AU2018224044B2 - Modified AAV capsid proteins and uses thereof - Google Patents

Modified AAV capsid proteins and uses thereof Download PDF

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AU2018224044B2
AU2018224044B2 AU2018224044A AU2018224044A AU2018224044B2 AU 2018224044 B2 AU2018224044 B2 AU 2018224044B2 AU 2018224044 A AU2018224044 A AU 2018224044A AU 2018224044 A AU2018224044 A AU 2018224044A AU 2018224044 B2 AU2018224044 B2 AU 2018224044B2
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AU2018224044A1 (en
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Sanford L. Boye
Shannon E. Boye
Paul D. Gamlin
Damien MARSIC
Hector Ruben Mendez-Gomez
Nicholas Muzyczka
Sergei Zolotukhin
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University of Florida Research Foundation Inc
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AU2018224044A 2017-02-21 2018-02-21 Modified AAV capsid proteins and uses thereof Active AU2018224044B2 (en)

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Application Number Priority Date Filing Date Title
US201762461770P 2017-02-21 2017-02-21
US62/461,770 2017-02-21
US201862625486P 2018-02-02 2018-02-02
US62/625,486 2018-02-02
PCT/US2018/019050 WO2018156654A1 (en) 2017-02-21 2018-02-21 Modified aav capsid proteins and uses thereof

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AU2018224044A1 AU2018224044A1 (en) 2019-09-19
AU2018224044B2 true AU2018224044B2 (en) 2024-01-25

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US (3) US10793606B2 (cg-RX-API-DMAC7.html)
EP (1) EP3585883A4 (cg-RX-API-DMAC7.html)
JP (1) JP7237843B2 (cg-RX-API-DMAC7.html)
AU (1) AU2018224044B2 (cg-RX-API-DMAC7.html)
CA (2) CA3114549A1 (cg-RX-API-DMAC7.html)
WO (1) WO2018156654A1 (cg-RX-API-DMAC7.html)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
JP7237843B2 (ja) 2017-02-21 2023-03-13 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 修飾されたaavキャプシドタンパク質およびその使用
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019077159A1 (en) 2017-10-20 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR
SG11202009854XA (en) * 2018-02-15 2020-11-27 Bjoerklund Tomas Modified viral capsids
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3113975A1 (en) 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
EP3867359A4 (en) * 2018-10-17 2022-09-21 Children's Medical Research Institute NUCLEIC ACID MOLECULES AND AAV VECTOR SELECTION METHODS
WO2020154535A1 (en) 2019-01-23 2020-07-30 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
AU2020308909A1 (en) 2019-06-27 2022-01-27 University Of Florida Research Foundation, Incorporated Enhancing AAV-mediated transduction of ocular tissues with hyaluronic acid
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
IL294781A (en) 2020-02-14 2022-09-01 Ultragenyx Pharmaceutical Inc Gene therapy for treating cdkl5 deficiency disorder
JP2023518415A (ja) 2020-03-19 2023-05-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えaavにおいてcapおよびrep配列の逆パッケージングを低減するための組成物および方法
JP2023520402A (ja) 2020-03-31 2023-05-17 ウルトラジェニックス ファーマシューティカル インコーポレイテッド プロピオン酸血症を処置するための遺伝子治療
US20230233708A1 (en) * 2020-04-14 2023-07-27 University Of Florida Research Foundation, Incorporated Enhancing aav-mediated delivery and transduction with polyvinyl alcohol
CN111729078B (zh) * 2020-07-24 2020-11-20 苏州世诺生物技术有限公司 鸡传染性贫血病毒基因工程疫苗
CA3190214A1 (en) 2020-07-29 2022-02-03 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies
JP2023550548A (ja) * 2020-11-10 2023-12-01 アビラマックス・バイオファーマ・インコーポレイテッド 操作されたウイルスカプシドおよび使用方法
WO2022150409A1 (en) * 2021-01-05 2022-07-14 Mayo Foundation For Medical Education And Research Treatments for intraocular pressure related disorders
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
CA3245327A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. MODIFIED BATCH-BASED AAV PRODUCTION SYSTEMS AND PROCESSES
AU2024234833A1 (en) 2023-03-10 2025-10-23 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2024229049A1 (en) 2023-05-01 2024-11-07 University Of Florida Research Foundation, Incorporated Dual aav vectors for treating stargardt disease
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025235555A1 (en) * 2024-05-06 2025-11-13 University Of Florida Research Foundation, Incorporated Rational design of aav vectors with limited liver targeting

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160369298A1 (en) * 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP2002538770A (ja) 1998-11-10 2002-11-19 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 ウイルスベクターとその製造及び投与の方法
EP1389090A2 (en) 2001-04-26 2004-02-18 Board of Regents, The University of Texas System Diagnostic imaging compositions, their methods of synthesis and use
WO2003031930A2 (en) 2001-10-09 2003-04-17 The Johns Hopkins University A phosphatase associated with metastasis
WO2003033695A1 (fr) 2001-10-11 2003-04-24 Katakura Industries Co., Ltd. Procede de purification de proteine fusionnee recombinee et procede de production de proteine utilisant ledit procede de purification
US20030225260A1 (en) 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
WO2003093479A1 (en) 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved raav expression systems and methods for enhancing transduction of mammalian neural cells
US20060093589A1 (en) 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
WO2004060302A2 (en) 2002-12-26 2004-07-22 Cemines, Llc. Methods and compositions for the diagnosis, prognosis, and treatment of cancer
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
SI2657248T1 (sl) * 2003-06-19 2017-07-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in njihova uporaba
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
WO2006066066A2 (en) 2004-12-15 2006-06-22 University Of North Carolina At Chapel Hill Chimeric vectors
EP4234687A2 (en) 2005-04-07 2023-08-30 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
TWI422594B (zh) 2007-02-02 2014-01-11 Baylor Res Inst 經由樹狀細胞去唾液酸糖蛋白受體(dc-asgpr)接合抗原呈現細胞之藥劑
MX2009008140A (es) 2007-02-02 2009-10-26 Baylor Res Inst Vacunas a base de antigeno dirigido a dcir expresado en celulas que presentan antigeno.
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
HRP20161150T1 (hr) 2007-04-09 2016-11-18 University Of Florida Research Foundation, Inc. PRIPRAVCI rAAV VEKTORA KOJI IMAJU TIROZIN-MODIFICIRANE CAPSID PROTEINE I POSTUPCI ZA UPORABU
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
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
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
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
WO2012064960A2 (en) 2010-11-10 2012-05-18 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
US9409953B2 (en) * 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
US9658216B2 (en) 2012-01-31 2017-05-23 Cell Cure Neurosciences Ltd. Methods of selecting retinal pigmented epithelial cells
HUE054087T2 (hu) * 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
WO2014144229A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
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
WO2015108610A1 (en) * 2013-11-08 2015-07-23 The Board Of Trustees Of The University Of Arkansas Adeno-associated virus "x" oncogene
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US11000597B2 (en) 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
CN107532177A (zh) 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
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
US11266748B2 (en) 2015-07-02 2022-03-08 University Of Florida Research Foundation, Incorporated Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
CA3008013A1 (en) * 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
JP7237843B2 (ja) 2017-02-21 2023-03-13 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 修飾されたaavキャプシドタンパク質およびその使用
JP2022544237A (ja) 2019-08-09 2022-10-17 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. ヒト神経膠芽腫細胞をターゲティングするためのaavカプシドバリアント

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160369298A1 (en) * 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy

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