HRP20211407T1 - Genetska konstrukcija - Google Patents

Genetska konstrukcija Download PDF

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HRP20211407T1
HRP20211407T1 HRP20211407TT HRP20211407T HRP20211407T1 HR P20211407 T1 HRP20211407 T1 HR P20211407T1 HR P20211407T T HRP20211407T T HR P20211407TT HR P20211407 T HRP20211407 T HR P20211407T HR P20211407 T1 HRP20211407 T1 HR P20211407T1
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seq
sequence
fragment
variant
optionally
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HRP20211407TT
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Peter Widdowson
Keith Martin
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Quethera Limited
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Claims (15)

1. Rekombinantni adeno-asocirani vektor (rAAV) koji sadrži genetsku konstrukciju koji sadrži, u orijentaciji 5’ prema 3’, promotor funkcionalno povezan sa prvom kodirajućom sekvencom, koja kodira receptorsku tirozin kinazu B (TrkB), i drugom kodirajućom sekvencom, koja kodira agonist receptora TrkB, pri čemu je agonist zreli BDNF, gdje druga kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira signalni peptid za agonist receptora TrkB, tako da, kada se eksprimira, signalni peptid je konjugiran za N-terminalni kraj zrelog BDNF, i pri čemu genetska konstrukcija sadrži sekvencu razmaknice smještenu između prve i druge kodirajuće sekvence, gdje sekvenca razmaknice kodira peptidnu razmaknicu koja je konfigurirana tako da se probavlja i tako proizvede receptor i agonist TrkB kao posebne molekule.
2. Rekombinantni vektor prema patentnom zahtjevu 1, gdje genetska konstrukcija sadrži nukleotidnu sekvencu koja kodira Woodchuckov post-transkripcioni regulatorni element virusa hepatitisa (WHPE), pri čemu izborno WHPE sadrži sekvencu nukleinske kiseline suštinski kao što je prikazano u SEQ ID No: 57 ili 58, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 57 ili 58, i/ili gdje konstrukcija sadrži nukleotidnu sekvencu koja kodira poliA rep, pri čemu izborno poliA rep sadrži sekvencu nukleinske kiseline suštinski kao što je prikazano u SEQ ID No: 59, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 59.
3. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje promotor predstavlja promotor humanog sinapsina I (SYN I), pri čemu izborno promotor sadrži sekvencu nukleinske kiseline suštinski kao što je prikazano u SEQ ID No: 1, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 1, ili gdje promotor predstavlja CAG promotor, pri čemu izborno promotor sadrži sekvencu nukleinske kiseline suštinski kao što je prikazano u SEQ ID No: 2, 3 ili 48, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 2, 3, ili 48.
4. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje sekvenca razmaknice sadrži i kodira virusnu peptidnu sekvencu razmaknice, poželjnije virusnu 2A peptidnu sekvencu razmaknice.
5. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje peptidna sekvenca razmaknice sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u SEQ ID NO. 4, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 4, ili gdje sekvenca razmaknice sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID NO.5, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 5, ili gdje peptidna sekvenca razmaknice sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u SEQ ID NO. 6, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 6, ili gdje sekvenca razmaknice sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID NO. 7, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 7, ili gdje peptidna sekvenca razmaknice sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u SEQ ID NO. 8, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 8.
6. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje prva kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira humanu kanonsku izoformu TrkB, pri čemu kanonska izoforma TrkB sadrži aminokiselinsku sekvencu prikazanu u SEQ ID NO. 9, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 9, i/ili gdje prva kodirajuća sekvenca sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID NO. 10, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 10, i/ili gdje prva kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira izoformu 4 TrkB, pri čemu izborno izoforma 4 TrkB sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u SEQ ID NO. 11, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 11, i gdje izborno prva kodirajuća sekvenca sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID NO. 12, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 12.
7. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje prva kodirajuća sekvenca sadrži nukleotidnu sekvencu u skladu sa SEQ ID NO: 9, pri čemu je jedan ili više tirozinskih ostataka na položajima 516, 701, 705, 706 i/ili 816 u SEQ ID No: 9 modificirano u drugačiji aminokiselinski ostatak, gdje su izborno najmanje dva, tri ili četiri tirozinska ostatka na položajima 516, 701, 705, 706 i/ili 816 u SEQ ID No: 9 modificirani u drugačije aminokiselinske ostatke, pri čemu je izborno svih pet tirozinskih ostataka na položajima 516, 701, 705, 706 i/ili 816 u SEQ ID No: 9 modificirano u drugačije aminokiselinske ostatke, i pri čemu je izborno naveden ili svaki tirozinski ostatak modificiran u glutaminsku kiselinu.
8. Rekombinantni vektor prema patentnom zahtjevu 7, gdje modificirani oblik receptora TrkB sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u SEQ ID NO. 13, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 13, gdje izborno prva kodirajuća sekvenca sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID NO. 14, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 14.
9. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje druga kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira zreli BDNF, pri čemu izborno zreli BDNF sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u SEQ ID NO. 18, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 18, i gdje izborno druga kodirajuća sekvenca sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID NO. 19, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 19.
10. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje druga kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira signalni peptid za BDNF, pri čemu izborno nukleotidna sekvenca kodira kanonski signalni peptid za BDNF, gdje druga kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira signalni peptid koji sadrži aminokiselinsku sekvencu kao što je prikazano u SEQ ID NO. 20, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 20, ili gdje druga kodirajuća sekvenca sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID NO. 21, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No: 21.
11. Rekombinantni vektor prema bilo kojem prethodnom patentnom zahtjevu, gdje druga kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira peptid signalne sekvence suštinski kao što je prikazano u bilo kojoj od SEQ ID NO. 23, 25, 27 ili 29, ili gdje signalni peptid sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u bilo kojoj od SEQ ID NO. 22, 24, 26 ili 28, i/ili gdje druga kodirajuća sekvenca sadrži nukleotidnu sekvencu koja kodira peptid signalne sekvence suštinski kao što je prikazano u bilo kojoj od SEQ ID NO. 31, 33, 35, 37, 39, 41, 43, 45, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83, 85, 87, 89, 91, 93, 95, 97, 99, 101 ili 103; ili gdje signalni peptid sadrži aminokiselinsku sekvencu suštinski kao što je prikazano u bilo kojoj od SEQ ID NO. 30, 32, 34, 36, 38, 40, 42, 44, 60, 62, 64, 66, 68, 70, 72, 74, 76, 78, 80, 82, 84, 86, 88, 90, 92, 94, 96, 98, 100 ili 102, i/ili gdje konstrukcija sadrži nukleotidnu sekvencu suštinski kao što je prikazano u SEQ ID No: 107 ili 108, ili fragment ili varijantu sa najmanje 65% identičnosti sekvence sa SEQ ID No:107 ili 108 i/ili gdje je rAAV AAV-1, AAV-2, AAV-3A, AAV-3B, AAV-4, AAV-5, AAV-6, AAV-7, AAV-8, AAV-9, AAV-10 ili AAV-11, i gdje izborno rAAV predstavlja rAAV serotipa-2.
12. Rekombinantni vektor prema bilo kojem od patentnih zahtjeva 1-11, za uporabu kao lijek ili u terapiji.
13. Rekombinantni vektor prema bilo kojem od patentnih zahtjeva 1-11, za uporabu u liječenju, prevenciji ili ublažavanju poremećaja optičkog živca, ili za poticanje regeneracije i/ili preživljavanja živca, gdje je izborno poremećaj optičkog živca koji se liječi bilo koje patofiziološko stanje koje rezultira gubitkom RGC, kao što je trauma glave ili lica ili vaskularne povrede, na primjer djelomičan ili potpuni gubitak snabdjevanja krvlju očnih struktura ili regija mozga koje primaju impulse iz optičkog živca, ili se koristi kao podrška zamjeni RGC putem uvođenja netransformiranih ili transformiranih matičnih stanica u oko ili regije povezane sa vidom kod pacijenata, pri čemu je izborno poremećaj optičkog živca koji se liječi glaukom.
14. Farmaceutski pripravak koji sadrži rekombinantni vektor prema bilo kojem od patentnih zahtjeva 1-11, i farmaceutski prihvatljiv vehikul.
15. Postupak za pripremu farmaceutskog pripravka prema patentnom zahtjevu 14, pri čemu postupak obuhvaća dovođenje u kontakt rekombinantnog vektora prema bilo kojem od patentnih zahtjeva 1-11, sa farmaceutski prihvatljivim vehikulom.
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