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 (https=) | 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 (https=) |
| EP (1) | EP3585883A4 (https=) |
| JP (1) | JP7237843B2 (https=) |
| AU (1) | AU2018224044B2 (https=) |
| CA (2) | CA3054131C (https=) |
| WO (1) | WO2018156654A1 (https=) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| 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 |
| 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 |
| AU2018224044B2 (en) | 2017-02-21 | 2024-01-25 | The Uab Research Foundation | Modified AAV capsid proteins and uses thereof |
| 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 |
| JP7420710B2 (ja) | 2017-10-20 | 2024-01-23 | アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） | 組換えａａｖ９由来ベクターの治療有効量の網膜下送達を含む、対象の錐体視細胞において目的のポリヌクレオチドを発現させる方法 |
| CN112105630A (zh) * | 2018-02-15 | 2020-12-18 | T·比约尔克隆德 | 经修饰的病毒衣壳 |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3861131A4 (en) | 2018-10-01 | 2022-06-15 | Ultragenyx Pharmaceutical Inc. | GENE THERAPY FOR THE TREATMENT OF PROPIONIC ACIDosis |
| WO2020077411A1 (en) * | 2018-10-17 | 2020-04-23 | Children's Medical Research Institute | Nucleic acid molecules and methods for aav vector selection |
| US20220133909A1 (en) * | 2019-01-23 | 2022-05-05 | 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 |
| CA3153133A1 (en) * | 2019-09-30 | 2021-04-08 | Chris Bartolome | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| US12544437B2 (en) | 2019-10-04 | 2026-02-10 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant AAV |
| MX2022009982A (es) | 2020-02-14 | 2022-09-12 | Ultragenyx Pharmaceutical Inc | Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5. |
| 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 |
| WO2021202532A1 (en) | 2020-03-31 | 2021-10-07 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| WO2021211641A2 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Researchfoundation, 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 |
| WO2022026632A2 (en) | 2020-07-29 | 2022-02-03 | University Of Florida Research Foundation, Incorporated | Improved aav-mediated x-linked retinoschisis therapies |
| EP4243848A4 (en) * | 2020-11-10 | 2025-07-23 | Avirmax Biopharma Inc | MODIFIED VIRAL CAPSIDS AND METHODS OF USE |
| TW202235618A (zh) * | 2021-01-05 | 2022-09-16 | 美國馬友醫藥教育研究基金會 | 治療眼內壓相關疾患 |
| WO2023019168A1 (en) | 2021-08-11 | 2023-02-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| CN119137263A (zh) | 2022-03-07 | 2024-12-13 | 奥特吉尼克斯制药公司 | 改进的分批aav产生系统和方法 |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| AU2024265002A1 (en) | 2023-05-01 | 2025-10-30 | Atsena Therapeutics, Inc. | 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) |
| 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 |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US20030003048A1 (en) | 2001-04-26 | 2003-01-02 | Chun Li | Diagnostic imaging compositions, their methods of synthesis and use |
| AU2002362646A1 (en) | 2001-10-09 | 2003-04-22 | The Johns Hopkins University | A phosphatase associated with metastasis |
| 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 |
| US20060127358A1 (en) | 2002-05-01 | 2006-06-15 | Nicholas Muzyczka | 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 |
| CA2511816A1 (en) | 2002-12-26 | 2004-07-22 | Cemines, Inc. | 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 |
| SI3235827T1 (sl) * | 2003-06-19 | 2021-05-31 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| 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 |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| 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 |
| 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 |
| DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| 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 |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| 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 |
| 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 |
| AU2013249202B2 (en) * | 2012-04-18 | 2018-08-09 | 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 |
| JP6396988B2 (ja) | 2013-03-15 | 2018-09-26 | 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 | 二重グリカン結合性ａａｖベクターのための方法および組成物 |
| KR102234672B1 (ko) | 2013-05-21 | 2021-04-06 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도 |
| WO2015048534A1 (en) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| 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 |
| ES2768763T3 (es) | 2014-03-04 | 2020-06-23 | Univ Florida | Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR |
| 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 |
| 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 |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| WO2017070476A2 (en) | 2015-10-22 | 2017-04-27 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav3 capsid library |
| EP4659816A3 (en) * | 2015-12-14 | 2025-12-31 | The University of North Carolina at Chapel Hill | MODIFIED CAPSID PROTEINS FOR IMPROVED PARVOVIRUS VECTOR DELIVERY |
| AU2018224044B2 (en) | 2017-02-21 | 2024-01-25 | The Uab Research Foundation | Modified AAV capsid proteins and uses thereof |
| EP4010357A2 (en) | 2019-08-09 | 2022-06-15 | University of Florida Research Foundation, Incorporated | Aav capsid variants for targeting human glioblastoma cells |
-
2018
- 2018-02-21 AU AU2018224044A patent/AU2018224044B2/en active Active
- 2018-02-21 CA CA3054131A patent/CA3054131C/en active Active
- 2018-02-21 WO PCT/US2018/019050 patent/WO2018156654A1/en not_active Ceased
- 2018-02-21 CA CA3114549A patent/CA3114549A1/en active Pending
- 2018-02-21 JP JP2019546041A patent/JP7237843B2/ja active Active
- 2018-02-21 EP EP18756857.1A patent/EP3585883A4/en active Pending
-
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 (https=) | ||
| 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 (https=) | ||
| CA3054131C (en) | Modified aav capsid proteins and uses thereof | |
| JP2020028308A5 (https=) | ||
| JP2014239686A5 (https=) | ||
| JP2015501156A5 (https=) | ||
| FI3445773T3 (fi) | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä | |
| 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 (https=) | ||
| IL318395A (en) | Recombinant viral particles with altered tropism, and their uses for targeted introduction of genetic material into human cells | |
| FI2968461T3 (fi) | Fuusioproteiineja, jotka käsittävät pdgf:ää ja vegf:ää sitovia osia, ja menetelmiä niiden käyttämiseksi | |
| WO2012172277A4 (en) | Simian adenovirus and hybrid adenoviral vectors | |
| AR124216A1 (es) | Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen | |
| HRP20241577T1 (hr) | Virioni adeno-pridruženog virusa s varijantom kapside i postupci njihove upotrebe | |
| JP2020511167A5 (https=) | ||
| JPWO2020223236A5 (https=) | ||
| 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 (https=) | ||
| JP2020533968A5 (https=) | ||
| PH12022551878A1 (en) | Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof | |
| CN111718418B (zh) | 一种增强基因编辑的融合蛋白及其应用 |