CN111819281B - 用于神经变性疾病的基因疗法 - Google Patents
用于神经变性疾病的基因疗法 Download PDFInfo
- Publication number
- CN111819281B CN111819281B CN201880081355.4A CN201880081355A CN111819281B CN 111819281 B CN111819281 B CN 111819281B CN 201880081355 A CN201880081355 A CN 201880081355A CN 111819281 B CN111819281 B CN 111819281B
- Authority
- CN
- China
- Prior art keywords
- nucleic acid
- ser
- vector
- pro
- raav
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
-
- 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
- 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
-
- 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
-
- 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
-
- 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
- C12N15/11—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
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- 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
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- 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
-
- 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
-
- 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
- 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
-
- 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
-
- 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)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Immunology (AREA)
- Hospice & Palliative Care (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)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202110196181.XA CN113005123A (zh) | 2017-10-23 | 2018-10-23 | 用于神经变性疾病的基因疗法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762575795P | 2017-10-23 | 2017-10-23 | |
| US62/575,795 | 2017-10-23 | ||
| US201862742723P | 2018-10-08 | 2018-10-08 | |
| US62/742,723 | 2018-10-08 | ||
| PCT/US2018/057187 WO2019084068A1 (en) | 2017-10-23 | 2018-10-23 | GENE THERAPIES FOR NEURODEGENERATIVE DISEASE |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202110196181.XA Division CN113005123A (zh) | 2017-10-23 | 2018-10-23 | 用于神经变性疾病的基因疗法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN111819281A CN111819281A (zh) | 2020-10-23 |
| CN111819281B true CN111819281B (zh) | 2024-12-31 |
Family
ID=66247008
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201880081355.4A Active CN111819281B (zh) | 2017-10-23 | 2018-10-23 | 用于神经变性疾病的基因疗法 |
| CN202110196181.XA Pending CN113005123A (zh) | 2017-10-23 | 2018-10-23 | 用于神经变性疾病的基因疗法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202110196181.XA Pending CN113005123A (zh) | 2017-10-23 | 2018-10-23 | 用于神经变性疾病的基因疗法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210261981A1 (https=) |
| EP (1) | EP3701030A4 (https=) |
| JP (2) | JP7413256B2 (https=) |
| KR (1) | KR20200075865A (https=) |
| CN (2) | CN111819281B (https=) |
| AU (1) | AU2018354195A1 (https=) |
| BR (1) | BR112020008033A2 (https=) |
| CA (2) | CA3083582A1 (https=) |
| IL (2) | IL274129A (https=) |
| MX (1) | MX2020004207A (https=) |
| WO (1) | WO2019084068A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| JP2021517168A (ja) * | 2018-03-09 | 2021-07-15 | アブロバイオ,インコーポレーテッド | パーキンソン病を処置するための組成物及び方法 |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP7664848B2 (ja) | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス,インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| WO2021081236A1 (en) * | 2019-10-22 | 2021-04-29 | Applied Genetic Technologies Corporation | Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases |
| 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 |
| EP4229213A1 (en) | 2020-10-15 | 2023-08-23 | Prevail Therapeutics, Inc. | Assay for measuring potency of gene therapy drug product |
| MX2023004420A (es) | 2020-10-15 | 2023-07-05 | Prevail Therapeutics Inc | Composiciones de virus adenoasociados recombinantes y métodos para su producción. |
| CN116507727A (zh) | 2020-11-06 | 2023-07-28 | 住友制药株式会社 | Rps25基因的表达和/或功能调节剂 |
| WO2022164259A1 (ko) | 2021-01-28 | 2022-08-04 | 주식회사 에이브레인 | 신경 퇴행성 질환의 치료를 위한 유전자 요법 |
| CA3206769A1 (en) | 2021-01-28 | 2022-08-04 | Kyung Won Park | Gene therapy for treating neurodegenerative diseases |
| KR102707250B1 (ko) | 2021-10-28 | 2024-09-13 | 한국해양과학기술원 | 신규 화합물 및 이를 유효성분으로 포함하는 신경염증성 뇌질환 예방 또는 치료용 조성물 |
| WO2023133574A1 (en) * | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for treatment of c9orf72-mediated disorders |
| EP4511482A4 (en) * | 2022-04-19 | 2026-03-18 | Shanghai Vitalgen Biopharma Co Ltd | Recombinant AAV vectors for the treatment of neurodegenerative disorders |
| JPWO2024257847A1 (https=) | 2023-06-16 | 2024-12-19 |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105637090A (zh) * | 2013-10-11 | 2016-06-01 | Ionis制药公司 | 用于调节c9orf72表达的组合物 |
Family Cites Families (21)
| 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 |
