JP2020508667A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020508667A5 JP2020508667A5 JP2019546041A JP2019546041A JP2020508667A5 JP 2020508667 A5 JP2020508667 A5 JP 2020508667A5 JP 2019546041 A JP2019546041 A JP 2019546041A JP 2019546041 A JP2019546041 A JP 2019546041A JP 2020508667 A5 JP2020508667 A5 JP 2020508667A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- raav
- amino acid
- serotype
- phenylalanine
- 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.)
- Granted
Links
- 150000001413 amino acids Chemical group 0.000 claims 27
- 108090000565 Capsid Proteins Proteins 0.000 claims 24
- 102100023321 Ceruloplasmin Human genes 0.000 claims 24
- 235000001014 amino acid Nutrition 0.000 claims 20
- 238000006467 substitution reaction Methods 0.000 claims 16
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 13
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical group OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 claims 13
- 235000003704 aspartic acid Nutrition 0.000 claims 13
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Chemical group OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 claims 13
- 229940024606 amino acid Drugs 0.000 claims 12
- 239000002245 particle Substances 0.000 claims 11
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Chemical group OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims 10
- 235000008729 phenylalanine Nutrition 0.000 claims 10
- COLNVLDHVKWLRT-QMMMGPOBSA-N phenylalanine group Chemical group N[C@@H](CC1=CC=CC=C1)C(=O)O COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims 10
- 239000000203 mixture Substances 0.000 claims 7
- 210000004027 cell Anatomy 0.000 claims 6
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical group C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 claims 5
- 235000004279 alanine Nutrition 0.000 claims 5
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical group NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 4
- 230000002207 retinal effect Effects 0.000 claims 4
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Chemical group OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 claims 3
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 claims 3
- WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical group OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 claims 3
- AYFVYJQAPQTCCC-GBXIJSLDSA-N L-threonine Chemical compound C[C@@H](O)[C@H](N)C(O)=O AYFVYJQAPQTCCC-GBXIJSLDSA-N 0.000 claims 3
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 claims 3
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims 3
- 239000004473 Threonine Substances 0.000 claims 3
- 235000013922 glutamic acid Nutrition 0.000 claims 3
- 239000004220 glutamic acid Chemical group 0.000 claims 3
- 235000013930 proline Nutrition 0.000 claims 3
- 235000018102 proteins Nutrition 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 235000008521 threonine Nutrition 0.000 claims 3
- 239000004471 Glycine Chemical group 0.000 claims 2
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 claims 2
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- 239000004474 valine Substances 0.000 claims 2
- 235000014393 valine Nutrition 0.000 claims 2
- 239000004475 Arginine Substances 0.000 claims 1
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims 1
- 108010001857 Cell Surface Receptors Proteins 0.000 claims 1
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 1
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 1
- 102000019034 Chemokines Human genes 0.000 claims 1
- 108010012236 Chemokines Proteins 0.000 claims 1
- 102000004127 Cytokines Human genes 0.000 claims 1
- 108090000695 Cytokines Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000008394 Immunoglobulin Fragments Human genes 0.000 claims 1
- 108010021625 Immunoglobulin Fragments Proteins 0.000 claims 1
- 102000004310 Ion Channels Human genes 0.000 claims 1
- 108090000862 Ion Channels Proteins 0.000 claims 1
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 claims 1
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical group OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 claims 1
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 claims 1
- 108010052285 Membrane Proteins Proteins 0.000 claims 1
- 101710163270 Nuclease Proteins 0.000 claims 1
- 230000003213 activating effect Effects 0.000 claims 1
- 210000000411 amacrine cell Anatomy 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- 235000009697 arginine Nutrition 0.000 claims 1
- 235000009582 asparagine Nutrition 0.000 claims 1
- 229960001230 asparagine Drugs 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000010362 genome editing Methods 0.000 claims 1
- 239000003292 glue Substances 0.000 claims 1
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 1
- 235000004554 glutamine Nutrition 0.000 claims 1
