PL3411484T3 - Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego - Google Patents
Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowegoInfo
- Publication number
- PL3411484T3 PL3411484T3 PL17747935.9T PL17747935T PL3411484T3 PL 3411484 T3 PL3411484 T3 PL 3411484T3 PL 17747935 T PL17747935 T PL 17747935T PL 3411484 T3 PL3411484 T3 PL 3411484T3
- Authority
- PL
- Poland
- Prior art keywords
- adenosatellite
- virus
- complementary
- threaded
- injection
- Prior art date
Links
- 241000700605 Viruses Species 0.000 title 1
- 210000004556 brain Anatomy 0.000 title 1
- 239000012530 fluid Substances 0.000 title 1
- 238000002347 injection Methods 0.000 title 1
- 239000007924 injection Substances 0.000 title 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61P37/00—Drugs for immunological or allergic disorders
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662292157P | 2016-02-05 | 2016-02-05 | |
| PCT/US2017/014914 WO2017136202A1 (en) | 2016-02-05 | 2017-01-25 | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3411484T3 true PL3411484T3 (pl) | 2024-02-19 |
Family
ID=59500060
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL17747935.9T PL3411484T3 (pl) | 2016-02-05 | 2017-01-25 | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US12070510B2 (pl) |
| EP (2) | EP4299079A3 (pl) |
| JP (4) | JP2019504888A (pl) |
| AU (1) | AU2017214283B2 (pl) |
| CA (1) | CA3012653A1 (pl) |
| DK (1) | DK3411484T3 (pl) |
| ES (1) | ES2962860T3 (pl) |
| FI (1) | FI3411484T3 (pl) |
| HR (1) | HRP20231451T1 (pl) |
| HU (1) | HUE064408T2 (pl) |
| IL (2) | IL300254B2 (pl) |
| LT (1) | LT3411484T (pl) |
| PL (1) | PL3411484T3 (pl) |
| PT (1) | PT3411484T (pl) |
| SI (1) | SI3411484T1 (pl) |
| WO (1) | WO2017136202A1 (pl) |
Families Citing this family (38)
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| RS67077B1 (sr) | 2009-05-02 | 2025-08-29 | Genzyme Corp | Genska terapija za neurodegenerativne poremećaje |
| RU2692251C2 (ru) | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| IL300254B2 (en) | 2016-02-05 | 2025-06-01 | Univ Emory | Administration of single stranded or self-complementary adeno-associated virus 9 by injection into the cerebrospinal fluid to target gene therapy to the central nervous system |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| CA3042706A1 (en) | 2016-11-09 | 2018-05-17 | Intrexon Corporation | Frataxin expression constructs |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| 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 |
| US11802294B2 (en) | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| IL280198B1 (en) * | 2018-07-18 | 2025-09-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| KR20210084459A (ko) | 2018-09-26 | 2021-07-07 | 캘리포니아 인스티튜트 오브 테크놀로지 | 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물 |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CA3120923A1 (en) * | 2018-11-26 | 2020-06-04 | Universitat Autonoma De Barcelona | Fibroblast growth factor 21 (fgf21) gene therapy |
| SG11202107983TA (en) * | 2019-02-04 | 2021-08-30 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus delivery of cln6 polynucleotide |
| CN114206393A (zh) * | 2019-03-21 | 2022-03-18 | Ptc医疗公司 | 用于治疗天使综合征的载体和方法 |
| WO2020210713A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| DK3953377T3 (da) | 2019-04-10 | 2025-12-01 | Prevail Therapeutics Inc | Genterapier til lysosomale lidelser |
| EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
| WO2020210595A1 (en) * | 2019-04-12 | 2020-10-15 | University Of Massachusetts | Aav-mediated gene therapy for maple syrup urine disease (msud) |
| AU2020416291A1 (en) * | 2019-12-31 | 2022-07-21 | Swanbio Therapeutics Limited | Improved AAV-ABCD1 constructs and use for treatment or prevention of adrenoleukodystrophy (ALD) and/or adrenomyeloneuropathy (AMN) |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| EP4171659A4 (en) * | 2020-06-24 | 2025-04-09 | Lycia Therapeutics, Inc. | BIFUNCTIONAL BRIDGE COMPOSITIONS FOR VIRAL TRANSDUCTION |
| US20230279426A1 (en) * | 2020-06-24 | 2023-09-07 | Lycia Therapeutics, Inc. | Modified viral compositions for viral transduction |
| CA3200192A1 (en) * | 2020-12-01 | 2022-06-09 | Justin PERCIVAL | Compositions and uses thereof for treatment of angelman syndrome |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023288033A1 (en) | 2021-07-14 | 2023-01-19 | Lycia Therapeutics, Inc. | Asgpr cell surface receptor binding compounds and conjugates |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6110743A (en) | 1995-02-10 | 2000-08-29 | The Regents Of The University Of California | Development and use of human pancreatic cell lines |
