HRP20220036T1 - Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe - Google Patents
Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe Download PDFInfo
- Publication number
- HRP20220036T1 HRP20220036T1 HRP20220036TT HRP20220036T HRP20220036T1 HR P20220036 T1 HRP20220036 T1 HR P20220036T1 HR P20220036T T HRP20220036T T HR P20220036TT HR P20220036 T HRP20220036 T HR P20220036T HR P20220036 T1 HRP20220036 T1 HR P20220036T1
- Authority
- HR
- Croatia
- Prior art keywords
- raav
- virion
- polypeptide
- use according
- capsid protein
- Prior art date
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- 210000002845 virion Anatomy 0.000 title claims 19
- 241000702421 Dependoparvovirus Species 0.000 title claims 6
- 210000000234 capsid Anatomy 0.000 title 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 14
- 229920001184 polypeptide Polymers 0.000 claims 13
- 102000004196 processed proteins & peptides Human genes 0.000 claims 13
- 108090000565 Capsid Proteins Proteins 0.000 claims 9
- 102100023321 Ceruloplasmin Human genes 0.000 claims 9
- 239000000825 pharmaceutical preparation Substances 0.000 claims 8
- 108090000623 proteins and genes Proteins 0.000 claims 7
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 150000001413 amino acids Chemical group 0.000 claims 4
- 208000030533 eye disease Diseases 0.000 claims 4
- 230000037431 insertion Effects 0.000 claims 4
- 238000003780 insertion Methods 0.000 claims 4
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 claims 3
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- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 claims 3
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 2
- 108091005462 Cation channels Proteins 0.000 claims 2
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
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- 210000004027 cell Anatomy 0.000 claims 2
- 230000004927 fusion Effects 0.000 claims 2
- 208000002780 macular degeneration Diseases 0.000 claims 2
- 210000000964 retinal cone photoreceptor cell Anatomy 0.000 claims 2
- 230000002207 retinal effect Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- ZOOGRGPOEVQQDX-UUOKFMHZSA-N 3',5'-cyclic GMP Chemical compound C([C@H]1O2)OP(O)(=O)O[C@H]1[C@@H](O)[C@@H]2N1C(N=C(NC2=O)N)=C2N=C1 ZOOGRGPOEVQQDX-UUOKFMHZSA-N 0.000 claims 1
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- 206010012688 Diabetic retinal oedema Diseases 0.000 claims 1
- 206010012689 Diabetic retinopathy Diseases 0.000 claims 1
- 102400001368 Epidermal growth factor Human genes 0.000 claims 1
- 101800003838 Epidermal growth factor Proteins 0.000 claims 1
- 108090000379 Fibroblast growth factor 2 Proteins 0.000 claims 1
- 102000003974 Fibroblast growth factor 2 Human genes 0.000 claims 1
- 208000010412 Glaucoma Diseases 0.000 claims 1
- 102000034615 Glial cell line-derived neurotrophic factor Human genes 0.000 claims 1
- 108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 claims 1
- 102000003693 Hedgehog Proteins Human genes 0.000 claims 1
- 108090000031 Hedgehog Proteins Proteins 0.000 claims 1
- 101000771083 Homo sapiens Cyclic nucleotide-gated cation channel beta-3 Proteins 0.000 claims 1
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- 102100040375 Peripherin-2 Human genes 0.000 claims 1
- 101710135995 Peripherin-2 Proteins 0.000 claims 1
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- 102000004590 Peripherins Human genes 0.000 claims 1
- 102100031176 Retinoid isomerohydrolase Human genes 0.000 claims 1
- 102100039507 Retinoschisin Human genes 0.000 claims 1
- 101710111169 Retinoschisin Proteins 0.000 claims 1
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- 108700031544 X-Linked Inhibitor of Apoptosis Proteins 0.000 claims 1
- 102000050257 X-Linked Inhibitor of Apoptosis Human genes 0.000 claims 1
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- 239000003900 neurotrophic factor Substances 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
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- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 210000005047 peripherin Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 108020003175 receptors Proteins 0.000 claims 1
- 102000005962 receptors Human genes 0.000 claims 1
- 201000007714 retinoschisis Diseases 0.000 claims 1
