HRP20220050T1 - Adenoasocirani virus, namijenjen terapijskom unosu u središnji živčani sustav - Google Patents
Adenoasocirani virus, namijenjen terapijskom unosu u središnji živčani sustav Download PDFInfo
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- HRP20220050T1 HRP20220050T1 HRP20220050TT HRP20220050T HRP20220050T1 HR P20220050 T1 HRP20220050 T1 HR P20220050T1 HR P20220050T T HRP20220050T T HR P20220050TT HR P20220050 T HRP20220050 T HR P20220050T HR P20220050 T1 HRP20220050 T1 HR P20220050T1
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- immunosuppressant
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- 210000003169 central nervous system Anatomy 0.000 title 1
- 230000001225 therapeutic effect Effects 0.000 title 1
- 241000124008 Mammalia Species 0.000 claims 7
- 229960003444 immunosuppressant agent Drugs 0.000 claims 5
- 230000001861 immunosuppressant effect Effects 0.000 claims 5
- 239000003018 immunosuppressive agent Substances 0.000 claims 5
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 3
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 3
- 108010002350 Interleukin-2 Proteins 0.000 claims 2
- NWIBSHFKIJFRCO-WUDYKRTCSA-N Mytomycin Chemical compound C1N2C(C(C(C)=C(N)C3=O)=O)=C3[C@@H](COC(N)=O)[C@@]2(OC)[C@@H]2[C@H]1N2 NWIBSHFKIJFRCO-WUDYKRTCSA-N 0.000 claims 2
- 108060008682 Tumor Necrosis Factor Proteins 0.000 claims 2
- 102000000852 Tumor Necrosis Factor-alpha Human genes 0.000 claims 2
- RJURFGZVJUQBHK-UHFFFAOYSA-N actinomycin D Natural products CC1OC(=O)C(C(C)C)N(C)C(=O)CN(C)C(=O)C2CCCN2C(=O)C(C(C)C)NC(=O)C1NC(=O)C1=C(N)C(=O)C(C)=C2OC(C(C)=CC=C3C(=O)NC4C(=O)NC(C(N5CCCC5C(=O)N(C)CC(=O)N(C)C(C(C)C)C(=O)OC4C)=O)C(C)C)=C3N=C21 RJURFGZVJUQBHK-UHFFFAOYSA-N 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- 108010006654 Bleomycin Proteins 0.000 claims 1
- 201000006306 Cor pulmonale Diseases 0.000 claims 1
- CMSMOCZEIVJLDB-UHFFFAOYSA-N Cyclophosphamide Chemical compound ClCCN(CCCl)P1(=O)NCCCO1 CMSMOCZEIVJLDB-UHFFFAOYSA-N 0.000 claims 1
- PMATZTZNYRCHOR-CGLBZJNRSA-N Cyclosporin A Chemical compound CC[C@@H]1NC(=O)[C@H]([C@H](O)[C@H](C)C\C=C\C)N(C)C(=O)[C@H](C(C)C)N(C)C(=O)[C@H](CC(C)C)N(C)C(=O)[C@H](CC(C)C)N(C)C(=O)[C@@H](C)NC(=O)[C@H](C)NC(=O)[C@H](CC(C)C)N(C)C(=O)[C@H](C(C)C)NC(=O)[C@H](CC(C)C)N(C)C(=O)CN(C)C1=O PMATZTZNYRCHOR-CGLBZJNRSA-N 0.000 claims 1
- 108010036949 Cyclosporine Proteins 0.000 claims 1
- 108010092160 Dactinomycin Proteins 0.000 claims 1
- GHASVSINZRGABV-UHFFFAOYSA-N Fluorouracil Chemical compound FC1=CNC(=O)NC1=O GHASVSINZRGABV-UHFFFAOYSA-N 0.000 claims 1
- 206010053759 Growth retardation Diseases 0.000 claims 1
- 101001057504 Homo sapiens Interferon-stimulated gene 20 kDa protein Proteins 0.000 claims 1
- 101001055144 Homo sapiens Interleukin-2 receptor subunit alpha Proteins 0.000 claims 1
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims 1
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims 1
- 102000000521 Immunophilins Human genes 0.000 claims 1
- 108010016648 Immunophilins Proteins 0.000 claims 1