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| 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. |
| US9056048B2 (en) * | 2001-08-16 | 2015-06-16 | The Trustees Of The University Of Pennsylvania | Synthesis and use of cationic steroids for anti-inflammatory drug therapy |
| US7122525B2 (en) * | 2001-11-21 | 2006-10-17 | Activbiotics, Inc. | Targeted therapeutics and uses thereof |
| PT1625210E (pt) | 2003-05-21 | 2011-03-15 | Genzyme Corp | Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| WO2009097129A1 (en) | 2008-01-29 | 2009-08-06 | Applied Genetic Technologies Corporation | Recombinant virus production using mammalian cells in suspension |
| WO2010114948A2 (en) | 2009-04-02 | 2010-10-07 | University Of Florida Research Foundation, Inc. | An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors |
| US8692332B2 (en) | 2010-01-14 | 2014-04-08 | United Microelectronics Corp. | Strained-silicon transistor and method of making the same |
| PT2529020T (pt) | 2010-01-28 | 2018-07-30 | Childrens Hospital Philadelphia | Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica |
| CA2827375C (en) | 2011-02-14 | 2022-07-19 | The Children's Hospital Of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| CN104704123A (zh) * | 2012-06-21 | 2015-06-10 | 肌肉学研究协会 | 基因治疗载体的广泛的基因递送 |
| EP3769789A1 (en) * | 2012-08-01 | 2021-01-27 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| AU2013388083B2 (en) * | 2013-05-01 | 2019-08-22 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| MX2017009336A (es) | 2015-01-16 | 2017-11-15 | Voyager Therapeutics Inc | Polinucleótidos dirigidos al sistema nervioso central. |
| IL300254B2 (en) | 2016-02-05 | 2025-06-01 | Univ Emory | Administration of single stranded or self-complementary adeno-associated virus 9 by injection into the cerebrospinal fluid to target gene therapy to the central nervous system |
-
2017
- 2017-01-25 IL IL300254A patent/IL300254B2/en unknown
- 2017-01-25 EP EP23194145.1A patent/EP4299079A3/en not_active Withdrawn
- 2017-01-25 PT PT177479359T patent/PT3411484T/pt unknown
- 2017-01-25 CA CA3012653A patent/CA3012653A1/en active Pending
- 2017-01-25 PL PL17747935.9T patent/PL3411484T3/pl unknown
- 2017-01-25 HU HUE17747935A patent/HUE064408T2/hu unknown
- 2017-01-25 FI FIEP17747935.9T patent/FI3411484T3/fi active
- 2017-01-25 ES ES17747935T patent/ES2962860T3/es active Active
- 2017-01-25 SI SI201731434T patent/SI3411484T1/sl unknown
- 2017-01-25 JP JP2018560432A patent/JP2019504888A/ja not_active Withdrawn
- 2017-01-25 AU AU2017214283A patent/AU2017214283B2/en active Active
- 2017-01-25 WO PCT/US2017/014914 patent/WO2017136202A1/en not_active Ceased
- 2017-01-25 HR HRP20231451TT patent/HRP20231451T1/hr unknown
- 2017-01-25 DK DK17747935.9T patent/DK3411484T3/da active
- 2017-01-25 EP EP17747935.9A patent/EP3411484B1/en active Active
- 2017-01-25 LT LTEPPCT/US2017/014914T patent/LT3411484T/lt unknown
- 2017-01-25 US US16/075,122 patent/US12070510B2/en active Active
-
2018
- 2018-08-02 IL IL260954A patent/IL260954B2/en unknown
-
2022
- 2022-04-22 JP JP2022070524A patent/JP2022101648A/ja active Pending
-
2023
- 2023-11-07 US US18/504,029 patent/US20240108761A1/en not_active Abandoned
-
2024
- 2024-04-03 JP JP2024059869A patent/JP2024084797A/ja active Pending
- 2024-07-12 US US18/771,234 patent/US20250135041A1/en active Pending
- 2024-12-26 JP JP2024230721A patent/JP2025041933A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2022101648A (ja) | 2022-07-06 |
| IL260954B1 (en) | 2023-03-01 |
| JP2025041933A (ja) | 2025-03-26 |
| EP4299079A2 (en) | 2024-01-03 |
| JP2024084797A (ja) | 2024-06-25 |
| HUE064408T2 (hu) | 2024-03-28 |
| IL300254B1 (en) | 2025-02-01 |
| PT3411484T (pt) | 2023-11-17 |
| JP2019504888A (ja) | 2019-02-21 |
| IL300254B2 (en) | 2025-06-01 |
| EP3411484A4 (en) | 2019-08-07 |
| US12070510B2 (en) | 2024-08-27 |
| EP3411484B1 (en) | 2023-10-04 |
| US20250135041A1 (en) | 2025-05-01 |
| ES2962860T3 (es) | 2024-03-21 |
| SI3411484T1 (sl) | 2023-12-29 |
| CA3012653A1 (en) | 2017-08-10 |
| US20240108761A1 (en) | 2024-04-04 |
| IL300254A (en) | 2023-03-01 |
| IL260954A (pl) | 2018-09-20 |
| FI3411484T3 (fi) | 2023-11-15 |
| DK3411484T3 (da) | 2023-11-20 |
| IL260954B2 (en) | 2023-07-01 |
| EP3411484A1 (en) | 2018-12-12 |
| HRP20231451T1 (hr) | 2024-03-01 |
| WO2017136202A1 (en) | 2017-08-10 |
| US20190038777A1 (en) | 2019-02-07 |
| AU2017214283B2 (en) | 2023-06-29 |
| EP4299079A3 (en) | 2024-05-01 |
| LT3411484T (lt) | 2023-11-27 |
| AU2017214283A1 (en) | 2018-08-02 |
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