JP2023524414A5 - - Google Patents

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Publication number
JP2023524414A5
JP2023524414A5 JP2022565544A JP2022565544A JP2023524414A5 JP 2023524414 A5 JP2023524414 A5 JP 2023524414A5 JP 2022565544 A JP2022565544 A JP 2022565544A JP 2022565544 A JP2022565544 A JP 2022565544A JP 2023524414 A5 JP2023524414 A5 JP 2023524414A5
Authority
JP
Japan
Prior art keywords
dnaj
fusion protein
group
aav
tdp
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
JP2022565544A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023524414A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/029289 external-priority patent/WO2021222168A2/en
Publication of JP2023524414A publication Critical patent/JP2023524414A/ja
Publication of JP2023524414A5 publication Critical patent/JP2023524414A5/ja
Pending legal-status Critical Current

Links

JP2022565544A 2020-04-28 2021-04-27 Tdp-43プロテイノパチーの処置のための組成物および方法 Pending JP2023524414A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063016707P 2020-04-28 2020-04-28
US63/016,707 2020-04-28
US202063035437P 2020-06-05 2020-06-05
US63/035,437 2020-06-05
PCT/US2021/029289 WO2021222168A2 (en) 2020-04-28 2021-04-27 Compositions and methods for the treatment of tdp-43 proteinopathies

Publications (2)

Publication Number Publication Date
JP2023524414A JP2023524414A (ja) 2023-06-12
JP2023524414A5 true JP2023524414A5 (https=) 2024-05-09

Family

ID=75905076

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022565544A Pending JP2023524414A (ja) 2020-04-28 2021-04-27 Tdp-43プロテイノパチーの処置のための組成物および方法

Country Status (10)

Country Link
US (1) US20240327477A1 (https=)
EP (1) EP4143215A2 (https=)
JP (1) JP2023524414A (https=)
KR (1) KR20230025659A (https=)
CN (1) CN115836129A (https=)
AU (1) AU2021265088A1 (https=)
BR (1) BR112022021604A2 (https=)
MX (1) MX2022013288A (https=)
TW (1) TW202206446A (https=)
WO (1) WO2021222168A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023548632A (ja) * 2020-10-26 2023-11-17 ソラ・バイオサイエンシズ・エルエルシー アルツハイマー病の処置のための組成物および方法
WO2023147473A2 (en) * 2022-01-27 2023-08-03 The Trustees Of Columbia University In The City Of New York Systems, methods, and compositions for rescuing protein misfolding
WO2023185017A1 (zh) * 2022-03-30 2023-10-05 呈诺再生医学科技(珠海横琴新区)有限公司 经修饰的rnf112或包含其的外泌体递送系统
CN114874332B (zh) * 2022-03-30 2023-01-10 呈诺再生医学科技(珠海横琴新区)有限公司 经修饰的rnf112作为治疗als药物的应用
AR133834A1 (es) * 2023-09-15 2025-11-05 Prothena Biosciences Ltd Agentes de penetración célular y sus usos
WO2025264861A1 (en) * 2024-06-18 2025-12-26 Yale University Compositions of bi-functional alpha helical peptides and methods thereof for treating tdp-43 proteinopathies

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8918616D0 (en) 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
US5804413A (en) 1992-07-31 1998-09-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains for gene transfer
GB9415319D0 (en) 1994-07-29 1994-09-21 Medical Res Council HSV viral vector
US5846782A (en) 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
AU8605598A (en) 1997-07-31 1999-02-22 University Of Pittsburgh Targeted hsv vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
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
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
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8865411B2 (en) * 2009-03-26 2014-10-21 National Institutes Of Health (Nih) Methods of identifying modulators of TDP-43 mediated cellular toxicity
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
US9587014B2 (en) 2011-10-28 2017-03-07 Biogen International Neuroscience Gmbh TDP-43 specific binding molecules
WO2014038884A1 (ko) * 2012-09-07 2014-03-13 주식회사 마크로젠 융합 단백질 및 이를 포함하는 암 진단용 조성물
WO2014053879A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
US9758807B2 (en) * 2012-12-05 2017-09-12 SOLA Biosciences, LLC Protein expression enhancing polypeptides
US20160220649A1 (en) * 2013-09-13 2016-08-04 Fundación Pública Andaluza Progreso Y Salud Combinations of aggregating proteins and molecular chaperone proteins for the treatment of proteinopathies or conformational diseases
WO2016053610A1 (en) 2014-10-03 2016-04-07 Academia Sinica Antibodies against pathological forms of tdp-43 and uses thereof
CA2874083C (en) * 2014-12-05 2024-01-02 Universite Laval Tdp-43-binding polypeptides useful for the treatment of neurodegenerative diseases
WO2018218252A1 (en) 2017-05-26 2018-11-29 California Institute Of Technology Method and apparatus for dynamic rf lens focusing and tracking of wireless power recovery unit
AU2019205090A1 (en) 2018-01-05 2020-08-06 Ac Immune Sa Misfolded TDP-43 binding molecules
EP3770262B1 (en) 2018-03-16 2026-01-14 National University Corporation Shiga University of Medical Science Antibody fragment degrading and removing abnormal tdp-43

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