MX2020004207A - Terapias génicas para enfermedades neurodegenerativas. - Google Patents
Terapias génicas para enfermedades neurodegenerativas.Info
- Publication number
- MX2020004207A MX2020004207A MX2020004207A MX2020004207A MX2020004207A MX 2020004207 A MX2020004207 A MX 2020004207A MX 2020004207 A MX2020004207 A MX 2020004207A MX 2020004207 A MX2020004207 A MX 2020004207A MX 2020004207 A MX2020004207 A MX 2020004207A
- Authority
- MX
- Mexico
- Prior art keywords
- disease
- ftd
- als
- expression constructs
- disclosure provides
- Prior art date
Links
- 230000004770 neurodegeneration Effects 0.000 title abstract 2
- 208000015122 neurodegenerative disease Diseases 0.000 title abstract 2
- 238000001415 gene therapy Methods 0.000 title 1
- 201000011240 Frontotemporal dementia Diseases 0.000 abstract 3
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 abstract 3
- 102000043334 C9orf72 Human genes 0.000 abstract 2
- 108700030955 C9orf72 Proteins 0.000 abstract 2
- 208000024827 Alzheimer disease Diseases 0.000 abstract 1
- 101150014718 C9orf72 gene Proteins 0.000 abstract 1
- 206010067889 Dementia with Lewy bodies Diseases 0.000 abstract 1
- 208000015872 Gaucher disease Diseases 0.000 abstract 1
- 101000834926 Homo sapiens Transmembrane protein 106B Proteins 0.000 abstract 1
- 201000002832 Lewy body dementia Diseases 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 208000018737 Parkinson disease Diseases 0.000 abstract 1
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 102100026232 Transmembrane protein 106B Human genes 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 230000008685 targeting Effects 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
- C12N15/11—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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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- 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
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- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- 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
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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
- C12N15/1138—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 receptors or cell surface proteins
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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
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- 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]
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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
- 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
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14041—Use of virus, viral particle or viral elements as a vector
- C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
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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
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
- 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
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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
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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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
- 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
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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Neurology (AREA)
- Medicinal Chemistry (AREA)
- Neurosurgery (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychology (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Hospice & Palliative Care (AREA)
- Immunology (AREA)
- Psychiatry (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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762575795P | 2017-10-23 | 2017-10-23 | |
| US201862742723P | 2018-10-08 | 2018-10-08 | |
| PCT/US2018/057187 WO2019084068A1 (en) | 2017-10-23 | 2018-10-23 | GENE THERAPIES FOR NEURODEGENERATIVE DISEASE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020004207A true MX2020004207A (es) | 2020-11-11 |
Family
ID=66247008
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020004207A MX2020004207A (es) | 2017-10-23 | 2018-10-23 | Terapias génicas para enfermedades neurodegenerativas. |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210261981A1 (enExample) |
| EP (1) | EP3701030A4 (enExample) |
| JP (2) | JP7413256B2 (enExample) |
| KR (1) | KR20200075865A (enExample) |
| CN (2) | CN111819281B (enExample) |
| AU (1) | AU2018354195A1 (enExample) |
| BR (1) | BR112020008033A2 (enExample) |
| CA (2) | CA3083582A1 (enExample) |
| IL (2) | IL274129A (enExample) |
| MX (1) | MX2020004207A (enExample) |
| WO (1) | WO2019084068A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| KR20220010062A (ko) | 2017-10-03 | 2022-01-25 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| CA3092961A1 (en) * | 2018-03-09 | 2019-09-12 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| FI3953377T3 (fi) | 2019-04-10 | 2025-11-26 | Prevail Therapeutics Inc | Geeniterapioita lysosomaalisiin sairauksiin |
| EP3953378A1 (en) | 2019-04-10 | 2022-02-16 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP2023501897A (ja) * | 2019-10-22 | 2023-01-20 | アプライド ジェネティック テクノロジーズ コーポレイション | Ｃ９ｏｒｆ７２関連疾患の治療のための三重機能アデノ随伴ウイルス（ａａｖ）ベクター |
