PL3294323T3 - Wirus związany z adenowirusem do terapeutycznego dostarczania do ośrodkowego układu nerwowego - Google Patents
Wirus związany z adenowirusem do terapeutycznego dostarczania do ośrodkowego układu nerwowegoInfo
- Publication number
- PL3294323T3 PL3294323T3 PL16797013T PL16797013T PL3294323T3 PL 3294323 T3 PL3294323 T3 PL 3294323T3 PL 16797013 T PL16797013 T PL 16797013T PL 16797013 T PL16797013 T PL 16797013T PL 3294323 T3 PL3294323 T3 PL 3294323T3
- Authority
- PL
- Poland
- Prior art keywords
- adeno
- nervous system
- central nervous
- associated virus
- therapeutic delivery
- Prior art date
Links
Classifications
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- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/66—Phosphorus compounds
- A61K31/675—Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0083—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 administration regime
-
- 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
- A61K9/0043—Nose
-
- 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
- C12N15/63—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
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562162174P | 2015-05-15 | 2015-05-15 | |
US201562252055P | 2015-11-06 | 2015-11-06 | |
US201662301980P | 2016-03-01 | 2016-03-01 | |
US201662331156P | 2016-05-03 | 2016-05-03 | |
EP16797013.6A EP3294323B1 (en) | 2015-05-15 | 2016-05-13 | Adeno-associated virus for therapeutic delivery to central nervous system |
PCT/US2016/032392 WO2016187017A1 (en) | 2015-05-15 | 2016-05-13 | Adeno-associated for therapeutic delivery to central nervous system |
Publications (1)
Publication Number | Publication Date |
---|---|
PL3294323T3 true PL3294323T3 (pl) | 2022-06-20 |
Family
ID=57320277
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL16797013T PL3294323T3 (pl) | 2015-05-15 | 2016-05-13 | Wirus związany z adenowirusem do terapeutycznego dostarczania do ośrodkowego układu nerwowego |
Country Status (22)
Country | Link |
---|---|
US (5) | US20180289839A1 (pl) |
EP (2) | EP3294323B1 (pl) |
JP (3) | JP2018515615A (pl) |
KR (2) | KR102406615B1 (pl) |
CN (1) | CN108367055A (pl) |
AU (2) | AU2016263119B2 (pl) |
BR (1) | BR112017024519A2 (pl) |
CA (1) | CA2986252A1 (pl) |
CY (1) | CY1125229T1 (pl) |
DK (1) | DK3294323T3 (pl) |
ES (1) | ES2901766T3 (pl) |
HK (2) | HK1252871A1 (pl) |
HR (1) | HRP20220050T1 (pl) |
HU (1) | HUE057795T2 (pl) |
LT (1) | LT3294323T (pl) |
MX (2) | MX2017014443A (pl) |
PL (1) | PL3294323T3 (pl) |
PT (1) | PT3294323T (pl) |
RS (1) | RS63927B1 (pl) |
RU (1) | RU2751952C2 (pl) |
SI (1) | SI3294323T1 (pl) |
WO (1) | WO2016187017A1 (pl) |
Families Citing this family (25)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2014152832A1 (en) | 2013-03-14 | 2014-09-25 | Immusoft Corporation | Methods for in vitro memory b cell differentiation and transduction with vsv-g pseudotyped viral vectors |
SG11201507507PA (en) | 2013-03-15 | 2015-10-29 | Univ Pennsylvania | Compositions and methods for treating mpsi |
CA2912678C (en) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
DK3294323T3 (da) * | 2015-05-15 | 2022-01-10 | Univ Minnesota | Adeno-associeret virus til terapeutisk indgivelse til centralnervesystemet |
WO2017136500A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
WO2018093925A1 (en) * | 2016-11-15 | 2018-05-24 | Laoharawee Kanut | Method for improving neurological function in mpsi and mpsii and other neurological disorders |
SG11201909361SA (en) | 2017-04-14 | 2019-11-28 | Regenxbio Inc | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells |
AU2018256887A1 (en) * | 2017-04-27 | 2019-11-07 | Immusoft Corporation | B cells for in vivo delivery of therapeutic agents and dosages thereof |
CA3068328A1 (en) * | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
JP7254815B2 (ja) | 2017-10-03 | 2023-04-10 | プリベイル セラピューティクス,インコーポレーテッド | ライソゾーム病の遺伝子治療 |
KR20200078513A (ko) | 2017-10-03 | 2020-07-01 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
MX2020003965A (es) | 2017-10-03 | 2020-10-05 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
EA202091352A1 (ru) | 2017-11-30 | 2020-08-27 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Генотерапия мукополисахаридоза iiib |
WO2019199841A1 (en) * | 2018-04-09 | 2019-10-17 | Cure Ahc, Inc. | Aav-mediated delivery of atp1a3 genes to central nervous system |
CA3106651A1 (en) * | 2018-07-18 | 2020-01-23 | Stephen YOO | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus) |
JP2022526021A (ja) | 2019-04-10 | 2022-05-20 | 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 |
SG11202111867XA (en) * | 2019-05-03 | 2021-11-29 | Univ Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
WO2020251954A1 (en) * | 2019-06-10 | 2020-12-17 | Homology Medicines, Inc. | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |
WO2022035903A2 (en) * | 2020-08-10 | 2022-02-17 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
