JP2022554267A5 - - Google Patents

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Publication number
JP2022554267A5
JP2022554267A5 JP2022525193A JP2022525193A JP2022554267A5 JP 2022554267 A5 JP2022554267 A5 JP 2022554267A5 JP 2022525193 A JP2022525193 A JP 2022525193A JP 2022525193 A JP2022525193 A JP 2022525193A JP 2022554267 A5 JP2022554267 A5 JP 2022554267A5
Authority
JP
Japan
Prior art keywords
seq
composition according
sequence identity
polypeptide
polynucleotide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022525193A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022554267A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/058247 external-priority patent/WO2021087282A1/en
Publication of JP2022554267A publication Critical patent/JP2022554267A/ja
Publication of JP2022554267A5 publication Critical patent/JP2022554267A5/ja
Pending legal-status Critical Current

Links

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)

Applications Claiming Priority (7)

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

Publications (2)

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

Family

ID=75716480

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022525193A Pending JP2022554267A (ja) 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

Country Status (9)

Country Link
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=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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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