- VBEQCZHXXJYVRD-GACYYNSASA-N uroanthelone Chemical compound C([C@@H](C(=O)N[C@H](C(=O)N[C@@H](CS)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CS)C(=O)N[C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](CO)C(=O)NCC(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CS)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(O)=O)C(C)C)[C@@H](C)O)NC(=O)[C@H](CO)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CO)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@@H](NC(=O)[C@H](CC=1NC=NC=1)NC(=O)[C@H](CCSC)NC(=O)[C@H](CS)NC(=O)[C@@H](NC(=O)CNC(=O)CNC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CS)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)CNC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@H](CO)NC(=O)[C@H]1N(CCC1)C(=O)[C@H](CS)NC(=O)CNC(=O)[C@H]1N(CCC1)C(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@@H](N)CC(N)=O)C(C)C)[C@@H](C)CC)C1=CC=C(O)C=C1 VBEQCZHXXJYVRD-GACYYNSASA-N 0.000 claims 1
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Claims (13)
1. Virion rekombinantnog adeno-povezanog virusa (rAAV) ili farmaceutski pripravak koji sadrži navedeni virion, naznačen time što je za upotrebu u postupku liječenja očne bolesti kod ljudskog ili neljudskog primata kojem je to potrebno, pri čemu pripravak nadalje sadrži farmaceutski prihvatljivu pomoćnu tvar, te pri čemu virion rekombinantnog adeno-povezanog virusa (rAAV) sadrži:
a) varijantu AAV2 kapsidnog proteina, pri čemu varijanta AAV kapsidnog proteina sadrži umetanje peptida u GH petlju kapsidnog proteina u odnosu na odgovarajući roditeljski AAV kapsidni protein, pri čemu umetanje sadrži aminokiselinsku sekvencu LALGETTRPA (SEQ ID NO:45); i
b) heterolognu nukleinsku kiselinu koja sadrži nukleotidnu sekvencu koja kodira genski proizvod;
pri čemu varijanta AAV2 kapsidnog proteina daje povećanu infektivnost stanice mrežnice pomoću viriona rAAV, u usporedbi s infektivnošću stanice mrežnice pomoću viriona AAV koji sadrži divlji tip AAV2 kapsidnog proteina, te
pri čemu je navedeno liječenje intravitrealnom injekcijom viriona rAAV ili farmaceutskog pripravka.
2. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 1, naznačen time što je mjesto umetanja unutar aminokiselina 570-611 AAV2 kapsidnog proteina navedenog u SEQ ID NO:1.
3. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 1, naznačen time što je mjesto umetanja unutar aminokiselina 587-588 AAV2 kapsidnog proteina navedenog u SEQ ID NO:1.
4. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 3, naznačen time što se umetanje sastoji od aminokiselinske sekvence LALGETTRPA (SEQ ID NO:45) tako da AAV2 kapsidni protein sadrži sekvencu NLALGETTRPAR.
5. Virion rAAV ili farmaceutski pripravak za upotrebu prema bilo kojem od patentnih zahtjeva 1-4, naznačen time što je očna bolest glaukom, retinitis pigmentosa, makularna degeneracija, retinoshiza, Leberova urođena amauroza, dijabetička retinopatija, akromotopsija ili sljepoća za boje.
6. Virion rAAV ili farmaceutski pripravak za upotrebu prema bilo kojem od patentnih zahtjeva 1-4, naznačen time što je genski proizvod interferirajuća RNA, aptamer ili polipeptid.
7. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 6, naznačen time što je genski proizvod polipeptid odabran iz skupine koju čine: neurotrofni faktor izveden iz glija, faktor rasta fibroblasta 2, neurturin, cilijarni neurotrofni faktor, faktor rasta živaca, neurotrofni faktor izveden iz mozga, epidermalni faktor rasta, rodopsin, X-vezani inhibitor apoptoze, retinoshizin, RPE65, retinitis pigmentosa GTPaza-interagirajući protein-1, periferin, periferin-2, rodopsin, i Sonic hedgehog.
8. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 6, naznačen time što je genski proizvod polipeptid odabran od receptora topljivog vaskularnog endotelnog faktora rasta (VEGF), antitijela koje veže VEGF, i Fc fuzijskog polipeptida koji sadrži topljivi Fit polipeptid.
9. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 6, naznačen time što je genski proizvod polipeptid, te pri čemu je polipeptid Fc fuzijski polipeptid koji sadrži topljivi Flt polipeptid.
10. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 6, naznačen time što je genski proizvod polipeptid, te pri čemu je polipeptid konusni fotoreceptor alfa podjedinice kationskog kanala s upravljanjem cGMP-a (CNGA3) ili konusni fotoreceptor beta podjedinice kationskog kanala s upravljanjem cGMP-a (CNGB3).
11. Virion rAAV ili farmaceutski pripravak za upotrebu prema patentnom zahtjevu 6, naznačen time što je genski proizvod polipeptid, te pri čemu je polipeptid L-opsin, M-opsin ili S-opsin.
12. Virion rAAV ili farmaceutski pripravak za upotrebu prema bilo kojem od patentnih zahtjeva 1-4 ili 6-11, naznačen time što je očna bolest eksudativna makularna degeneracija povezana sa starenjem.
13. Virion rAAV ili farmaceutski pripravak za upotrebu prema bilo kojem od patentnih zahtjeva 1-4 ili 6-11, naznačen time što je očna bolest dijabetički makularni edem.
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