JPWO2021138559A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2021138559A5
JPWO2021138559A5 JP2022540917A JP2022540917A JPWO2021138559A5 JP WO2021138559 A5 JPWO2021138559 A5 JP WO2021138559A5 JP 2022540917 A JP2022540917 A JP 2022540917A JP 2022540917 A JP2022540917 A JP 2022540917A JP WO2021138559 A5 JPWO2021138559 A5 JP WO2021138559A5
Authority
JP
Japan
Prior art keywords
acid sequence
seq
optionally
amino acid
promoter
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
JP2022540917A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023509443A5 (https=
JP2023509443A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/067664 external-priority patent/WO2021138559A1/en
Publication of JP2023509443A publication Critical patent/JP2023509443A/ja
Publication of JP2023509443A5 publication Critical patent/JP2023509443A5/ja
Publication of JPWO2021138559A5 publication Critical patent/JPWO2021138559A5/ja
Pending legal-status Critical Current

Links

JP2022540917A 2019-12-31 2020-12-31 改善されたａａｖ－ａｂｃｄ１コンストラクトならびに副腎白質ジストロフィー（ａｌｄ）および／または副腎脊髄ニューロパチー（ａｍｎ）の処置または予防のための使用 Pending JP2023509443A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962955667P 2019-12-31 2019-12-31
US62/955,667 2019-12-31
PCT/US2020/067664 WO2021138559A1 (en) 2019-12-31 2020-12-31 Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)

Publications (3)

Publication Number Publication Date
JP2023509443A JP2023509443A (ja) 2023-03-08
JP2023509443A5 JP2023509443A5 (https=) 2023-07-05
JPWO2021138559A5 true JPWO2021138559A5 (https=) 2023-07-05

Family

ID=76686949

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022540917A Pending JP2023509443A (ja) 2019-12-31 2020-12-31 改善されたａａｖ－ａｂｃｄ１コンストラクトならびに副腎白質ジストロフィー（ａｌｄ）および／または副腎脊髄ニューロパチー（ａｍｎ）の処置または予防のための使用

Country Status (11)

Country Link
US (2) US11779655B2 (https=)
EP (1) EP4085143B1 (https=)
JP (1) JP2023509443A (https=)
KR (1) KR20220128632A (https=)
CN (1) CN116096904A (https=)
AU (1) AU2020416291A1 (https=)
BR (1) BR112022013027A2 (https=)
CA (1) CA3166374A1 (https=)
IL (1) IL294278A (https=)
MX (1) MX2022008201A (https=)
WO (1) WO2021138559A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3228344A1 (en) * 2021-08-06 2023-02-09 Wuhan Neurophth Biotechnology Limited Company Composition and method for treating leber's hereditary optic neuropathy caused by nd4 mutation
KR20250009975A (ko) * 2022-05-12 2025-01-20 아브너진 인코포레이티드 재조합 파르보바이러스 생산을 위한 조성물 및 방법
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
AU2024315073A1 (en) * 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8466271B2 (en) * 2008-07-23 2013-06-18 Boehringer Ingelheim Pharma Gmbh & Co, Kg Regulatory elements
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
RU2014100160A (ru) * 2011-06-10 2015-07-20 Блуберд Байо, Инк. Векторы генной терапии адренолейкодистрофии и адреномиелонейропатии
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
EP2940131B1 (en) * 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
WO2015153889A2 (en) * 2014-04-02 2015-10-08 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
JP2018510160A (ja) * 2015-03-20 2018-04-12 ブルーバード バイオ， インコーポレイテッド ベクター製剤
CA3019315A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
MX2018005689A (es) 2015-11-05 2018-08-01 Massachusetts Gen Hospital Administracion intratecal de secuencias de acido nucleico que codifican abcd1 para el tratamiento de la adrenomieloneuropatia.
PL3411484T3 (pl) * 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
EP3413925A1 (en) * 2016-02-12 2018-12-19 Combigene AB Vector
CN107287239B (zh) * 2016-04-11 2020-09-22 厦门继景生物技术有限责任公司 一种用于视网膜色素变性的基因治疗载体及药物
CN109843306A (zh) * 2016-08-19 2019-06-04 卡琳缪恩股份有限公司 使用自身互补型重组腺相关病毒治疗病症的方法和组合物
WO2018136434A1 (en) * 2017-01-17 2018-07-26 Children's Medical Center Corporation Compositions and methods for diagnosing and treating peroxisomal diseases
EP3592137B1 (en) * 2017-03-10 2024-08-07 The Medical College of Wisconsin, Inc. Riboswitch modulated gene therapy for retinal diseases
WO2019060649A1 (en) * 2017-09-22 2019-03-28 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
MX2020004092A (es) * 2017-10-18 2021-01-08 Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas.
GB201817470D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy

Similar Documents

Publication Publication Date Title
JP7237843B2 (ja) 修飾されたａａｖキャプシドタンパク質およびその使用
US12359219B2 (en) Transgene cassettes designed to express a human MECP2 gene
WO2004020600A2 (en) Modified aav
US20240376498A1 (en) Transgene cassettes, aav vectors and aav viral vectors for the expression of human codon-optimized slc6a1
US12534741B2 (en) Compositions and methods for treatment of neurological disorders
CN115151647A (zh) Dna扩增方法
US20250283111A1 (en) Recombinant adeno-associated viral vector for gene delivery
JPWO2020132059A5 (https=)
JPWO2021138559A5 (https=)
JPWO2020069339A5 (https=)
WO2016037931A1 (en) Nucleic acid constructs and expression vectors for gene therapy of acute porphyrias and other diseases
WO2023081683A1 (en) Gene therapy for prader willi syndrome
US20240318198A1 (en) Gene therapy for angelman syndrome
EP4409008A1 (en) Slc13a5 gene therapy vectors and uses thereof
CN117003833A (zh) 肝脏特异性的腺相关病毒血清型及应用
US12480098B1 (en) Adeno-associated virus variant
WO2025162449A1 (en) Compositions and methods for the treatment of heart disease
WO2024163335A2 (en) Pten gene therapy vectors and uses thereof
CN118955653A (zh) 腺相关病毒衣壳蛋白的突变体、编码其的核酸及应用
OA21075A (en) Codon-optimized nucleic acid that encodes SMN1 protein, and use thereof
CN121086028A (zh) 定向进化构建的aav衣壳变体及其用途
JPWO2023213831A5 (https=)