MX2021001395A - Arni variante contra alfa-sinucleína. - Google Patents
Arni variante contra alfa-sinucleína.Info
- Publication number
- MX2021001395A MX2021001395A MX2021001395A MX2021001395A MX2021001395A MX 2021001395 A MX2021001395 A MX 2021001395A MX 2021001395 A MX2021001395 A MX 2021001395A MX 2021001395 A MX2021001395 A MX 2021001395A MX 2021001395 A MX2021001395 A MX 2021001395A
- Authority
- MX
- Mexico
- Prior art keywords
- synuclein
- rnai
- rnai against
- against alpha
- further provided
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title abstract 5
- 108090000185 alpha-Synuclein Proteins 0.000 title abstract 2
- 102000003802 alpha-Synuclein Human genes 0.000 title 1
- 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
- 206010067889 Dementia with Lewy bodies Diseases 0.000 abstract 1
- 101000834898 Homo sapiens Alpha-synuclein Proteins 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)
- 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 |
|---|---|
| MX2021001395A true MX2021001395A (es) | 2021-08-11 |
Family
ID=67587976
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021001395A MX2021001395A (es) | 2018-08-03 | 2019-08-02 | Arni variante contra alfa-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) | TW202022116A (enExample) |
| WO (1) | WO2020028816A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019060726A1 (en) * | 2017-09-22 | 2019-03-28 | 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 |
| JP2023544385A (ja) * | 2020-10-01 | 2023-10-23 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | Ｓｎｃａ関連神経変性疾患を治療又は予防するためのｓｎｃａ ｉｒｎａ組成物及びその使用方法 |
| US20250382611A1 (en) * | 2021-06-21 | 2025-12-18 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| CN119234040A (zh) * | 2022-04-06 | 2024-12-31 | 建新公司 | Dm-1肌强直性营养不良的靶向基因疗法 |
| IL319688A (en) * | 2022-09-29 | 2025-05-01 | Atalanta Therapeutics Inc | siRNA compositions and methods targeting alpha-synuclein nucleic acids |
| CN121002180A (zh) * | 2023-04-05 | 2025-11-21 | 建新公司 | Dm-1肌强直性营养不良的靶向基因疗法 |
| 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 |
|---|---|---|---|---|
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | 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 |
| JP4827353B2 (ja) | 1999-08-09 | 2011-11-30 | ターゲティッド ジェネティクス コーポレイション | 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大 |
| WO2001092551A2 (en) | 2000-06-01 | 2001-12-06 | University Of North Carolina At Chapel Hill | 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 |
| SG168422A1 (en) | 2001-11-13 | 2011-02-28 | 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 |
| ES2549122T3 (es) | 2007-05-31 | 2015-10-23 | University Of Iowa Research Foundation | Reducción de toxicidad por interferencia de ARN desviada de su diana |
| GB0816778D0 (en) * | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| EP3540055A1 (en) | 2010-04-23 | 2019-09-18 | 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 |
| EP3521451A1 (en) | 2010-11-17 | 2019-08-07 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| MX374399B (es) | 2012-02-29 | 2025-03-06 | Sangamo Biosciences Inc | Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| MA40819B1 (fr) | 2015-02-10 | 2020-04-30 | Genzyme Corp | Variant d'arni |
| 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 AR ARP190102204A patent/AR114540A1/es unknown
- 2019-08-02 TW TW108127564A patent/TW202022116A/zh unknown
- 2019-08-02 AU AU2019314529A patent/AU2019314529B2/en active Active
- 2019-08-02 PH PH1/2021/550254A patent/PH12021550254A1/en unknown
- 2019-08-02 CA CA3108526A patent/CA3108526A1/en active Pending
- 2019-08-02 JP JP2021529252A patent/JP2021534814A/ja active Pending
- 2019-08-02 CN CN201980063972.6A patent/CN112805382A/zh active Pending
- 2019-08-02 SG SG11202101072UA patent/SG11202101072UA/en unknown
- 2019-08-02 MX MX2021001395A patent/MX2021001395A/es unknown
- 2019-08-02 KR KR1020217006255A patent/KR20210062627A/ko active Pending
- 2019-08-02 TW TW113117876A patent/TW202509215A/zh unknown
- 2019-08-02 EP EP19752626.2A patent/EP3830265A1/en active Pending
- 2019-08-02 US US17/265,489 patent/US12365895B2/en active Active
- 2019-08-02 BR BR112021001980-4A patent/BR112021001980A2/pt 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 |
|---|---|
| WO2020028816A1 (en) | 2020-02-06 |
| TW202022116A (zh) | 2020-06-16 |
| AU2019314529A1 (en) | 2021-03-25 |
| PH12021550254A1 (en) | 2023-02-06 |
| KR20210062627A (ko) | 2021-05-31 |
| AR114540A1 (es) | 2020-09-16 |
| AU2019314529B2 (en) | 2025-09-11 |
| SG11202101072UA (en) | 2021-03-30 |
| EP3830265A1 (en) | 2021-06-09 |
| IL280581A (en) | 2021-03-25 |
| BR112021001980A2 (pt) | 2021-05-04 |
| TW202509215A (zh) | 2025-03-01 |
| CO2021002637A2 (es) | 2021-07-30 |
| CN112805382A (zh) | 2021-05-14 |
| US20210309999A1 (en) | 2021-10-07 |
| JP2024156715A (ja) | 2024-11-06 |
| JP2021534814A (ja) | 2021-12-16 |
| CA3108526A1 (en) | 2020-02-06 |
| US12365895B2 (en) | 2025-07-22 |
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