HUE064408T2 - Egyszálú vagy önkomplementer adeno-asszociált vírus 9 injekciója cerebrospinális folyadékba - Google Patents
Egyszálú vagy önkomplementer adeno-asszociált vírus 9 injekciója cerebrospinális folyadékbaInfo
- Publication number
- HUE064408T2 HUE064408T2 HUE17747935A HUE17747935A HUE064408T2 HU E064408 T2 HUE064408 T2 HU E064408T2 HU E17747935 A HUE17747935 A HU E17747935A HU E17747935 A HUE17747935 A HU E17747935A HU E064408 T2 HUE064408 T2 HU E064408T2
- Authority
- HU
- Hungary
- Prior art keywords
- stranded
- injection
- self
- cerebrospinal fluid
- associated virus
- Prior art date
Links
- 241000649044 Adeno-associated virus 9 Species 0.000 title 1
- 210000001175 cerebrospinal fluid Anatomy 0.000 title 1
- 238000002347 injection Methods 0.000 title 1
- 239000007924 injection Substances 0.000 title 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
- C12N15/63—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
-
- 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
- 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)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (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 (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662292157P | 2016-02-05 | 2016-02-05 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE064408T2 true HUE064408T2 (hu) | 2024-03-28 |
Family
ID=59500060
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE17747935A HUE064408T2 (hu) | 2016-02-05 | 2017-01-25 | Egyszálú vagy önkomplementer adeno-asszociált vírus 9 injekciója cerebrospinális folyadékba |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US12070510B2 (hu) |
| EP (2) | EP4299079A3 (hu) |
| JP (3) | JP2019504888A (hu) |
| AU (1) | AU2017214283B2 (hu) |
| CA (1) | CA3012653A1 (hu) |
| DK (1) | DK3411484T3 (hu) |
| ES (1) | ES2962860T3 (hu) |
| FI (1) | FI3411484T3 (hu) |
| HR (1) | HRP20231451T1 (hu) |
| HU (1) | HUE064408T2 (hu) |
| IL (2) | IL300254A (hu) |
| LT (1) | LT3411484T (hu) |
| PL (1) | PL3411484T3 (hu) |
| PT (1) | PT3411484T (hu) |
| SI (1) | SI3411484T1 (hu) |
| WO (1) | WO2017136202A1 (hu) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | 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 |
| JP2019504888A (ja) | 2016-02-05 | 2019-02-21 | エモリー ユニバーシティ | 一本鎖アデノ随伴ウイルス９または自己相補型アデノ随伴ウイルス９の脳脊髄液への注射 |
| EP3452104A1 (en) | 2016-04-15 | 2019-03-13 | The Trustees of The University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| KR102392236B1 (ko) | 2016-05-18 | 2022-05-03 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| EP3831281A1 (en) | 2016-08-30 | 2021-06-09 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| EP3538660A1 (en) | 2016-11-09 | 2019-09-18 | Intrexon Corporation | Frataxin expression constructs |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | 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 |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| 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 |
| KR102693318B1 (ko) | 2017-10-03 | 2024-08-07 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| CN112481269A (zh) | 2017-10-03 | 2021-03-12 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| US20210275647A1 (en) * | 2018-07-18 | 2021-09-09 | Regenxbio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| CA3114199A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| KR20210124299A (ko) * | 2019-02-04 | 2021-10-14 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | Cln6 폴리뉴클레오티드의 아데노-관련 바이러스 전달 |
| WO2020191366A1 (en) * | 2019-03-21 | 2020-09-24 | Ptc Therapeutics, Inc. | Vector and method for treating angelman syndrome |
| WO2020210698A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20220006527A (ko) | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| EP3953484A4 (en) * | 2019-04-12 | 2023-04-12 | University of Massachusetts | AAV-MEDIATED GENE THERAPY FOR LEUCINOSIS |
| EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
| 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 |
| WO2021263060A2 (en) * | 2020-06-24 | 2021-12-30 | Lycia Therapeutics, Inc. | Bifunctional bridging compositions for viral transduction |
