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MX2018007237A - Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus. - Google Patents

Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus.

Info

Publication number
MX2018007237A
MX2018007237A MX2018007237A MX2018007237A MX2018007237A MX 2018007237 A MX2018007237 A MX 2018007237A MX 2018007237 A MX2018007237 A MX 2018007237A MX 2018007237 A MX2018007237 A MX 2018007237A MX 2018007237 A MX2018007237 A MX 2018007237A
Authority
MX
Mexico
Prior art keywords
modified
improved supply
parvovirus vectors
proteins
capside proteins
Prior art date
Application number
MX2018007237A
Other languages
English (en)
Other versions
MX395524B (es
Inventor
Jude Samulski Richard
Warischalk Jayme
Original Assignee
Univ North Carolina Chapel Hill
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ North Carolina Chapel Hill filed Critical Univ North Carolina Chapel Hill
Publication of MX2018007237A publication Critical patent/MX2018007237A/es
Publication of MX395524B publication Critical patent/MX395524B/es

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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Abstract

Esta invención se refiere a proteínas modificadas de cápside de parvovirus con una mejor eficiencia de transducción, a vectores virales que las comprenden, y a métodos de uso de las mismas para el suministro de ácidos nucleicos a una célula o un sujeto.
MX2018007237A 2015-12-14 2016-12-14 Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus. MX395524B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562266941P 2015-12-14 2015-12-14
PCT/US2016/066466 WO2017106236A1 (en) 2015-12-14 2016-12-14 Modified capsid proteins for enhanced delivery of parvovirus vectors

Publications (2)

Publication Number Publication Date
MX2018007237A true MX2018007237A (es) 2018-12-11
MX395524B MX395524B (es) 2025-03-25

Family

ID=59057532

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018007237A MX395524B (es) 2015-12-14 2016-12-14 Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus.
MX2022006652A MX2022006652A (es) 2015-12-14 2018-06-13 Proteinas de capside modificadas para un suministro mejorado de vectores de parvovirus.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022006652A MX2022006652A (es) 2015-12-14 2018-06-13 Proteinas de capside modificadas para un suministro mejorado de vectores de parvovirus.

Country Status (13)

Country Link
US (3) US11124546B2 (es)
EP (2) EP3390429B1 (es)
JP (3) JP7406783B2 (es)
CN (2) CN116478254A (es)
AU (2) AU2016371779C1 (es)
BR (1) BR112018011881A2 (es)
CA (1) CA3008013A1 (es)
DK (1) DK3390429T3 (es)
ES (1) ES3040934T3 (es)
MX (2) MX395524B (es)
PL (1) PL3390429T3 (es)
RU (1) RU2751953C2 (es)
WO (1) WO2017106236A1 (es)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP3831842A1 (en) 2015-09-28 2021-06-09 The University of North Carolina at Chapel Hill Methods and compositions for antibody-evading virus vectors
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
JP7406783B2 (ja) * 2015-12-14 2023-12-28 ザ 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 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
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
CA3114549A1 (en) * 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
CN112218880A (zh) * 2018-03-29 2021-01-12 阿斯克肋匹奥生物制药公司 逃避中和的肝趋向性重组aav6载体
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
EP3813845A4 (en) 2018-05-15 2022-10-26 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
WO2020219656A1 (en) * 2019-04-26 2020-10-29 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
EP3999120A4 (en) * 2019-07-04 2024-02-21 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療ｃ型尼曼—匹克病之腺相關病毒載體
CN110950934B (zh) * 2019-12-31 2022-11-04 复旦大学 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
MY209860A (en) 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
US20230374542A1 (en) 2020-10-07 2023-11-23 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21)
CN117083070A (zh) * 2020-12-23 2023-11-17 森特尼医疗公司 用于基因疗法的原细小病毒和四细小病毒组合物和方法
AU2022307365A1 (en) 2021-07-08 2024-02-01 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
TW202421787A (zh) 2022-09-06 2024-06-01 美商特納亞治療股份有限公司 保護心臟之心臟病療法
WO2024112813A2 (en) * 2022-11-21 2024-05-30 Shape Therapeutics Inc. Systems for amplification of aav cap protein
WO2024197242A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024220452A2 (en) * 2023-04-17 2024-10-24 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced intra-articular delivery of adenovirus-associated virus (aav) vectors

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
JPH01503675A (ja) 1986-09-08 1989-12-14 アプライド・バイオテクノロジー・インコーポレーテツド 中空ウイルスキヤプシドのワクチン
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (en) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
JP4002605B2 (ja) 1995-06-07 2007-11-07 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 筋組織のａａｖ形質導入
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
EP0883344B2 (en) 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
NZ511171A (en) 1998-09-22 2004-02-27 Univ Florida Methods for large-scale production of recombinant AAV vectors
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
AU2003223766A1 (en) 2002-04-30 2003-11-17 University Of North Carolina At Chapel Hill Secretion signal vectors
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
CA2591544A1 (en) 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
EP2357010B1 (en) 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
US20090191597A1 (en) * 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
ES2634118T3 (es) * 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
JP6387350B2 (ja) 2012-09-28 2018-09-05 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 希突起神経膠細胞を標的とするアデノ随伴ウイルスベクター
AU2014274457B2 (en) 2013-05-21 2019-10-24 University Of Florida Research Foundation, Inc. Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
JP6985795B2 (ja) * 2013-09-26 2021-12-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
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
EP3831842A1 (en) * 2015-09-28 2021-06-09 The University of North Carolina at Chapel Hill Methods and compositions for antibody-evading virus vectors
JP7406783B2 (ja) * 2015-12-14 2023-12-28 ザ 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 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

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