JP2022554267A - 組換えｃｄｋｌ５タンパク質、遺伝子療法、及び製造方法 - Google Patents

組換えｃｄｋｌ５タンパク質、遺伝子療法、及び製造方法 Download PDF

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JP2022554267A
JP2022554267A JP2022525193A JP2022525193A JP2022554267A JP 2022554267 A JP2022554267 A JP 2022554267A JP 2022525193 A JP2022525193 A JP 2022525193A JP 2022525193 A JP2022525193 A JP 2022525193A JP 2022554267 A JP2022554267 A JP 2022554267A
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cdkl5
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fusion protein
sequence
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ショーン クラーク，
ショーン サリバン，
ヒラリー グレイ，
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アミカス セラピューティックス インコーポレイテッド
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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    • C12Y—ENZYMES
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
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    • C—CHEMISTRY; METALLURGY
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    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
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    • C07K2319/50—Fusion polypeptide containing protease site
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2022525193A 2019-10-30 2020-10-30 組換えｃｄｋｌ５タンパク質、遺伝子療法、及び製造方法 Pending JP2022554267A (ja)

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Application Number Priority Date Filing Date Title
US201962928140P 2019-10-30 2019-10-30
US201962928134P 2019-10-30 2019-10-30
US62/928,140 2019-10-30
US62/928,134 2019-10-30
US202063090520P 2020-10-12 2020-10-12
US63/090,520 2020-10-12
PCT/US2020/058247 WO2021087282A1 (en) 2019-10-30 2020-10-30 Recombinant cdkl5 proteins, gene therapy and production methods

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JP2022554267A true JP2022554267A (ja) 2022-12-28
JP2022554267A5 JP2022554267A5 (https=) 2023-11-06

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US (1) US20230043046A1 (https=)
EP (1) EP4073231A4 (https=)
JP (1) JP2022554267A (https=)
KR (1) KR20220106981A (https=)
CN (1) CN114901802A (https=)
AU (1) AU2020372988A1 (https=)
CA (1) CA3156506A1 (https=)
TW (1) TW202136514A (https=)
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IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
MX2024004723A (es) * 2021-10-18 2024-07-19 Univ Pennsylvania Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd).
WO2023077143A1 (en) * 2021-11-01 2023-05-04 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
US20250369012A1 (en) * 2022-06-21 2025-12-04 Skyline Therapeutics Limited Recombinant aav for the gene therapy of sma disease
CN116377050A (zh) * 2022-12-16 2023-07-04 湖南家辉生物技术有限公司 一种发育性癫痫性脑病2型致病基因cdkl5突变位点的应用及其检测试剂和应用
WO2025050032A1 (en) * 2023-08-31 2025-03-06 Ultragenyx Pharmaceutical Inc. Methods of producing cdkl5 proteins and uses of the same
CN118638212B (zh) * 2024-06-18 2025-07-04 河北绶肽生物科技有限公司 一种烟草表达的重组人源ⅲ型胶原蛋白及其编码基因和制备方法

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JP2017507655A (ja) * 2014-02-28 2017-03-23 アルマ マータ ステューディオラム — ユニバーシタ ディ ボローニャ ＴＡＴｋ−ＣＤＫＬ５融合タンパク質、組成物、製剤、及びそれらの使用
WO2018126112A1 (en) * 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
WO2018129586A1 (en) * 2017-01-10 2018-07-19 Children's Medical Research Institute Polynucleotides and vectors for the expression of transgenes
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
WO2019108924A2 (en) * 2017-11-30 2019-06-06 Amicus Therapeutics, Inc. Cdkl5 expression variants and cdkl5 fusion proteins
JP2019522989A (ja) * 2016-06-28 2019-08-22 アルマ マータ ステューディオラム — ユニバーシタ ディ ボローニャ ＴＡＴκ−ＣＤＫＬ５融合タンパク質、組成物、製剤、及びそれらの使用

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ES2829199T3 (es) * 2010-11-22 2021-05-31 Amicus Therapeutics Inc Nuevas secuencias de señalización para mejorar la expresión y segregación de proteínas de enzimas recombinantes y otras proteínas
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
MX2017009336A (es) * 2015-01-16 2017-11-15 Voyager Therapeutics Inc Polinucleótidos dirigidos al sistema nervioso central.
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JP2017507655A (ja) * 2014-02-28 2017-03-23 アルマ マータ ステューディオラム — ユニバーシタ ディ ボローニャ ＴＡＴｋ−ＣＤＫＬ５融合タンパク質、組成物、製剤、及びそれらの使用
JP2019522989A (ja) * 2016-06-28 2019-08-22 アルマ マータ ステューディオラム — ユニバーシタ ディ ボローニャ ＴＡＴκ−ＣＤＫＬ５融合タンパク質、組成物、製剤、及びそれらの使用
WO2018126112A1 (en) * 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
WO2018129586A1 (en) * 2017-01-10 2018-07-19 Children's Medical Research Institute Polynucleotides and vectors for the expression of transgenes
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
WO2019108924A2 (en) * 2017-11-30 2019-06-06 Amicus Therapeutics, Inc. Cdkl5 expression variants and cdkl5 fusion proteins

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KR20220106981A (ko) 2022-08-01
AU2020372988A1 (en) 2023-06-01
WO2021087282A1 (en) 2021-05-06
CA3156506A1 (en) 2021-05-06
CN114901802A (zh) 2022-08-12
EP4073231A4 (en) 2024-06-05
EP4073231A1 (en) 2022-10-19
US20230043046A1 (en) 2023-02-09
TW202136514A (zh) 2021-10-01

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