- 239000003102 growth factor Substances 0.000 claims 1
- 210000002287 horizontal cell Anatomy 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
- 229940088597 hormone Drugs 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 230000006525 intracellular process Effects 0.000 claims 1
- 102000006240 membrane receptors Human genes 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 210000000608 photoreceptor cell Anatomy 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 239000013646 rAAV2 vector Substances 0.000 claims 1
- 210000003994 retinal ganglion cell Anatomy 0.000 claims 1
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 210000001585 trabecular meshwork Anatomy 0.000 claims 1
- 230000002463 transducing effect Effects 0.000 claims 1
Applications Claiming Priority (5)
| 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020508667A JP2020508667A (ja) | 2020-03-26 |
| JP2020508667A5 true JP2020508667A5 (enExample) | 2021-04-08 |
| JP7237843B2 JP7237843B2 (ja) | 2023-03-13 |
Family
ID=63253351
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019546041A Active JP7237843B2 (ja) | 2017-02-21 | 2018-02-21 | 修飾されたａａｖキャプシドタンパク質およびその使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US10793606B2 (enExample) |
| EP (1) | EP3585883A4 (enExample) |
| JP (1) | JP7237843B2 (enExample) |
| AU (1) | AU2018224044B2 (enExample) |
| CA (2) | CA3114549A1 (enExample) |
| WO (1) | WO2018156654A1 (enExample) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2714007T3 (es) | 2007-04-09 | 2019-05-24 | Univ Florida | Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso |
| 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 |
| WO2015048534A1 (en) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| EP3585883A4 (en) | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| CN111867635A (zh) * | 2017-10-20 | 2020-10-30 | 法国国家卫生及研究医学协会 | 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法 |
| 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 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| BR112021006052A2 (pt) | 2018-10-01 | 2021-09-08 | Ultragenyx Pharmaceutical Inc. | Terapia gênica para tratar acidemia propiônica |
| AU2019362280B2 (en) * | 2018-10-17 | 2026-03-19 | Children's Medical Research Institute | Nucleic acid molecules and methods for AAV vector selection |
| EP3914229A4 (en) * | 2019-01-23 | 2022-11-02 | University of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
| CA3144986A1 (en) | 2019-06-27 | 2020-12-30 | University Of Florida Research Foundation, Incorporated | Enhancing aav-mediated transduction of ocular tissues with hyaluronic acid |
| WO2021067448A1 (en) * | 2019-09-30 | 2021-04-08 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| WO2023172491A1 (en) | 2022-03-07 | 2023-09-14 | Ultragenyx Pharmaceutical Inc. | Modified batch aav production systems and methods |
| JP2023514204A (ja) | 2020-02-14 | 2023-04-05 | 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 | Ｃｄｋｌ５欠損障害を処置するための遺伝子治療 |
| US20230235353A1 (en) | 2020-03-19 | 2023-07-27 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| WO2021202532A1 (en) | 2020-03-31 | 2021-10-07 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| 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 |
| US20230265455A1 (en) | 2020-07-29 | 2023-08-24 | 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 |
| US20250019721A1 (en) | 2021-08-11 | 2025-01-16 | 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 |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | 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 |
| AU2024332181A1 (en) | 2023-08-31 | 2026-02-12 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2025235555A1 (en) * | 2024-05-06 | 2025-11-13 | University Of Florida Research Foundation, Incorporated | Rational design of aav vectors with limited liver targeting |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Family Cites Families (51)
| 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 |
| 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 |
| EP1610751A4 (en) | 2001-04-26 | 2006-05-24 | Univ Texas | THERAPEUTIC AGENT / LIGAND CONJUGATE COMPOSITIONS, ITS SYNTHESIS PROCESSES AND ITS APPLICATION |
| JP4353799B2 (ja) | 2001-10-09 | 2009-10-28 | ザ・ジョーンズ・ホプキンス・ユニバーシティ | 転移に関連するホスファターゼ |
| US20050106700A1 (en) | 2001-10-11 | 2005-05-19 | Tsuyoshi Nomura | Method of purifying recombinant fused protein and method of producing protein using the same |
| 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 |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| AU2003265855A1 (en) | 2002-08-28 | 2004-03-19 | University Of Florida | Modified aav |