| WO2022026531A1 (en) * | 2020-07-29 | 2022-02-03 | Alnylam Pharmaceuticals, Inc. | Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases |
| JP2023545830A (ja) | 2020-10-15 | 2023-10-31 | プリベイル セラピューティクス，インコーポレーテッド | 組換えアデノ随伴ウイルス組成物及びその生産方法 |
| IL301858A (en) | 2020-10-15 | 2023-06-01 | Prevail Therapeutics Inc | Assay for measuring potency of gene therapy drug product |
| WO2022097727A1 (ja) | 2020-11-06 | 2022-05-12 | 住友ファーマ株式会社 | Ｒｐｓ２５遺伝子の発現及び／又は機能調節剤 |
| WO2022164259A1 (ko) | 2021-01-28 | 2022-08-04 | 주식회사 에이브레인 | 신경 퇴행성 질환의 치료를 위한 유전자 요법 |
| JP2024508635A (ja) | 2021-01-28 | 2024-02-28 | エイブレイン | 神経変性疾患を治療するための遺伝子療法 |
| KR102707250B1 (ko) | 2021-10-28 | 2024-09-13 | 한국해양과학기술원 | 신규 화합물 및 이를 유효성분으로 포함하는 신경염증성 뇌질환 예방 또는 치료용 조성물 |
| AR128239A1 (es) * | 2022-01-10 | 2024-04-10 | Univ Pennsylvania | Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72 |
| WO2024257847A1 (ja) | 2023-06-16 | 2024-12-19 | 住友ファーマ株式会社 | Ｒｐｓ２５遺伝子の発現及び／又はその機能を調節するアンチセンスオリゴヌクレオチド |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US20040053876A1 (en) * | 2002-03-26 | 2004-03-18 | The Regents Of The University Of Michigan | siRNAs and uses therof |
| US7829694B2 (en) * | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2004069173A2 (en) | 2003-01-31 | 2004-08-19 | The Trustees Of The University Of Pennsylvania | Methods for modulating an inflammatory response |
| CA2596588C (en) * | 2005-01-31 | 2017-06-27 | University Of Iowa Research Foundation | Nucleic acid silencing of huntington's disease gene |
| US20110269826A1 (en) | 2008-11-11 | 2011-11-03 | Oxford Biomedica (Uk) Limited | Method |
| US20110142789A1 (en) * | 2009-12-10 | 2011-06-16 | The Trustees Of The University Of Pennsylvania | Compositions and Methods for the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis |
| BR112013021494B1 (pt) * | 2011-02-22 | 2021-09-08 | California Institute Of Technology | Vetor viral, polinucleotídeo isolado, uso de um vírus adeno-associado (aav) recombinante e método para produzir o mesmo |
| HUE033431T2 (en) | 2012-04-23 | 2017-11-28 | Biomarin Tech Bv | RNA modulating oligonucleotides with improved characteristics in the treatment of neuromuscular disorders |
| US20150284472A1 (en) | 2012-11-05 | 2015-10-08 | Genzyme Corporation | Compositions and methods for treating proteinopathies |
| US20140271550A1 (en) * | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| CN112080502A (zh) * | 2013-10-11 | 2020-12-15 | Ionis制药公司 | 用于调节c9orf72表达的组合物 |
| EP3750907A3 (en) | 2014-03-18 | 2021-04-28 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| HRP20211397T1 (hr) * | 2014-03-19 | 2021-12-10 | Ionis Pharmaceuticals, Inc. | Pripravci za modulaciju ekspresije ataksina 2 |
| WO2015153760A2 (en) * | 2014-04-01 | 2015-10-08 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a nervous system disorder |
| WO2016024205A1 (en) * | 2014-08-15 | 2016-02-18 | Pfizer Inc. | Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene |
| WO2016077687A1 (en) * | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CA3019315A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| US10967073B2 (en) * | 2015-05-07 | 2021-04-06 | The Mclean Hospital Corporation | Glucocerebrosidase gene therapy for Parkinson's disease |
| CA3016314A1 (en) * | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
| CA3038548A1 (en) * | 2016-09-30 | 2018-04-05 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
| CA3112007A1 (en) * | 2018-09-12 | 2020-03-19 | Uniqure Ip B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
-
2018
- 2018-10-23 US US16/758,077 patent/US20210261981A1/en not_active Abandoned
- 2018-10-23 CA CA3083582A patent/CA3083582A1/en active Pending
- 2018-10-23 CA CA3177979A patent/CA3177979A1/en active Pending
- 2018-10-23 CN CN201880081355.4A patent/CN111819281B/zh active Active
- 2018-10-23 MX MX2020004207A patent/MX2020004207A/es unknown
- 2018-10-23 CN CN202110196181.XA patent/CN113005123A/zh active Pending
- 2018-10-23 BR BR112020008033-0A patent/BR112020008033A2/pt not_active Application Discontinuation
- 2018-10-23 AU AU2018354195A patent/AU2018354195A1/en not_active Abandoned
- 2018-10-23 KR KR1020207014683A patent/KR20200075865A/ko active Pending
- 2018-10-23 JP JP2020523011A patent/JP7413256B2/ja active Active
- 2018-10-23 EP EP18870021.5A patent/EP3701030A4/en not_active Withdrawn
- 2018-10-23 WO PCT/US2018/057187 patent/WO2019084068A1/en not_active Ceased
-
2020
- 2020-04-22 IL IL274129A patent/IL274129A/en unknown
-
2021
- 2021-02-23 IL IL281057A patent/IL281057A/en unknown
-
2023
- 2023-12-27 JP JP2023220260A patent/JP2024045131A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN111819281B (zh) | 2024-12-31 |
| WO2019084068A1 (en) | 2019-05-02 |
| IL274129A (en) | 2020-06-30 |
| BR112020008033A2 (pt) | 2020-10-27 |
| EP3701030A4 (en) | 2022-04-20 |
| JP7413256B2 (ja) | 2024-01-15 |
| US20210261981A1 (en) | 2021-08-26 |
| JP2024045131A (ja) | 2024-04-02 |
| CN113005123A (zh) | 2021-06-22 |
| CN111819281A (zh) | 2020-10-23 |
| KR20200075865A (ko) | 2020-06-26 |
| JP2021500049A (ja) | 2021-01-07 |
| EP3701030A1 (en) | 2020-09-02 |
| IL281057A (en) | 2021-04-29 |
| CA3083582A1 (en) | 2019-05-02 |
| CA3177979A1 (en) | 2019-05-02 |
| AU2018354195A1 (en) | 2020-06-04 |
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