JP2020533968A5 - - Google Patents

Download PDF

Info

Publication number
JP2020533968A5
JP2020533968A5 JP2020511199A JP2020511199A JP2020533968A5 JP 2020533968 A5 JP2020533968 A5 JP 2020533968A5 JP 2020511199 A JP2020511199 A JP 2020511199A JP 2020511199 A JP2020511199 A JP 2020511199A JP 2020533968 A5 JP2020533968 A5 JP 2020533968A5
Authority
JP
Japan
Prior art keywords
amino acids
sequence
sequences
seq
raavrec2
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
JP2020511199A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020533968A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/047466 external-priority patent/WO2019040586A1/en
Publication of JP2020533968A publication Critical patent/JP2020533968A/ja
Publication of JP2020533968A5 publication Critical patent/JP2020533968A5/ja
Pending legal-status Critical Current

Links

JP2020511199A 2017-08-25 2018-08-22 組換えアデノ随伴ベクター Pending JP2020533968A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762550458P 2017-08-25 2017-08-25
US62/550,458 2017-08-25
PCT/US2018/047466 WO2019040586A1 (en) 2017-08-25 2018-08-22 RECOMBINANT ADENO-ASSOCIATE VECTORS

Publications (2)

Publication Number Publication Date
JP2020533968A JP2020533968A (ja) 2020-11-26
JP2020533968A5 true JP2020533968A5 (enExample) 2021-09-30

Family

ID=65439655

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020511199A Pending JP2020533968A (ja) 2017-08-25 2018-08-22 組換えアデノ随伴ベクター

Country Status (10)

Country Link
US (2) US20190085358A1 (enExample)
EP (1) EP3655041A4 (enExample)
JP (1) JP2020533968A (enExample)
KR (1) KR20200042935A (enExample)
CN (1) CN111163811A (enExample)
AU (1) AU2018320849A1 (enExample)
CA (1) CA3073937A1 (enExample)
IL (1) IL272655A (enExample)
MX (1) MX2020002148A (enExample)
WO (1) WO2019040586A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2594616A (en) 2018-11-21 2021-11-03 Certego Therapeutics Inc Gaboxadol for reducing risk of suicide and rapid relief of depression
US20230118087A1 (en) * 2019-09-03 2023-04-20 Krystal Biotech, Inc. Compositions and methods for the treatment of congenital ichthyoses
IL298334A (en) 2020-05-20 2023-01-01 Certego Therapeutics Inc Ring deuterated gaboxadol and its use for the treatment of psychiatric disorders
JP2023540756A (ja) * 2020-09-04 2023-09-26 オハイオ・ステイト・イノベーション・ファウンデーション 向上した形質導入効率及び脳内における広範な分布を有する組換えアデノ随伴ウイルスベクターの新規の操作されたキャプシド血清型
WO2023044306A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69939169D1 (de) * 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
CA2376400A1 (en) * 1999-06-08 2000-12-14 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
US7169594B2 (en) * 2002-08-12 2007-01-30 Regents Of The University Of Michigan Diagnosis and treatment of diseases arising from defects in the tuberous sclerosis pathway
US7427396B2 (en) * 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
US8999678B2 (en) * 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2692868A1 (en) * 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
US10821154B2 (en) * 2013-05-01 2020-11-03 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
CA3209883A1 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US20150313903A1 (en) * 2014-05-05 2015-11-05 Ovid Therapeutics Inc. Methods of treating cognitive impairment associated with neurodegenerative disorders
BR112016028023A2 (pt) * 2014-05-30 2017-08-22 Univ Leland Stanford Junior Composições e métodos de administração de tratamentos para infecções virais latentes
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3795580A1 (en) * 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
DK3387137T3 (da) * 2015-12-11 2021-05-03 California Inst Of Techn Målretningspeptider til styring af adeno-associerede virusser (aav`er)
WO2017106354A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
CN116333057A (zh) * 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
AU2018221738B2 (en) * 2017-02-15 2024-06-27 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
CN110621318A (zh) * 2017-03-15 2019-12-27 奥维德医疗公司 排他地由设计者药物激活的设计者受体在治疗癫痫发作紊乱中的用途
EP3710010A4 (en) * 2017-12-20 2021-09-22 Ovid Therapeutics Inc. USE OF HM4DI IN TREATMENT OF SEQUENCE DISEASES
WO2019241624A1 (en) * 2018-06-14 2019-12-19 Ovid Therapeutics Inc. Use of mir-92a or mir-145 in the treatment of angelman syndrome
CN113966399A (zh) * 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
KR20220098384A (ko) * 2019-11-19 2022-07-12 아스클레피오스 바이오파마슈티컬, 인크. 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스
IL293431A (en) * 2019-12-05 2022-07-01 Univ Texas Transgene cassettes designed to express human mecp2 gene
CA3116391A1 (en) * 2020-09-14 2022-03-14 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
US20240309055A1 (en) * 2021-02-03 2024-09-19 The University Of North Carolina At Chapel Hill Protein m analogs and fusion proteins and their use for inhibiting antibody function
WO2022235702A1 (en) * 2021-05-04 2022-11-10 California Institute Of Technology Recombinant aavs for delivery to central nervous system and brain vasculature
CN114259502A (zh) * 2021-12-24 2022-04-01 南京鼓楼医院 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用
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

Similar Documents

Publication Publication Date Title
JP2020533968A5 (enExample)
US12065467B2 (en) High-transduction-efficiency rAAV vectors, compositions, and methods of use
US11124544B2 (en) AAV vectors with high transduction efficiency and uses thereof for gene therapy
JP6805174B2 (ja) 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むaav分離株及び融合タンパク質
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
JP2022065003A (ja) パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
RU2013152648A (ru) Аденовирус обезьян и гибридные аденовирусные векторы
JP2020510428A5 (enExample)
WO2019169004A8 (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
JP2021520203A (ja) 減少した肝臓向性を有するAAV9とAAVrh74とのハイブリッド組換えアデノ随伴ウイルス血清型
PH12018502387A1 (en) Adeno-associated virus variant capsids and methods of use thereof
RU2017125234A (ru) Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний
JPWO2019193119A5 (enExample)
JP2017536116A5 (enExample)
JP2002538770A5 (enExample)
PH12021552637A1 (en) Compositions useful for treatment of pompe disease
RU2019126509A (ru) Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
US20230212609A1 (en) Codon-optimized nucleic acid encoding smn1 protein
CN114276419B (zh) 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
JP7811576B2 (ja) SARS-CoV-2に対するAAV5に基づくワクチン
WO2024220461A3 (en) Aav capsid proteins having mutations in the vp1 region
RU2019113940A (ru) Мозаичные вакцины от вируса ящура серотипа а
RU2021124726A (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе
HK40114472A (zh) 非包膜病毒的药物组合物