HUE057795T2 - Adenoasszociált vírus terápiás szerek központi idegrendszerbe történõ bejuttatására - Google Patents
Adenoasszociált vírus terápiás szerek központi idegrendszerbe történõ bejuttatásáraInfo
- Publication number
- HUE057795T2 HUE057795T2 HUE16797013A HUE16797013A HUE057795T2 HU E057795 T2 HUE057795 T2 HU E057795T2 HU E16797013 A HUE16797013 A HU E16797013A HU E16797013 A HUE16797013 A HU E16797013A HU E057795 T2 HUE057795 T2 HU E057795T2
- Authority
- HU
- Hungary
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Analytical Chemistry (AREA)
- Otolaryngology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
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 |
Publications (1)
Publication Number | Publication Date |
---|---|
HUE057795T2 true HUE057795T2 (hu) | 2022-06-28 |
Family
ID=57320277
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE16797013A HUE057795T2 (hu) | 2015-05-15 | 2016-05-13 | Adenoasszociált vírus terápiás szerek központi idegrendszerbe történõ bejuttatására |
Country Status (22)
Country | Link |
---|---|
US (5) | US20180289839A1 (hu) |
EP (2) | EP4000631A1 (hu) |
JP (3) | JP2018515615A (hu) |
KR (2) | KR102406615B1 (hu) |
CN (1) | CN108367055A (hu) |
AU (2) | AU2016263119B2 (hu) |
BR (1) | BR112017024519A2 (hu) |
CA (1) | CA2986252A1 (hu) |
CY (1) | CY1125229T1 (hu) |
DK (1) | DK3294323T3 (hu) |
ES (1) | ES2901766T3 (hu) |
HK (2) | HK1252871A1 (hu) |
HR (1) | HRP20220050T1 (hu) |
HU (1) | HUE057795T2 (hu) |
LT (1) | LT3294323T (hu) |
MX (2) | MX2017014443A (hu) |
PL (1) | PL3294323T3 (hu) |
PT (1) | PT3294323T (hu) |
RS (1) | RS63927B1 (hu) |
RU (1) | RU2751952C2 (hu) |
SI (1) | SI3294323T1 (hu) |
WO (1) | WO2016187017A1 (hu) |
Families Citing this family (27)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2971039B1 (en) | 2013-03-14 | 2020-01-01 | Immusoft Corporation | Methods for in vitro memory b cell differentiation and transduction with vsv-g pseudotyped viral vectors |
PL2984166T3 (pl) | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
BR112015028605A8 (pt) * | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
MX2017014443A (es) * | 2015-05-15 | 2018-08-01 | Univ Minnesota | Adeno-asociado para administracion terapeutica al sistema nervioso central. |
SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
CA3019427A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
IL309808A (en) * | 2016-11-15 | 2024-02-01 | Univ Minnesota | A method to improve neurological function in MPSI and MPSII and other neurological disorders |
IL307936A (en) * | 2017-04-14 | 2023-12-01 | Regenxbio Inc | Treatment for mucopolysaccharidosis 2 with the help of recombinant human iduronate-2 suplatase (ids) produced by human neuron or glial cells |
CA3061048A1 (en) * | 2017-04-27 | 2018-11-01 | Immusoft Corporation | B cells for in vivo delivery of therapeutic agents and dosages thereof |
EP3676385A1 (en) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
AU2018346105C1 (en) | 2017-10-03 | 2023-10-12 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
CN111542549A (zh) | 2017-10-03 | 2020-08-14 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
US11807849B2 (en) | 2017-10-03 | 2023-11-07 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
CN111989396A (zh) | 2017-11-30 | 2020-11-24 | 宾夕法尼亚州大学信托人 | 用于iiib型粘多糖贮积病的基因疗法 |
WO2019199841A1 (en) * | 2018-04-09 | 2019-10-17 | Cure Ahc, Inc. | Aav-mediated delivery of atp1a3 genes to central nervous system |
AU2019306236A1 (en) * | 2018-07-18 | 2021-03-11 | Regenxbio Inc. | Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-L-iduronidase (IDUS) |
CA3136117A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
AU2020270421A1 (en) * | 2019-05-03 | 2021-11-18 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
AU2020292256B2 (en) * | 2019-06-10 | 2023-01-19 | Homology Medicines, Inc. | Adeno-associated virus compositions for ARSA gene transfer and methods of use thereof |
KR20220112283A (ko) | 2019-12-10 | 2022-08-10 | 다케다 야쿠힌 고교 가부시키가이샤 | Hunter 질환 치료용 아데노-연합된 바이러스 벡터 |