- 102100026720 Interferon beta Human genes 0.000 claims 1
- 102100037850 Interferon gamma Human genes 0.000 claims 1
- 108090000467 Interferon-beta Proteins 0.000 claims 1
- 108010074328 Interferon-gamma Proteins 0.000 claims 1
- 102100026878 Interleukin-2 receptor subunit alpha Human genes 0.000 claims 1
- FBOZXECLQNJBKD-ZDUSSCGKSA-N L-methotrexate Chemical compound C=1N=C2N=C(N)N=C(N)C2=NC=1CN(C)C1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 FBOZXECLQNJBKD-ZDUSSCGKSA-N 0.000 claims 1
- 108700026244 Open Reading Frames Proteins 0.000 claims 1
- 206010072610 Skeletal dysplasia Diseases 0.000 claims 1
- QJJXYPPXXYFBGM-LFZNUXCKSA-N Tacrolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1\C=C(/C)[C@@H]1[C@H](C)[C@@H](O)CC(=O)[C@H](CC=C)/C=C(C)/C[C@H](C)C[C@H](OC)[C@H]([C@H](C[C@H]2C)OC)O[C@@]2(O)C(=O)C(=O)N2CCCC[C@H]2C(=O)O1 QJJXYPPXXYFBGM-LFZNUXCKSA-N 0.000 claims 1
- RJURFGZVJUQBHK-IIXSONLDSA-N actinomycin D Chemical compound C[C@H]1OC(=O)[C@H](C(C)C)N(C)C(=O)CN(C)C(=O)[C@@H]2CCCN2C(=O)[C@@H](C(C)C)NC(=O)[C@H]1NC(=O)C1=C(N)C(=O)C(C)=C2OC(C(C)=CC=C3C(=O)N[C@@H]4C(=O)N[C@@H](C(N5CCC[C@H]5C(=O)N(C)CC(=O)N(C)[C@@H](C(C)C)C(=O)O[C@@H]4C)=O)C(C)C)=C3N=C21 RJURFGZVJUQBHK-IIXSONLDSA-N 0.000 claims 1
- 229940100198 alkylating agent Drugs 0.000 claims 1
- 239000002168 alkylating agent Substances 0.000 claims 1
- 229940045799 anthracyclines and related substance Drugs 0.000 claims 1
- 230000000340 anti-metabolite Effects 0.000 claims 1
- 229940100197 antimetabolite Drugs 0.000 claims 1
- 239000002256 antimetabolite Substances 0.000 claims 1
- 229960002170 azathioprine Drugs 0.000 claims 1
- LMEKQMALGUDUQG-UHFFFAOYSA-N azathioprine Chemical compound CN1C=NC([N+]([O-])=O)=C1SC1=NC=NC2=C1NC=N2 LMEKQMALGUDUQG-UHFFFAOYSA-N 0.000 claims 1
- 230000003115 biocidal effect Effects 0.000 claims 1
- 229960001561 bleomycin Drugs 0.000 claims 1
- OYVAGSVQBOHSSS-UAPAGMARSA-O bleomycin A2 Chemical compound N([C@H](C(=O)N[C@H](C)[C@@H](O)[C@H](C)C(=O)N[C@@H]([C@H](O)C)C(=O)NCCC=1SC=C(N=1)C=1SC=C(N=1)C(=O)NCCC[S+](C)C)[C@@H](O[C@H]1[C@H]([C@@H](O)[C@H](O)[C@H](CO)O1)O[C@@H]1[C@H]([C@@H](OC(N)=O)[C@H](O)[C@@H](CO)O1)O)C=1N=CNC=1)C(=O)C1=NC([C@H](CC(N)=O)NC[C@H](N)C(N)=O)=NC(N)=C1C OYVAGSVQBOHSSS-UAPAGMARSA-O 0.000 claims 1
- 229960001265 ciclosporin Drugs 0.000 claims 1
- 229960004397 cyclophosphamide Drugs 0.000 claims 1
- 229930182912 cyclosporin Natural products 0.000 claims 1
- 239000000824 cytostatic agent Substances 0.000 claims 1
- 231100000433 cytotoxic Toxicity 0.000 claims 1
- 230000001472 cytotoxic effect Effects 0.000 claims 1
- 229960000640 dactinomycin Drugs 0.000 claims 1
- CFCUWKMKBJTWLW-UHFFFAOYSA-N deoliosyl-3C-alpha-L-digitoxosyl-MTM Natural products CC=1C(O)=C2C(O)=C3C(=O)C(OC4OC(C)C(O)C(OC5OC(C)C(O)C(OC6OC(C)C(O)C(C)(O)C6)C5)C4)C(C(OC)C(=O)C(O)C(C)O)CC3=CC2=CC=1OC(OC(C)C1O)CC1OC1CC(O)C(O)C(C)O1 CFCUWKMKBJTWLW-UHFFFAOYSA-N 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 229960002949 fluorouracil Drugs 0.000 claims 1
