한 변이형 RNAi |
-
2019
- 2019-08-02 KR KR1020217006255A patent/KR102931619B1/ko active Active
- 2019-08-02 BR BR112021001980-4A patent/BR112021001980A2/pt unknown
- 2019-08-02 JP JP2021529252A patent/JP2021534814A/ja active Pending
- 2019-08-02 SG SG11202101072UA patent/SG11202101072UA/en unknown
- 2019-08-02 TW TW113117876A patent/TW202509215A/zh unknown
- 2019-08-02 MX MX2021001395A patent/MX2021001395A/es unknown
- 2019-08-02 WO PCT/US2019/044924 patent/WO2020028816A1/en not_active Ceased
- 2019-08-02 AU AU2019314529A patent/AU2019314529B2/en active Active
- 2019-08-02 US US17/265,489 patent/US12365895B2/en active Active
- 2019-08-02 CA CA3108526A patent/CA3108526A1/en active Pending
- 2019-08-02 TW TW108127564A patent/TW202022116A/zh 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 PH PH1/2021/550254A patent/PH12021550254A1/en unknown
- 2019-08-02 AR ARP190102204A patent/AR114540A1/es unknown
-
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 |
|---|---|
| AU2019314529B2 (en) | 2025-09-11 |
| WO2020028816A1 (en) | 2020-02-06 |
| EP3830265A1 (en) | 2021-06-09 |
| TW202022116A (zh) | 2020-06-16 |
| JP2021534814A (ja) | 2021-12-16 |
| KR102931619B1 (ko) | 2026-02-26 |
| PH12021550254A1 (en) | 2023-02-06 |
| IL280581A (en) | 2021-03-25 |
| JP2024156715A (ja) | 2024-11-06 |
| AU2019314529A1 (en) | 2021-03-25 |
| CN112805382A (zh) | 2021-05-14 |
| BR112021001980A2 (pt) | 2021-05-04 |
| AR114540A1 (es) | 2020-09-16 |
| US12365895B2 (en) | 2025-07-22 |
| CA3108526A1 (en) | 2020-02-06 |
| KR20210062627A (ko) | 2021-05-31 |
| TW202509215A (zh) | 2025-03-01 |
| US20210309999A1 (en) | 2021-10-07 |
| MX2021001395A (es) | 2021-08-11 |
| SG11202101072UA (en) | 2021-03-30 |
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