US20230310654A1 (en) * | 2020-08-10 | 2023-10-05 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
WO2022121860A1 (en) * | 2020-12-07 | 2022-06-16 | East China University Of Science And Technology | Methods and kits for inducing immunotolerance to gene delivering target vehicle |
EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
CA3209611A1 (en) | 2021-02-10 | 2022-08-18 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) |
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 |
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US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
AU688428B2 (en) | 1993-11-09 | 1998-03-12 | Johns Hopkins University, The | Generation of high titers of recombinant AAV vectors |
ES2220923T3 (es) | 1993-11-09 | 2004-12-16 | Medical College Of Ohio | Lineas celulares estables capaces de expresar el gen de replicacion del virus adeno-asociado. |
EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | 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 |
JP4397972B2 (ja) | 1996-11-19 | 2010-01-13 | サージックス コーポレイション | 過渡電圧保護素子およびその製造方法 |
CA2270285A1 (en) | 1996-12-18 | 1998-06-25 | Targeted Genetics Corporation | Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors |
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 |
UA115649C2 (uk) * | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Способи та композиції для доставки до цнс ідуронат-2-сульфатази |
EP3546584B1 (en) * | 2011-03-31 | 2024-02-14 | University of Iowa Research Foundation | Compositions for treating lysosmal storage disease (lsd) |
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 |
BR112015028605A8 (pt) * | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
US10391184B2 (en) * | 2013-07-26 | 2019-08-27 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
MX2017014443A (es) * | 2015-05-15 | 2018-08-01 | Univ Minnesota | Adeno-asociado para administracion terapeutica al sistema nervioso central. |
IL309808A (en) * | 2016-11-15 | 2024-02-01 | Univ Minnesota | A method to improve neurological function in MPSI and MPSII and other neurological disorders |
-
2016
- 2016-05-13 MX MX2017014443A patent/MX2017014443A/es unknown
- 2016-05-13 DK DK16797013.6T patent/DK3294323T3/da active
- 2016-05-13 US US15/574,432 patent/US20180289839A1/en not_active Abandoned
- 2016-05-13 PT PT167970136T patent/PT3294323T/pt unknown
- 2016-05-13 CA CA2986252A patent/CA2986252A1/en active Pending
- 2016-05-13 KR KR1020177036062A patent/KR102406615B1/ko active IP Right Grant
- 2016-05-13 ES ES16797013T patent/ES2901766T3/es active Active
- 2016-05-13 RU RU2017143640A patent/RU2751952C2/ru active
- 2016-05-13 WO PCT/US2016/032392 patent/WO2016187017A1/en active Application Filing
- 2016-05-13 EP EP21201643.0A patent/EP4000631A1/en active Pending
- 2016-05-13 JP JP2018511353A patent/JP2018515615A/ja active Pending
- 2016-05-13 CN CN201680037625.2A patent/CN108367055A/zh active Pending
- 2016-05-13 BR BR112017024519A patent/BR112017024519A2/pt active Search and Examination
- 2016-05-13 LT LTEPPCT/US2016/032392T patent/LT3294323T/lt unknown
- 2016-05-13 EP EP16797013.6A patent/EP3294323B1/en active Active
- 2016-05-13 KR KR1020227018782A patent/KR20220082100A/ko not_active Application Discontinuation
- 2016-05-13 RS RS20211570A patent/RS63927B1/sr unknown
- 2016-05-13 SI SI201631430T patent/SI3294323T1/sl unknown
- 2016-05-13 AU AU2016263119A patent/AU2016263119B2/en active Active
- 2016-05-13 PL PL16797013T patent/PL3294323T3/pl unknown
- 2016-05-13 HR HRP20220050TT patent/HRP20220050T1/hr unknown
- 2016-05-13 HU HUE16797013A patent/HUE057795T2/hu unknown
-
2017
- 2017-11-10 MX MX2022012524A patent/MX2022012524A/es unknown
- 2017-11-15 US US15/813,908 patent/US20180071373A1/en not_active Abandoned
-
2018
- 2018-09-21 HK HK18112215.4A patent/HK1252871A1/zh unknown
- 2018-12-03 HK HK18115428.0A patent/HK1256341A1/zh unknown
-
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
-
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
-
2023
- 2023-01-05 US US18/150,740 patent/US20230226225A1/en active Pending
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