| EP1844148A2 (en) * | 2005-01-31 | 2007-10-17 | University of Iowa Research Foundation | Nucleic acid silencing of huntington's disease gene |
| JP5559185B2 (ja) | 2008-11-11 | 2014-07-23 | オックスフォード バイオメディカ(ユーケー)リミテッド | 方法 |
| 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 |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| LT2841578T (lt) | 2012-04-23 | 2017-09-25 | Biomarin Technologies B.V. | Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui |
| AU2013337354A1 (en) | 2012-11-05 | 2015-05-21 | 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 |
| CA2942515C (en) | 2014-03-18 | 2025-12-09 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| EP3119888B1 (en) * | 2014-03-19 | 2021-07-28 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| 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) |
| EP3285788B1 (en) * | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| CA2985235A1 (en) * | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| US20190328906A1 (en) * | 2016-03-02 | 2019-10-31 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| CN109862785B (zh) * | 2016-09-30 | 2022-09-06 | 瑞泽恩制药公司 | C9orf72基因座中具有六核苷酸重复扩增的非人类动物 |
| WO2020053258A1 (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 BR BR112020008033-0A patent/BR112020008033A2/pt not_active Application Discontinuation
- 2018-10-23 AU AU2018354195A patent/AU2018354195A1/en not_active Abandoned
- 2018-10-23 CA CA3083582A patent/CA3083582A1/en active Pending
- 2018-10-23 CN CN201880081355.4A patent/CN111819281B/zh active Active
- 2018-10-23 US US16/758,077 patent/US20210261981A1/en not_active Abandoned
- 2018-10-23 JP JP2020523011A patent/JP7413256B2/ja active Active
- 2018-10-23 KR KR1020207014683A patent/KR20200075865A/ko active Pending
- 2018-10-23 WO PCT/US2018/057187 patent/WO2019084068A1/en not_active Ceased
- 2018-10-23 CA CA3177979A patent/CA3177979A1/en active Pending
- 2018-10-23 MX MX2020004207A patent/MX2020004207A/es unknown
- 2018-10-23 CN CN202110196181.XA patent/CN113005123A/zh active Pending
- 2018-10-23 EP EP18870021.5A patent/EP3701030A4/en not_active Withdrawn
-
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
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105637090A (zh) * | 2013-10-11 | 2016-06-01 | Ionis制药公司 | 用于调节c9orf72表达的组合物 |
Non-Patent Citations (2)
| Title |
|---|
| Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders;Maria Sara Cipolat Mis 等;Molecular Neurobiology;第54卷(第6期);4466-4476 * |
| guanine nucleotide exchange C9orf72 isoform a [Homo sapiens];无;NCBI Reference Sequence: NP_060795.1;1-3 * |
Also Published As
| Publication number | Publication date |
|---|---|
| IL281057A (en) | 2021-04-29 |
| JP7413256B2 (ja) | 2024-01-15 |
| WO2019084068A1 (en) | 2019-05-02 |
| US20210261981A1 (en) | 2021-08-26 |
| JP2024045131A (ja) | 2024-04-02 |
| EP3701030A4 (en) | 2022-04-20 |
| EP3701030A1 (en) | 2020-09-02 |
| CN111819281A (zh) | 2020-10-23 |
| KR20200075865A (ko) | 2020-06-26 |
| AU2018354195A1 (en) | 2020-06-04 |
| JP2021500049A (ja) | 2021-01-07 |
| IL274129A (en) | 2020-06-30 |
| CA3177979A1 (en) | 2019-05-02 |
| CA3083582A1 (en) | 2019-05-02 |
| MX2020004207A (es) | 2020-11-11 |
| BR112020008033A2 (pt) | 2020-10-27 |
| CN113005123A (zh) | 2021-06-22 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CN111819281B (zh) | 用于神经变性疾病的基因疗法 | |
| AU2023214366B2 (en) | Gene therapies for lysosomal disorders | |
| KR102879598B1 (ko) | 무세포 합성으로부터 수득된 폐쇄 말단 DNA 벡터 및 ceDNA 벡터를 수득하는 방법 | |
| AU2020205228B2 (en) | Gene therapies for lysosomal disorders | |
| AU2020260485B2 (en) | Gene therapies for lysosomal disorders | |
| KR20220006527A (ko) | 리소좀 장애에 대한 유전자 요법 | |
| JP2024028931A (ja) | 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現 | |
| KR20210096168A (ko) | 신경퇴행성 질환을 위한 유전자 요법 | |
| CN116157527A (zh) | 用于溶酶体病症的基因疗法 | |
| KR20230009444A (ko) | 디스페린 이중 벡터에 의한 유전자 요법 | |
| HK40055729A (en) | Gene therapies for neurodegenerative disease | |
| HK40039908A (en) | Gene therapies for neurodegenerative disease | |
| RU2820586C2 (ru) | ДНК-ВЕКТОРЫ С ЗАМКНУТЫМИ КОНЦАМИ, ПОЛУЧАЕМЫЕ ПУТЕМ БЕСКЛЕТОЧНОГО СИНТЕЗА, И СПОСОБ ПОЛУЧЕНИЯ зкДНК-ВЕКТОРОВ | |
| RU2816871C2 (ru) | КОНТРОЛИРУЕМАЯ ЭКСПРЕССИЯ ТРАНСГЕНОВ С ИСПОЛЬЗОВАНИЕМ ДНК-ВЕКТОРОВ С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК) | |
| HK40047428A (en) | Gene therapies for lysosomal disorders | |
| HK40048832A (en) | Gene therapies for lysosomal disorders | |
| HK40046514A (en) | Gene therapies for lysosomal disorders | |
| HK40048832B (zh) | 用於溶酶体障碍的基因疗法 | |
| KR20210150487A (ko) | 리소좀 장애를 위한 유전자 요법 | |
| HK40067825A (zh) | 用於溶酶体障碍的基因疗法 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| REG | Reference to a national code |
Ref country code: HK Ref legal event code: DE Ref document number: 40039908 Country of ref document: HK |
|
| GR01 | Patent grant | ||
| GR01 | Patent grant |