CN117642173A (zh) * | 2020-12-07 | 2024-03-01 | 华东理工大学 | 用于诱导对基因递送靶媒剂的免疫耐受性的方法和试剂盒 |
EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
WO2022212616A1 (en) * | 2021-04-01 | 2022-10-06 | The University Of North Carolina At Chapel Hill | Aav-ids vectors for treatment of mucopolysaccharidosis ii |
Family Cites Families (19)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
JP3952312B2 (ja) | 1993-11-09 | 2007-08-01 | メディカル カレッジ オブ オハイオ | アデノ関連ウイルス複製遺伝子を発現可能な安定な細胞株 |
PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
US5656785A (en) | 1995-08-07 | 1997-08-12 | The Charles Stark Draper Laboratory, Inc. | Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor |
AU5445998A (en) | 1996-11-19 | 1998-06-10 | Surgx Corporation | A transient voltage protection device and method of making same |
JP2001506133A (ja) | 1996-12-18 | 2001-05-15 | ターゲティッド ジェネティクス コーポレイション | 組換えaavベクターの産生における使用のための、aavスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株 |
EP1915986A1 (en) | 2006-10-23 | 2008-04-30 | BIOPHARM GESELLSCHAFT ZUR BIOTECHNOLOGISCHEN ENTWICKLUNG VON PHARMAKA mbH | Lipid growth factor formulations |
ES2634118T3 (es) * | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
KR20230159646A (ko) * | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
US9849195B2 (en) * | 2011-03-31 | 2017-12-26 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
US8609088B2 (en) * | 2011-05-10 | 2013-12-17 | Regents Of The University Of Minnesota | Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases |
US20130039888A1 (en) * | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
AU2012328682B2 (en) * | 2011-10-27 | 2017-09-21 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of the HPRT locus |
CA2912678C (en) * | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
RU2018128780A (ru) * | 2013-07-26 | 2018-12-05 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
DK3294323T3 (da) * | 2015-05-15 | 2022-01-10 | Univ Minnesota | Adeno-associeret virus til terapeutisk indgivelse til centralnervesystemet |
WO2018093925A1 (en) * | 2016-11-15 | 2018-05-24 | Laoharawee Kanut | Method for improving neurological function in mpsi and mpsii and other neurological disorders |
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2016
- 2016-05-13 DK DK16797013.6T patent/DK3294323T3/da active
- 2016-05-13 CN CN201680037625.2A patent/CN108367055A/zh active Pending
- 2016-05-13 PT PT167970136T patent/PT3294323T/pt unknown
- 2016-05-13 RS RS20211570A patent/RS63927B1/sr unknown
- 2016-05-13 PL PL16797013T patent/PL3294323T3/pl unknown
- 2016-05-13 WO PCT/US2016/032392 patent/WO2016187017A1/en active Application Filing
- 2016-05-13 CA CA2986252A patent/CA2986252A1/en active Pending
- 2016-05-13 SI SI201631430T patent/SI3294323T1/sl unknown
- 2016-05-13 EP EP16797013.6A patent/EP3294323B1/en active Active
- 2016-05-13 MX MX2017014443A patent/MX2017014443A/es unknown
- 2016-05-13 HU HUE16797013A patent/HUE057795T2/hu unknown
- 2016-05-13 BR BR112017024519A patent/BR112017024519A2/pt active Search and Examination
- 2016-05-13 RU RU2017143640A patent/RU2751952C2/ru active
- 2016-05-13 AU AU2016263119A patent/AU2016263119B2/en active Active
- 2016-05-13 EP EP21201643.0A patent/EP4000631A1/en active Pending
- 2016-05-13 LT LTEPPCT/US2016/032392T patent/LT3294323T/lt unknown
- 2016-05-13 KR KR1020177036062A patent/KR102406615B1/ko active IP Right Grant
- 2016-05-13 HR HRP20220050TT patent/HRP20220050T1/hr unknown
- 2016-05-13 US US15/574,432 patent/US20180289839A1/en not_active Abandoned
- 2016-05-13 ES ES16797013T patent/ES2901766T3/es active Active
- 2016-05-13 JP JP2018511353A patent/JP2018515615A/ja active Pending
- 2016-05-13 KR KR1020227018782A patent/KR20220082100A/ko active Application Filing
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2017
- 2017-11-10 MX MX2022012524A patent/MX2022012524A/es unknown
- 2017-11-15 US US15/813,908 patent/US20180071373A1/en not_active Abandoned
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2018
- 2018-09-21 HK HK18112215.4A patent/HK1252871A1/zh unknown
- 2018-12-03 HK HK18115428.0A patent/HK1256341A1/zh unknown
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2021
- 2021-06-10 US US17/344,308 patent/US20210369871A1/en not_active Abandoned
- 2021-07-05 JP JP2021111318A patent/JP2021167332A/ja active Pending
- 2021-11-12 AU AU2021266344A patent/AU2021266344A1/en active Pending
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2022
- 2022-01-12 US US17/574,118 patent/US20220211875A1/en active Pending
- 2022-01-13 CY CY20221100032T patent/CY1125229T1/el unknown
- 2022-07-19 JP JP2022114547A patent/JP2022137261A/ja active Pending
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2023
- 2023-01-05 US US18/150,740 patent/US20230226225A1/en active Pending
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