| WO2021263061A2 (en) * | 2020-06-24 | 2021-12-30 | Lycia Therapeutics, Inc. | Modified viral compositions for viral transduction |
| AU2021392642A1 (en) * | 2020-12-01 | 2023-06-22 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
Family Cites Families (24)
| 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 |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| WO2000028061A2 (en) | 1998-11-05 | 2000-05-18 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| EP1204739B1 (en) | 1999-08-09 | 2008-08-06 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| 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 |
| WO2003045319A2 (en) * | 2001-11-21 | 2003-06-05 | Activbiotics, Inc. | Targeted therapeutics and uses thereof |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| DK2292780T3 (en) | 2003-09-30 | 2017-12-04 | Univ Pennsylvania | Clades and sequences of adeno-associated virus (AAV), vectors containing them, and uses thereof |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| AU2009209408B2 (en) | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | 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 |
| BR112012018899A2 (pt) | 2010-01-28 | 2015-09-15 | Philadelphia Children Hospital | "método para purificar partículas de vetor de vírus adeno-associado." |
| EP2675484B1 (en) | 2011-02-14 | 2018-05-30 | The Children's Hospital of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| WO2013190059A1 (en) * | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
| BR112015002168A2 (pt) * | 2012-08-01 | 2017-11-07 | Nationwide Childrens Hospital | liberação intratecal de vírus 9 adeno-associado recombinante |
| MX2015015244A (es) * | 2013-05-01 | 2016-07-05 | Genzyme Corp | Composiciones y metodos para tratar la atrofia muscular espinal. |
| EP3632923A1 (en) * | 2015-01-16 | 2020-04-08 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
| JP2019504888A (ja) | 2016-02-05 | 2019-02-21 | エモリー ユニバーシティ | 一本鎖アデノ随伴ウイルス９または自己相補型アデノ随伴ウイルス９の脳脊髄液への注射 |
-
2017
- 2017-01-25 JP JP2018560432A patent/JP2019504888A/ja not_active Withdrawn
- 2017-01-25 IL IL300254A patent/IL300254A/en unknown
- 2017-01-25 PL PL17747935.9T patent/PL3411484T3/pl unknown
- 2017-01-25 PT PT177479359T patent/PT3411484T/pt unknown
- 2017-01-25 HU HUE17747935A patent/HUE064408T2/hu unknown
- 2017-01-25 DK DK17747935.9T patent/DK3411484T3/da active
- 2017-01-25 FI FIEP17747935.9T patent/FI3411484T3/fi active
- 2017-01-25 EP EP23194145.1A patent/EP4299079A3/en active Pending
- 2017-01-25 WO PCT/US2017/014914 patent/WO2017136202A1/en active Application Filing
- 2017-01-25 US US16/075,122 patent/US12070510B2/en active Active
- 2017-01-25 SI SI201731434T patent/SI3411484T1/sl unknown
- 2017-01-25 ES ES17747935T patent/ES2962860T3/es active Active
- 2017-01-25 EP EP17747935.9A patent/EP3411484B1/en active Active
- 2017-01-25 AU AU2017214283A patent/AU2017214283B2/en active Active
- 2017-01-25 LT LTEPPCT/US2017/014914T patent/LT3411484T/lt unknown
- 2017-01-25 CA CA3012653A patent/CA3012653A1/en active Pending
- 2017-01-25 HR HRP20231451TT patent/HRP20231451T1/hr unknown
-
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
Also Published As
| Publication number | Publication date |
|---|---|
| IL260954A (hu) | 2018-09-20 |
| EP3411484B1 (en) | 2023-10-04 |
| CA3012653A1 (en) | 2017-08-10 |
| ES2962860T3 (es) | 2024-03-21 |
| EP4299079A2 (en) | 2024-01-03 |
| US20240108761A1 (en) | 2024-04-04 |
| WO2017136202A1 (en) | 2017-08-10 |
| US20190038777A1 (en) | 2019-02-07 |
| AU2017214283B2 (en) | 2023-06-29 |
| JP2024084797A (ja) | 2024-06-25 |
| DK3411484T3 (da) | 2023-11-20 |
| IL260954B1 (en) | 2023-03-01 |
| PT3411484T (pt) | 2023-11-17 |
| US12070510B2 (en) | 2024-08-27 |
| LT3411484T (lt) | 2023-11-27 |
| JP2019504888A (ja) | 2019-02-21 |
| PL3411484T3 (pl) | 2024-02-19 |
| IL300254A (en) | 2023-03-01 |
| HRP20231451T1 (hr) | 2024-03-01 |
| AU2017214283A1 (en) | 2018-08-02 |
| IL260954B2 (en) | 2023-07-01 |
| JP2022101648A (ja) | 2022-07-06 |
| EP3411484A1 (en) | 2018-12-12 |
| SI3411484T1 (sl) | 2023-12-29 |
| EP4299079A3 (en) | 2024-05-01 |
| EP3411484A4 (en) | 2019-08-07 |
| FI3411484T3 (fi) | 2023-11-15 |
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