KR20220140537A - Cdkl5 결핍 장애를 치료하기 위한 유전자 요법 - Google Patents

Cdkl5 결핍 장애를 치료하기 위한 유전자 요법 Download PDF

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KR20220140537A
KR20220140537A KR1020227029963A KR20227029963A KR20220140537A KR 20220140537 A KR20220140537 A KR 20220140537A KR 1020227029963 A KR1020227029963 A KR 1020227029963A KR 20227029963 A KR20227029963 A KR 20227029963A KR 20220140537 A KR20220140537 A KR 20220140537A
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raav
sequence
promoter
ser
cdkl5
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샤릴 린 파이피-마리시흐
매튜 스콧 풀러
마가렛 캐롤라인 라이트
로렐라이 이오아나 스토이카
스튜어트 크레이그
션 크리스토퍼 도허티
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울트라제닉스 파마수티컬 인코포레이티드
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11022—Cyclin-dependent kinase (2.7.11.22)

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020227029963A 2020-02-14 2021-02-11 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법 Pending KR20220140537A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062976483P 2020-02-14 2020-02-14
US62/976,483 2020-02-14
US202063090492P 2020-10-12 2020-10-12
US63/090,492 2020-10-12
PCT/US2021/017656 WO2021163322A1 (en) 2020-02-14 2021-02-11 Gene therapy for treating cdkl5 deficiency disorder

Publications (1)

Publication Number Publication Date
KR20220140537A true KR20220140537A (ko) 2022-10-18

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KR1020227029963A Pending KR20220140537A (ko) 2020-02-14 2021-02-11 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법

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US (1) US20230054144A1 (https=)
EP (1) EP4103724A1 (https=)
JP (1) JP2023514204A (https=)
KR (1) KR20220140537A (https=)
CN (1) CN115151648A (https=)
AU (1) AU2021218703A1 (https=)
BR (1) BR112022015921A2 (https=)
CA (1) CA3164714A1 (https=)
IL (1) IL294781A (https=)
MX (1) MX2022009982A (https=)
TW (1) TWI904135B (https=)
WO (1) WO2021163322A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021202532A1 (en) * 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
IL297635A (en) * 2020-04-27 2022-12-01 Univ Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
WO2022225978A1 (en) * 2021-04-21 2022-10-27 The Regents Of The University Of California Use of a split dcas fusion protein system for epigenetic editing
EP4387641A4 (en) * 2021-08-18 2025-07-09 Ultragenyx Pharmaceutical Inc RECOMBINANT AAV FORMULATIONS
EP4349365A1 (en) * 2022-10-07 2024-04-10 Hansa Biopharma AB Co-treatment for gene therapy
CN116350801A (zh) 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
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
WO2024238958A1 (en) 2023-05-18 2024-11-21 Ultragenyx Pharmaceutical Inc. Formulations comprising recombinant aav and methods of administering the same
WO2025122548A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2140408B (en) 1982-12-23 1987-03-18 Suwa Seikosha Kk Process for producing quartz glass
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
DE602004030327D1 (de) 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
SI3235827T1 (sl) 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
ES2685611T3 (es) 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
BR112017005892A2 (pt) 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
GB201502305D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
GB201502306D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
ES2877754T3 (es) 2016-02-04 2021-11-17 Genovis Ab Nuevas proteasas estreptocócicas
US10179176B2 (en) 2016-02-16 2019-01-15 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
EP3827812B1 (en) 2016-07-29 2025-10-29 The Regents of the University of California Adeno-associated virus virions with variant capsid and methods of use thereof
AU2018224044B2 (en) 2017-02-21 2024-01-25 The Uab Research Foundation Modified AAV capsid proteins and uses thereof
CA3056410A1 (en) * 2017-03-15 2018-09-20 Ovid Therapeutics, Inc. Use of designer receptors exclusively activated by designer drugs in the treatment of seizure disorders
CA3063464A1 (en) * 2017-05-19 2018-11-22 Encoded Therapeutics, Inc. High activity regulatory elements
WO2018222503A1 (en) 2017-05-31 2018-12-06 The Regents Of The University Of California Adeno-associated virus with variant capsid and methods of use thereof
CA3065946A1 (en) 2017-06-05 2018-12-13 Research Institute At Nationwide Children's Hospital Enhanced modified viral capsid proteins
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
IL276859B2 (en) * 2018-02-27 2025-12-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
AU2020215682B2 (en) 2019-01-28 2025-12-18 Duke University Compositions and methods for evading humoral immunity
US12544437B2 (en) * 2019-10-04 2026-02-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant AAV

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BR112022015921A2 (pt) 2022-10-04
TWI904135B (zh) 2025-11-11
AU2021218703A1 (en) 2022-07-28
US20230054144A1 (en) 2023-02-23
JP2023514204A (ja) 2023-04-05
MX2022009982A (es) 2022-09-12
TW202144577A (zh) 2021-12-01
WO2021163322A1 (en) 2021-08-19
CA3164714A1 (en) 2021-08-19
CN115151648A (zh) 2022-10-04
EP4103724A1 (en) 2022-12-21
IL294781A (en) 2022-09-01

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