JP2022513318A5 - - Google Patents

Info

Publication number
JP2022513318A5
JP2022513318A5 JP2021542078A JP2021542078A JP2022513318A5 JP 2022513318 A5 JP2022513318 A5 JP 2022513318A5 JP 2021542078 A JP2021542078 A JP 2021542078A JP 2021542078 A JP2021542078 A JP 2021542078A JP 2022513318 A5 JP2022513318 A5 JP 2022513318A5
Authority
JP
Japan
Prior art keywords
sequence
raav
pccb
pcca
item
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
JP2021542078A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022513318A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/054003 external-priority patent/WO2020072451A1/en
Publication of JP2022513318A publication Critical patent/JP2022513318A/ja
Publication of JP2022513318A5 publication Critical patent/JP2022513318A5/ja
Pending legal-status Critical Current

Links

JP2021542078A 2018-10-01 2019-10-01 プロピオン酸血症を処置するための遺伝子治療 Pending JP2022513318A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862739471P 2018-10-01 2018-10-01
US62/739,471 2018-10-01
PCT/US2019/054003 WO2020072451A1 (en) 2018-10-01 2019-10-01 Gene therapy for treating propionic acidemia

Publications (2)

Publication Number Publication Date
JP2022513318A JP2022513318A (ja) 2022-02-07
JP2022513318A5 true JP2022513318A5 (enExample) 2022-10-12

Family

ID=70055045

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021542078A Pending JP2022513318A (ja) 2018-10-01 2019-10-01 プロピオン酸血症を処置するための遺伝子治療

Country Status (11)

Country Link
US (2) US12472268B2 (enExample)
EP (1) EP3861131A4 (enExample)
JP (1) JP2022513318A (enExample)
KR (1) KR20210082460A (enExample)
CN (1) CN112955557A (enExample)
AR (1) AR116569A1 (enExample)
AU (1) AU2019354296A1 (enExample)
BR (1) BR112021006052A2 (enExample)
CA (1) CA3113975A1 (enExample)
TW (1) TW202028472A (enExample)
WO (1) WO2020072451A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020264353A1 (en) * 2019-06-27 2020-12-30 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha
EP4045640A1 (en) * 2019-10-15 2022-08-24 The United States of America, as represented by the Secretary, Department of Health and Human Services Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase beta
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2022198079A1 (en) * 2021-03-19 2022-09-22 Mayo Foundation For Medical Education And Research Methods and materials for treating propionic acidemia
WO2023130003A2 (en) * 2021-12-29 2023-07-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Improved gene therapy constructs for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha
CN118166004B (zh) * 2024-05-14 2024-12-03 北京剂泰医药科技有限公司 用于编码人PCCA或PCCB蛋白的mRNA及其用途

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) * 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
SI1633772T1 (sl) 2003-06-19 2016-06-30 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
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2675484B1 (en) 2011-02-14 2018-05-30 The Children's Hospital of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
NZ701693A (en) 2012-04-18 2017-02-24 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
US20140303901A1 (en) * 2013-04-08 2014-10-09 Ilan Sadeh Method and system for predicting a disease
US10022372B2 (en) * 2013-04-19 2018-07-17 Thomas Jefferson University Caveolin-1 related methods for treating glioblastoma with temozolomide
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
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
WO2016179138A1 (en) 2015-05-03 2016-11-10 The Regents Of The University Of Colorado Propionyl-coa carboxylase compositions and uses thereof
BR112017024514A2 (pt) 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
MY189674A (en) * 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
JP6947739B2 (ja) 2016-02-16 2021-10-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
WO2017173043A1 (en) * 2016-03-30 2017-10-05 Spark Therapeutics, Inc. Cell line for recombinant protein and/or viral vector production
SG11201808812RA (en) 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
US11191847B2 (en) * 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
WO2017192761A1 (en) * 2016-05-03 2017-11-09 The Regents Of The University Of Colorado, A Body Corporate Propionyl-coa carboxylase compositions and uses thereof
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
KR102738752B1 (ko) 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
BR112019013245A2 (pt) 2016-12-30 2020-02-11 The Trustees Of The University Of Pennsylvania Terapia gênica para tratamento da doença de wilson
WO2018144709A2 (en) 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
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
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
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia

Similar Documents

Publication Publication Date Title
JP2022513318A5 (enExample)
JP2022516283A5 (enExample)
US11896652B2 (en) Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
FI3906066T3 (fi) Geeniterapiakonstrukteja wilsonin taudin hoitamiseksi
JP2023514204A5 (enExample)
US11939590B1 (en) AAV vector compositions and methods for gene transfer to cells, organs and tissues
JP2022500066A5 (enExample)
IL299325B1 (en) Variants of acid alpha-glucosidase and their uses
JP7654562B2 (ja) 線維芽細胞増殖因子23関連低リン血症性疾患の遺伝子療法
JP2020503265A5 (enExample)
JP2023520402A5 (enExample)
JP2022533448A5 (enExample)
JP2024056832A5 (enExample)
JP2023534293A5 (enExample)
JP2021533799A (ja) 第ix因子をコードするヌクレオチド
JP2023543125A (ja) Glp-1受容体アゴニスト融合物をコードするウイルスベクター及び代謝性疾患の治療におけるその使用
JP2021511020A5 (enExample)
JP2023542241A5 (enExample)
US8063022B1 (en) Methods for preventing formation of inhibitory antibodies in the setting of gene therapy
JP2025503868A5 (enExample)
JP2024528997A (ja) 放射線誘発性唾液機能低下を予防するためのaqp1遺伝子療法
JP2023536067A5 (enExample)
JP2022514271A5 (enExample)
JP2021533098A5 (enExample)
JP7796769B2 (ja) 血友病における血友病性関節症の予防及び/又は治療のための方法及び手段