HRP20211197T1 - Rekombinantni glikoproteini i njihova uporaba - Google Patents

Rekombinantni glikoproteini i njihova uporaba Download PDF

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HRP20211197T1
HRP20211197T1 HRP20211197TT HRP20211197T HRP20211197T1 HR P20211197 T1 HRP20211197 T1 HR P20211197T1 HR P20211197T T HRP20211197T T HR P20211197TT HR P20211197 T HRP20211197 T HR P20211197T HR P20211197 T1 HRP20211197 T1 HR P20211197T1
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oligosaccharides
percentage
total
peak
rhaga
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HRP20211197TT
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English (en)
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Karen Lee
Christopher HWANG
Christine Demaria
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Genzyme Corporation
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Claims (14)

1. Rekombinantni humani protein α-galaktozidaze-A (rhAGA) koji ima: postotak ukupnih N-vezanih oligosaharida u vrhu 11 koji su bis-manoza-6-fosforilirani oligosaharidi koji je veći od 9%; postotak ukupnih N-vezanih oligosaharida u vrhu 3 koji su monosijalilirani, minus fukozni oligosaharidi koji je između 0.1% i 1.6%; postotak ukupnih N-vezanih oligosaharida u vrhu 4 koji su bisijalilirani oligosaharidi koji sadrže fukozu koji je veći od 13.5 %; postotak ukupnih N-vezanih oligosaharida u vrhu 6' koji su triantenarni, trisijalilirani oligosaharidi oblika 2 koji je veći od 2.0%; postotak ukupnih N-vezanih oligosaharida u vrhu 1 koji su neutralno nabijeni oligosaharidi koji je između 0.1 % i 3 .9%; postotak ukupnih N-vezanih oligosaharida u vrhu 2 koji su monosijalilirani oligosaharidi koji sadrže fukozu koji je između 0.1% i 3.0%; postotak ukupnih N-vezanih oligosaharida u vrhu 5 koji su bisijalilirani oligosaharidi koji je između 0.1 % i 5.3%; postotak ukupnih N-vezanih oligosaharida u vrhu 6 koji su triantenarni, trisijalilirani oligosaharidi oblika 1 koji je između 0.1% i 9.0%; postotak ukupnih N-vezanih oligosaharida u vrhu 7 koji su manoza-6-fosforilirani oligosaharidi koji je između 1% i 7.0%; postotak ukupnih N-vezanih oligosaharida u vrhu 8 koji su monofosforilirani oligosaharidi koji je veći od 14.8%; postotak ukupnih N-vezanih oligosaharida u vrhu 9 koji su tetrasijalilirani oligosaharidi koji je veći od 4.9%; i postotak ukupnih N-vezanih oligosaharida u vrhu 10 koji su monosijalilirani i monofosforilirani oligosaharidi koji je veći od 8.2%; pri čemu se vrhovi određuju profiliranjem N-vezanih oligosaharida obilježenih antranilnom kiselinom.
2. Farmaceutski pripravak naznačen time što sadrži protein rhAGA prema patentnom zahtjevu 1, i farmaceutski prihvatljiv nosač.
3. Farmaceutski pripravak prema patentnom zahtjevu 2, naznačen time što je pripravak formuliran za intravensku, intraarterijsku, intramuskularnu, intradermalnu, potkožnu ili intraperitonealnu primjenu
4. Farmaceutski pripravak prema patentnom zahtjevu 3, naznačen time što farmaceutski pripravak sadrži koncentraciju rhAGA od 4 mg/mL do 6 mg/mL.
5. Farmaceutski pripravak prema patentnom zahtjevu 4, naznačen time što farmaceutski pripravak sadrži koncentraciju rhAGA od 5 mg/mL.
6. Farmaceutski pripravak prema patentnom zahtjevu 2, naznačen time što je farmaceutski pripravak sterilni, liofilizirani prah.
7. Farmaceutski pripravak prema patentnom zahtjevu 6, naznačen time što je farmaceutski prihvatljiv nosač jedno ili više sredstava odabranih iz skupine koju čine: manitol, natrijev fosfat monobazni monohidrat i natrijev fosfat dvobazni heptahidrat.
8. Protein rhAGA prema patentnom zahtjevu 1 naznačen time što je za uporabu u liječenju Fabryjeve bolesti.
9. Protein rhAGA za uporabu prema patentnom zahtjevu 8, naznačen time što je navedena uporaba u postupku za povećanje razine proteina α-galaktozidaze-A u lizosomu u stanici sisavca kod subjekta, pri čemu je stanica sisavca u kontaktu s proteinom rhAGA.
10. Protein rhAGA za uporabu prema patentnom zahtjevu 8, naznačen time što je navedena upotreba u postupku za smanjenje razine globotriaozilceramida u serumu subjekta.
11. Protein rhAGA za uporabu prema bilo kojem od patentnih zahtjeva 8-10, naznačen time što se protein rhAGA primjenjuje intravenozno.
12. Protein rhAGA za uporabu prema patentnom zahtjevu 11, naznačen time što se protein rhAGA primjenjuje u dozi od 0.5 mg/kg tjelesne težine do 2.0 mg/kg tjelesne težine.
13. Protein rhAGA za uporabu prema patentnom zahtjevu 12, naznačen time što se protein rhAGA primjenjuje u dozi od 1.0 mg/kg tjelesne težine.
14. Protein rhAGA za uporabu prema bilo kojem od patentnih zahtjeva 8-10, naznačen time što se primjenjuju dvije ili više doza proteina rhAGA, pri čemu se najmanje dvije od dvije ili više doza proteina rhAGA primjenjuju u razmaku od dva tjedna.
HRP20211197TT 2013-10-23 2021-07-26 Rekombinantni glikoproteini i njihova uporaba HRP20211197T1 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361894879P 2013-10-23 2013-10-23
US201361901942P 2013-11-08 2013-11-08
EP14796346.6A EP3060660B1 (en) 2013-10-23 2014-10-22 Recombinant glycoproteins and uses thereof
PCT/US2014/061789 WO2015061464A2 (en) 2013-10-23 2014-10-22 Recombinant glycoproteins and uses thereof

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HRP20211197T1 true HRP20211197T1 (hr) 2021-11-12

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EP (2) EP3060660B1 (hr)
JP (2) JP6837334B2 (hr)
KR (2) KR102298080B1 (hr)
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AU (2) AU2014340152A1 (hr)
BR (1) BR112016008086A2 (hr)
CA (1) CA2927852A1 (hr)
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CR (1) CR20160225A (hr)
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DO (1) DOP2016000079A (hr)
EA (1) EA034515B1 (hr)
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HU (1) HUE055236T2 (hr)
IL (2) IL245255B (hr)
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CN114606217A (zh) 2022-06-10
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IL245255B (en) 2020-02-27
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KR102479886B1 (ko) 2022-12-20
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CR20160225A (es) 2016-06-24
EP3060660A2 (en) 2016-08-31
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PL3060660T3 (pl) 2021-12-06
JP2020089379A (ja) 2020-06-11
WO2015061464A3 (en) 2015-09-03
CA2927852A1 (en) 2015-04-30

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