| JP2006524035A (ja) | 2002-12-26 | 2006-10-26 | シーマインズ リミテッド ライアビリティ カンパニー | 癌の診断、予後診断および治療のための方法および組成物 |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| SI1633772T1 (sl) | 2003-06-19 | 2016-06-30 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| 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 |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| KR20090114430A (ko) | 2007-02-02 | 2009-11-03 | 베일러 리서치 인스티튜트 | 항원-제시 세포 상에서 발현된 ｄｃｉｒ에 대한 항원 표적화에 기초한 백신 |
| 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 |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| ES2714007T3 (es) | 2007-04-09 | 2019-05-24 | Univ Florida | Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso |
| 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 |
| WO2010011404A2 (en) * | 2008-05-20 | 2010-01-28 | Eos Neuroscience, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| 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 |
| EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| WO2012057363A1 (ja) | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| US10415056B2 (en) | 2010-11-10 | 2019-09-17 | Fred Hutchinson Cancer Research Center | Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination |
| WO2012109570A1 (en) * | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| AU2013216382B2 (en) | 2012-01-31 | 2018-08-02 | Hadasit Medical Research Services And Development Ltd. | Methods of selecting retinal pigmented epithelial cells |
| NZ701693A (en) * | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| ES2926774T3 (es) | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| EP3492597A3 (en) | 2013-05-21 | 2019-08-28 | University of Florida Research Foundation, Inc. | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer |
| WO2015048534A1 (en) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| US20160272687A1 (en) | 2013-11-08 | 2016-09-22 | 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 |
| US20170007720A1 (en) | 2014-02-21 | 2017-01-12 | 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 |
| WO2016133917A1 (en) | 2015-02-16 | 2016-08-25 | University Of Florida Research Foundation | Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ | アデノ随伴ウイルス変異体及びその使用方法 |
| 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 |
| WO2017004514A1 (en) | 2015-07-02 | 2017-01-05 | University Of Florida Research Foundation, Inc. | 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 |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| 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 |
| EP3585883A4 (en) | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| CA3146702A1 (en) | 2019-08-09 | 2021-02-18 | Sergei Zolotukhin | Aav capsid variants for targeting human glioblastoma cells |
-
2018
- 2018-02-21 EP EP18756857.1A patent/EP3585883A4/en active Pending
- 2018-02-21 JP JP2019546041A patent/JP7237843B2/ja active Active
- 2018-02-21 WO PCT/US2018/019050 patent/WO2018156654A1/en not_active Ceased
- 2018-02-21 AU AU2018224044A patent/AU2018224044B2/en active Active
- 2018-02-21 CA CA3114549A patent/CA3114549A1/en active Pending
- 2018-02-21 CA CA3054131A patent/CA3054131C/en active Active
-
2019
- 2019-08-21 US US16/547,532 patent/US10793606B2/en active Active
-
2020
- 2020-09-01 US US17/009,536 patent/US11332502B2/en active Active
-
2022
- 2022-03-08 US US17/689,324 patent/US11767346B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020508667A5 (enExample) | ||
| US20240209031A1 (en) | Methods and compositions for gene transfer across the vasculature | |
| US11124544B2 (en) | AAV vectors with high transduction efficiency and uses thereof for gene therapy | |
| JP2021121638A5 (enExample) | ||
| CA3054131C (en) | Modified aav capsid proteins and uses thereof | |
| JP2020028308A5 (enExample) | ||
| JP2014239686A5 (enExample) | ||
| FI3445773T3 (fi) | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä | |
| FI4211231T3 (fi) | Peptidimodifioitu aav-kapsidi | |
| WO2019060454A3 (en) | Adeno-associated virus variant capsids and methods of use thereof | |
| IL317938A (en) | Recombinant viral vectors with altered tropism and their uses for the targeted introduction of genetic material into human cells | |
| JP2011101646A5 (enExample) | ||
| IL318395A (en) | Recombinant viral particles with altered tropism, and their uses for targeted introduction of genetic material into human cells | |
| WO2012172277A4 (en) | Simian adenovirus and hybrid adenoviral vectors | |
| JP2018515096A5 (enExample) | ||
| HRP20241577T1 (hr) | Virioni adeno-pridruženog virusa s varijantom kapside i postupci njihove upotrebe | |
| JP2020511167A5 (enExample) | ||
| US20250059564A1 (en) | Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer | |
| CN115443288A (zh) | 一种免疫球蛋白降解酶IdeE的突变体 | |
| JPWO2020218419A5 (enExample) | ||
| CN111718418B (zh) | 一种增强基因编辑的融合蛋白及其应用 | |
| JP2020533968A5 (enExample) | ||
| PH12022551878A1 (en) | Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof | |
| WO2004106360A3 (en) | Viral vectors with improved properties | |
| WO2024220461A3 (en) | Aav capsid proteins having mutations in the vp1 region |