JP2020505317A - 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置 - Google Patents

臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置 Download PDF

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JP2020505317A
JP2020505317A JP2019522742A JP2019522742A JP2020505317A JP 2020505317 A JP2020505317 A JP 2020505317A JP 2019522742 A JP2019522742 A JP 2019522742A JP 2019522742 A JP2019522742 A JP 2019522742A JP 2020505317 A JP2020505317 A JP 2020505317A
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galc
nucleic acid
acid molecule
vector
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JP2020505317A5 (https=
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マリア ルイーザ エスコラー，
マリア ルイーザ エスコラー，
ポール サボルクス，
ポール サボルクス，
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University of Pittsburgh
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University of Pittsburgh
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    • A—HUMAN NECESSITIES
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/48—Reproductive organs
    • A61K35/51—Umbilical cord; Umbilical cord blood; Umbilical stem cells
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61K31/155—Amidines (), e.g. guanidine (H2N—C(=NH)—NH2), isourea (N=C(OH)—NH2), isothiourea (—N=C(SH)—NH2)
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/16—Amides, e.g. hydroxamic acids
    • A61K31/17—Amides, e.g. hydroxamic acids having the group >N—C(O)—N< or >N—C(S)—N<, e.g. urea, thiourea, carmustine
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
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    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
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    • A61K39/39558—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against tumor tissues, cells, antigens
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • 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)
    • C12Y302/01046—Galactosylceramidase (3.2.1.46)
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JP2019522742A 2017-01-20 2018-01-19 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置 Withdrawn JP2020505317A (ja)

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JP2023016007A JP2023041950A (ja) 2017-01-20 2023-02-06 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

Applications Claiming Priority (3)

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US201762448433P 2017-01-20 2017-01-20
US62/448,433 2017-01-20
PCT/US2018/014370 WO2018136710A1 (en) 2017-01-20 2018-01-19 Treatment of krabbe disease with umbilical cord blood transplantion (ucbt) and increased galactocerebrosidase (galc) expression

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JP2023016007A Division JP2023041950A (ja) 2017-01-20 2023-02-06 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

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JP2020505317A5 JP2020505317A5 (https=) 2021-02-25

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JP2023016007A Pending JP2023041950A (ja) 2017-01-20 2023-02-06 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置

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US (1) US12599636B2 (https=)
EP (1) EP3571298B1 (https=)
JP (2) JP2020505317A (https=)
KR (1) KR20190106990A (https=)
CN (2) CN118078860A (https=)
AU (1) AU2018210326B9 (https=)
CA (1) CA3042467A1 (https=)
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WO2020132385A1 (en) * 2018-12-21 2020-06-25 The University Of North Carolina At Chapel Hill Optimized galc genes and expression cassettes and their use
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US20230210911A1 (en) * 2020-06-26 2023-07-06 University Of Pittsburgh- Of The Commonwealth System Of Higher Education Methods and materials for treating non-malignant disorders or diseases with cord blood

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023526311A (ja) * 2020-05-12 2023-06-21 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クラッベ病の治療に有用な組成物
JP7832898B2 (ja) 2020-05-12 2026-03-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クラッベ病の治療に有用な組成物

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AU2018210326B2 (en) 2024-03-07
AU2018210326A1 (en) 2019-05-23
EP3571298A4 (en) 2020-10-21
IL266287A (en) 2019-06-30
EP3571298B1 (en) 2026-03-04
CN110168085A (zh) 2019-08-23
JP2023041950A (ja) 2023-03-24
KR20190106990A (ko) 2019-09-18
AU2018210326B9 (en) 2024-04-04
CA3042467A1 (en) 2018-07-26
US12599636B2 (en) 2026-04-14
WO2018136710A1 (en) 2018-07-26
EP3571298A1 (en) 2019-11-27
CN118078860A (zh) 2024-05-28
US20190336540A1 (en) 2019-11-07

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