TW202136514A - 重組cdkl5蛋白、基因療法及生產方法 - Google Patents

重組cdkl5蛋白、基因療法及生產方法 Download PDF

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TW202136514A
TW202136514A TW109137883A TW109137883A TW202136514A TW 202136514 A TW202136514 A TW 202136514A TW 109137883 A TW109137883 A TW 109137883A TW 109137883 A TW109137883 A TW 109137883A TW 202136514 A TW202136514 A TW 202136514A
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seq
cdkl5
polypeptide
fusion protein
sequence
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TW109137883A
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Chinese (zh)
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尚恩 克拉克
尚恩 蘇利文
希拉蕊 葛蕾
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美商阿米庫斯醫療股份有限公司
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TW109137883A 2019-10-30 2020-10-30 重組cdkl5蛋白、基因療法及生產方法 TW202136514A (zh)

Applications Claiming Priority (6)

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

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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=)
WO (1) WO2021087282A1 (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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EP2435562A1 (en) * 2009-05-26 2012-04-04 Biolex Therapeutics, Inc. Compositions and methods for production of aglycosylated plasminogen
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
SG10202000537RA (en) * 2014-02-28 2020-03-30 Univ Bologna Alma Mater Studiorum Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof
MX2017009336A (es) * 2015-01-16 2017-11-15 Voyager Therapeutics Inc Polinucleótidos dirigidos al sistema nervioso central.
US11446398B2 (en) * 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit systems
EP3475418A4 (en) * 2016-06-28 2020-03-04 Alma Mater Studiorum -Universita' di Bologna TATK-CDKL5 FUSION PROTEINS, COMPOSITIONS, FORMULATIONS AND THEIR USE
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
PT3568483T (pt) * 2017-01-10 2025-11-18 Childrens Medical Res Institute Polinucleótidos e vetores para a expressão de transgenes
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
JP2021505135A (ja) * 2017-11-30 2021-02-18 アミカス セラピューティックス インコーポレイテッド Cdkl5発現変異体及びcdkl5融合タンパク質
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
IT201900008877A1 (it) * 2019-06-13 2020-12-13 Univ Bologna Alma Mater Studiorum Nuovi costrutti per terapia genica

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KR20220106981A (ko) 2022-08-01
AU2020372988A1 (en) 2023-06-01
WO2021087282A1 (en) 2021-05-06
CA3156506A1 (en) 2021-05-06
JP2022554267A (ja) 2022-12-28
CN114901802A (zh) 2022-08-12
EP4073231A4 (en) 2024-06-05
EP4073231A1 (en) 2022-10-19
US20230043046A1 (en) 2023-02-09

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