- 239000003862 glucocorticoid Substances 0.000 claims 1
- 231100000001 growth retardation Toxicity 0.000 claims 1
- HAWPXGHAZFHHAD-UHFFFAOYSA-N mechlorethamine Chemical class ClCCN(C)CCCl HAWPXGHAZFHHAD-UHFFFAOYSA-N 0.000 claims 1
- 229960004961 mechlorethamine Drugs 0.000 claims 1
- GLVAUDGFNGKCSF-UHFFFAOYSA-N mercaptopurine Chemical compound S=C1NC=NC2=C1NC=N2 GLVAUDGFNGKCSF-UHFFFAOYSA-N 0.000 claims 1
- 229960001428 mercaptopurine Drugs 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 229960000485 methotrexate Drugs 0.000 claims 1
- CFCUWKMKBJTWLW-BKHRDMLASA-N mithramycin Chemical compound O([C@@H]1C[C@@H](O[C@H](C)[C@H]1O)OC=1C=C2C=C3C[C@H]([C@@H](C(=O)C3=C(O)C2=C(O)C=1C)O[C@@H]1O[C@H](C)[C@@H](O)[C@H](O[C@@H]2O[C@H](C)[C@H](O)[C@H](O[C@@H]3O[C@H](C)[C@@H](O)[C@@](C)(O)C3)C2)C1)[C@H](OC)C(=O)[C@@H](O)[C@@H](C)O)[C@H]1C[C@@H](O)[C@H](O)[C@@H](C)O1 CFCUWKMKBJTWLW-BKHRDMLASA-N 0.000 claims 1
- 229960004857 mitomycin Drugs 0.000 claims 1
- 230000007106 neurocognition Effects 0.000 claims 1
- OSTGTTZJOCZWJG-UHFFFAOYSA-N nitrosourea Chemical compound NC(=O)N=NO OSTGTTZJOCZWJG-UHFFFAOYSA-N 0.000 claims 1
- 150000003058 platinum compounds Chemical class 0.000 claims 1
- 229960003171 plicamycin Drugs 0.000 claims 1
- ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 claims 1
- 108020003175 receptors Proteins 0.000 claims 1
- 102000005962 receptors Human genes 0.000 claims 1
- 229960002930 sirolimus Drugs 0.000 claims 1
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 claims 1
- 229960001967 tacrolimus Drugs 0.000 claims 1
- QJJXYPPXXYFBGM-SHYZHZOCSA-N tacrolimus Natural products CO[C@H]1C[C@H](CC[C@@H]1O)C=C(C)[C@H]2OC(=O)[C@H]3CCCCN3C(=O)C(=O)[C@@]4(O)O[C@@H]([C@H](C[C@H]4C)OC)[C@@H](C[C@H](C)CC(=C[C@@H](CC=C)C(=O)C[C@H](O)[C@H]2C)C)OC QJJXYPPXXYFBGM-SHYZHZOCSA-N 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
- 230000003442 weekly effect Effects 0.000 claims 1
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
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- 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
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- 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
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- 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)
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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
- 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
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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
- 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
Claims (12)
1. Pripravak koji sadrži djelotvornu količinu rekombinantnog adenoasociranog virusnog (rAAV) vektora koji sadrži otvoreni okvir čitanja koji kodira iduronat-2-sulfatazu, naznačen time što je namijenjen upotrebi u postupku poboljšavanja neurokognicije kod sisavca koji ima poremećaj mukopolisaharidoze tip II (MPSII) u odnosu na sisavca koji ima MPSII na kojem nije primijenjen rAAV, gdje je rAAV rAAV9 ili rAAVrh10.
