HRP20230023T1 - Postupci liječenja infekcije hepatitisom b - Google Patents

Postupci liječenja infekcije hepatitisom b Download PDF

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HRP20230023T1
HRP20230023T1 HRP20230023TT HRP20230023T HRP20230023T1 HR P20230023 T1 HRP20230023 T1 HR P20230023T1 HR P20230023T T HRP20230023T T HR P20230023TT HR P20230023 T HRP20230023 T HR P20230023T HR P20230023 T1 HRP20230023 T1 HR P20230023T1
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preparation
oligonucleotide
positions
nucleotides
preparation according
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HRP20230023TT
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Martin KOSER
Marc Abrams
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Dicerna Pharmaceuticals, Inc.
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Application filed by Dicerna Pharmaceuticals, Inc. filed Critical Dicerna Pharmaceuticals, Inc.
Publication of HRP20230023T1 publication Critical patent/HRP20230023T1/hr

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Claims (15)

1. Oligonukleotid za smanjenje ekspresije površinskog antigena virusa hepatitisa B (HBsAg) mRNA, te oligonukleotid sadrži smisleni lanac koji tvori dupleks regiju s protusmislenim lancem, naznačen time što: se smisleni lanac sastoji od sekvence kako je navedeno u GACAAAAAUCCUCACAAUAAGCAGCCGAAAGGCUGC (SEQ ID NO:9) i sadrži 2'-fluoro modificirane nukleotide na položajima 3, 8-10, 12, 13, i 17, 2'-O-metil modificirane nukleotide na položajima 1, 2, 4-7, 11, 14-16, 18-26, i 31-36, i jednu fosforotioatnu internukleotidnu vezu između nukleotida na položajima 1 i 2, pri čemu je svaki od nukleotida iz -GAAA- sekvence na smislenom lancu konjugiran na jednovalentni GalNac dio, pri čemu -GAAA- sekvenca sadrži strukturu: [image] i protusmisleni lanac se sastoji od sekvence kako je navedeno u UUAUUGUGAGGAUUUUUGUCGG (SEQ ID NO: 6) i sadrži 2'-fluoro modificirane nukleotide na položajima 2, 3, 5, 7, 8, 10, 12, 14, 16, i 19, 2'-O-metil modificirane nukleotide na položajima 1, 4, 6, 9, 11, 13, 15, 17, 18, i 20-22, i pet fosforotioatnih internukleotidnih veza između nukleotida na položajima 1 i 2, između nukleotida na položajima 2 i 3, između nukleotida na položajima 3 i 4, između nukleotida na položajima 20 i 21, i između nukleotida na položajima 21 i 22, pri čemu 5'-nukleotid protusmislenog lanca ima sljedeću strukturu: [image]
2. Pripravak koji sadrži oligonukleotid prema zahtjevu 1 .
3. Pripravak prema zahtjevu 2, naznačen time što sadrži protuion.
4. Pripravak prema zahtjevu 2, naznačen time što sadrži Na+ protuione.
5. Pripravak prema bilo kojem od zahtjeva 2 do 4, naznačen time što sadrži farmaceutski prihvatljiv nosač.
6. Pripravak prema zahtjevu 5, naznačen time što je farmaceutski prihvatljiv nosač otapalo ili disperzijski medij koji sadrži vodu.
7. Pripravak prema bilo kojem od zahtjeva 2 do 6, naznačen time što sadrži pomoćnu tvar.
8. Pripravak prema bilo kojem od zahtjeva 2 do 7, naznačen time što sadrži otopinu soli s fosfatnim puferom.
9. In vitro postupak smanjenja ekspresije površinskog antigena (HBV) virusa hepatitisa B (HBV) u stanici, naznačen time što postupak uključuje isporuku u stanicu oligonukleotida prema zahtjevu 1, ili pripravka prema bilo kojem od zahtjeva 2 do 8.
10. Postupak prema zahtjevu 9, naznačen time što je stanica hepatocit.
11. Oligonukleotid prema zahtjevu 1 ili pripravak prema bilo kojem od zahtjeva 2 do 8, naznačen time što se koristi u postupku liječenja HBV infekcije kod subjekta.
12. Oligonukleotid ili pripravak za upotrebu prema zahtjevu 11, naznačen time što se oligonukleotid ili pripravak daje subjektu potkožno.
13. Oligonukleotid ili pripravak za upotrebu prema zahtjevu 11, naznačen time što se oligonukleotid ili pripravak daje subjektu intravenski.
14. Pripravak za upotrebu prema zahtjevu 13, naznačen time što sadrži farmaceutski prihvatljiv nosač, pri čemu je farmaceutski prihvatljiv nosač otapalo ili disperzijski medij koji sadrži vodu.
15. Oligonukleotid ili pripravak za upotrebu u skladu s bilo kojim od patentnih zahtjeva 11-14, naznačen time što se subjektu također daje učinkovita količina Entekavira.
HRP20230023TT 2017-10-20 2018-10-19 Postupci liječenja infekcije hepatitisom b HRP20230023T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762575358P 2017-10-20 2017-10-20
PCT/US2018/056801 WO2019079781A2 (en) 2017-10-20 2018-10-19 METHODS OF TREATING HEPATITIS B TYPE INFECTIONS
EP18868404.7A EP3684377B1 (en) 2017-10-20 2018-10-19 Methods for treating hepatitis b infection

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HRP20230023T1 true HRP20230023T1 (hr) 2023-03-17

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US (5) US10799524B2 (hr)
EP (2) EP4197544A1 (hr)
JP (2) JP7353276B2 (hr)
KR (1) KR20200083494A (hr)
CN (1) CN111372593B (hr)
AU (1) AU2018351649A1 (hr)
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