IL280581A - RNAi variant against alpha-synuclein - Google Patents
RNAi variant against alpha-synucleinInfo
- Publication number
- IL280581A IL280581A IL280581A IL28058121A IL280581A IL 280581 A IL280581 A IL 280581A IL 280581 A IL280581 A IL 280581A IL 28058121 A IL28058121 A IL 28058121A IL 280581 A IL280581 A IL 280581A
- Authority
- IL
- Israel
- Prior art keywords
- synuclein
- rnai against
- against alpha
- variant
- variant rnai
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title 1
- 102000003802 alpha-Synuclein Human genes 0.000 title 1
- 108090000185 alpha-Synuclein Proteins 0.000 title 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
- C12N15/11—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 (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 |
|---|---|
| IL280581A true IL280581A (en) | 2021-03-25 |
Family
ID=67587976
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL280581A IL280581A (en) | 2018-08-03 | 2021-02-02 | RNAi variant against alpha-synuclein |
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 |
| PH12021550254A1 (en) | 2018-08-03 | 2023-02-06 | Genzyme Corp | Variant rnai against alpha-synuclein |
| CN115777021A (zh) * | 2020-03-18 | 2023-03-10 | 马萨诸塞大学 | 用于snca调节的寡核苷酸 |
| WO2022072447A1 (en) * | 2020-10-01 | 2022-04-07 | Alnylam Pharmaceuticals, Inc. | Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases |
| EP4359525A1 (en) * | 2021-06-21 | 2024-05-01 | uniQure biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| AU2023248463A1 (en) * | 2022-04-06 | 2024-11-28 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| JP2025531512A (ja) * | 2022-09-29 | 2025-09-19 | アタランタ セラピューティクス，インコーポレーテッド | アルファシヌクレイン核酸を標的化するｓｉＲＮＡ組成物及び方法 |
| WO2024211480A2 (en) * | 2023-04-05 | 2024-10-10 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2025160429A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| TW202529779A (zh) * | 2024-01-30 | 2025-08-01 | 大陸商上海舶望製藥有限公司 | 用於抑制突觸核蛋白α（SNCA）基因表達的組合物和方法 |
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 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| 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 |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And 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 |
| PT2164967E (pt) | 2007-05-31 | 2015-10-27 | Univ Iowa Res Found | Redução da toxicidade não dirigida do arn de interferência |
| GB0816778D0 (en) | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| EP3514232A1 (en) | 2010-04-23 | 2019-07-24 | 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 |
| AU2011329777B2 (en) | 2010-11-17 | 2016-06-09 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| JP6224459B2 (ja) | 2011-02-17 | 2017-11-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法 |
| BR112014021104B1 (pt) | 2012-02-29 | 2023-03-28 | Sangamo Biosciences, Inc | Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington |
| EP4600255A3 (en) | 2014-05-02 | 2025-10-22 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| PH12021550254A1 (en) | 2018-08-03 | 2023-02-06 | Genzyme Corp | Variant rnai against alpha-synuclein |
-
2019
- 2019-08-02 PH PH1/2021/550254A patent/PH12021550254A1/en unknown
- 2019-08-02 TW TW113117876A patent/TW202509215A/zh unknown
- 2019-08-02 BR BR112021001980-4A patent/BR112021001980A2/pt unknown
- 2019-08-02 US US17/265,489 patent/US12365895B2/en active Active
- 2019-08-02 SG SG11202101072UA patent/SG11202101072UA/en unknown
- 2019-08-02 TW TW108127564A patent/TW202022116A/zh unknown
- 2019-08-02 MX MX2021001395A patent/MX2021001395A/es unknown
- 2019-08-02 AR ARP190102204A patent/AR114540A1/es unknown
- 2019-08-02 CN CN201980063972.6A patent/CN112805382A/zh active Pending
- 2019-08-02 KR KR1020217006255A patent/KR20210062627A/ko active Pending
- 2019-08-02 EP EP19752626.2A patent/EP3830265A1/en active Pending
- 2019-08-02 AU AU2019314529A patent/AU2019314529B2/en active Active
- 2019-08-02 WO PCT/US2019/044924 patent/WO2020028816A1/en not_active Ceased
- 2019-08-02 CA CA3108526A patent/CA3108526A1/en active Pending
- 2019-08-02 JP JP2021529252A patent/JP2021534814A/ja active Pending
-
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 |
| SG11202101072UA (en) | 2021-03-30 |
| MX2021001395A (es) | 2021-08-11 |
| CN112805382A (zh) | 2021-05-14 |
| US20210309999A1 (en) | 2021-10-07 |
| PH12021550254A1 (en) | 2023-02-06 |
| CO2021002637A2 (es) | 2021-07-30 |
| WO2020028816A1 (en) | 2020-02-06 |
| BR112021001980A2 (pt) | 2021-05-04 |
| JP2021534814A (ja) | 2021-12-16 |
| JP2024156715A (ja) | 2024-11-06 |
| US12365895B2 (en) | 2025-07-22 |
| AU2019314529A1 (en) | 2021-03-25 |
| AR114540A1 (es) | 2020-09-16 |
| TW202022116A (zh) | 2020-06-16 |
| KR20210062627A (ko) | 2021-05-31 |
| CA3108526A1 (en) | 2020-02-06 |
| TW202509215A (zh) | 2025-03-01 |
| EP3830265A1 (en) | 2021-06-09 |
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