2. Pripravak namijenjen upotrebi u skladu s patentnim zahtjevom 1, naznačen time što se sisavca ne liječi imunosupresivom.
3. Pripravak namijenjen upotrebi u skladu s patentnim zahtjevom 1 ili 2, naznačen time što se sisavca liječi imunosupresivom.
4. Pripravak namijenjen upotrebi u skladu s patentnim zahtjevom 3, naznačen time što imunosupresiv sadrži glukokortikoid, citostatska sredstva, uključujući alkilirajuće sredstvo, antimetabolit, citotoksični antibiotik, protutijelo, sredstvo aktivno na imunofilin, ciklofosfamid, dušični iperit, nitrozoureu, platinski spoj, methotrexate, azatioprin, merkaptopurin, fluoruracil, daktinomicin, antraciklin, mitomicin C, bleomicin, mitramicin, protutijela usmjerena na receptor za IL-2 (CD25) ili CD3, protutijela protiv IL-2, ciklosporin, takrolimus, sirolimus, IFN-β, IFN-γ, opioid, ili sredstvo koje se veže na TNF-α (čimbenik tumorske nekroze α).
5. Pripravak namijenjen upotrebi u skladu s patentnim zahtjevom 4, naznačen time što se rAAV suprimjenjuje s imunosupresivom ili se imunosupresiv se primjenjuje nakon rAAV.
6. Pripravak namijenjen upotrebi u skladu s bilo kojim od patentnih zahtjeva 1 do 5, naznačen time što sisavac nije imunotoleriziran prije primjene rAAV.
7. Pripravak namijenjen upotrebi u skladu s bilo kojim od patentnih zahtjeva 1 do 5, naznačen time što je sisavac imunotoleriziran prije primjene rAAV.
8. Pripravak namijenjen upotrebi u skladu s patentnim zahtjevom 1, naznačen time što je sisavac čovjek ili imunokompetentna odrasla osoba.
9. Pripravak namijenjen upotrebi u skladu s bilo kojim od patentnih zahtjeva 1 do 8, naznačen time što je rAAV kao vektor rAAV9 kao vektor.
10. Pripravak namijenjen upotrebi u skladu s bilo kojim od patentnih zahtjeva 1 do 9, naznačen time što se primjenjuje više doza, ili što se pripravak primjenjuje tjedno.
11. Pripravak namijenjen upotrebi u skladu s bilo kojim od patentnih zahtjeva 1 do 10, naznačen time što količina inhibira kašnjenje rasta, inhibira hepatospenomegaliju, inhibira kardiopulmonalnu bolest, ili inhibira displaziju kostura, ili bilo koju njihovu kombinaciju.
12. Pripravak namijenjen upotrebi u skladu s bilo kojim od patentnih zahtjeva 1 do 11, naznačen time što je rAAV rAAVrh10.
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 |
|---|---|
| HRP20220050T1 true HRP20220050T1 (hr) | 2022-10-14 |
Family
ID=57320277
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20220050TT HRP20220050T1 (hr) | 2015-05-15 | 2016-05-13 | Adenoasocirani virus, namijenjen terapijskom unosu u središnji živčani sustav |
Country Status (22)
| Country | Link |
|---|---|
| US (5) | US20180289839A1 (hr) |
| EP (2) | EP3294323B1 (hr) |
| JP (3) | JP2018515615A (hr) |
| KR (2) | KR102406615B1 (hr) |
| CN (1) | CN108367055A (hr) |
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Families Citing this family (25)
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| 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 |
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| 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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