JPWO2020047268A5 - - Google Patents
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- Publication number
- JPWO2020047268A5 JPWO2020047268A5 JP2021510957A JP2021510957A JPWO2020047268A5 JP WO2020047268 A5 JPWO2020047268 A5 JP WO2020047268A5 JP 2021510957 A JP2021510957 A JP 2021510957A JP 2021510957 A JP2021510957 A JP 2021510957A JP WO2020047268 A5 JPWO2020047268 A5 JP WO2020047268A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- composition
- viral vector
- nucleic acid
- gars
- 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
Links
- 239000013603 viral vector Substances 0.000 claims description 37
- 108020004707 nucleic acids Proteins 0.000 claims description 33
- 102000039446 nucleic acids Human genes 0.000 claims description 33
- 150000007523 nucleic acids Chemical class 0.000 claims description 33
- 108010064209 Phosphoribosylglycinamide formyltransferase Proteins 0.000 claims description 21
- 108010034343 phosphoribosylamine-glycine ligase Proteins 0.000 claims description 21
- 102100036589 Glycine-tRNA ligase Human genes 0.000 claims description 16
- 241000252141 Semionotiformes Species 0.000 claims description 16
- 201000008958 Charcot-Marie-Tooth disease type 2D Diseases 0.000 claims description 12
- 108091070501 miRNA Proteins 0.000 claims description 12
- 239000002679 microRNA Substances 0.000 claims description 12
- 108090000623 proteins and genes Proteins 0.000 claims description 12
- 108091030071 RNAI Proteins 0.000 claims description 10
- 230000009368 gene silencing by RNA Effects 0.000 claims description 10
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 8
- 101000606741 Homo sapiens Phosphoribosylglycinamide formyltransferase Proteins 0.000 claims description 8
- 210000000234 capsid Anatomy 0.000 claims description 8
- 108020004999 messenger RNA Proteins 0.000 claims description 8
- 108091033319 polynucleotide Proteins 0.000 claims description 8
- 102000040430 polynucleotide Human genes 0.000 claims description 8
- 239000002157 polynucleotide Substances 0.000 claims description 8
- 239000003937 drug carrier Substances 0.000 claims description 6
- 239000002773 nucleotide Substances 0.000 claims description 6
- 125000003729 nucleotide group Chemical group 0.000 claims description 6
- 108010051724 Glycine-tRNA Ligase Proteins 0.000 claims description 5
- 102000019220 Glycyl-tRNA synthetases Human genes 0.000 claims description 5
- 208000014720 distal hereditary motor neuropathy Diseases 0.000 claims description 5
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 4
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims description 4
- 241000713666 Lentivirus Species 0.000 claims description 4
- 101000738638 Mus musculus Phosphoribosylglycinamide formyltransferase Proteins 0.000 claims description 4
- 241000700584 Simplexvirus Species 0.000 claims description 4
- 241000700618 Vaccinia virus Species 0.000 claims description 4
- 241000700605 Viruses Species 0.000 claims description 4
- 230000000295 complement effect Effects 0.000 claims description 4
- 210000003061 neural cell Anatomy 0.000 claims description 4
- 210000002569 neuron Anatomy 0.000 claims description 4
- 241000701161 unidentified adenovirus Species 0.000 claims description 4
- 241000701447 unidentified baculovirus Species 0.000 claims description 4
- 241001430294 unidentified retrovirus Species 0.000 claims description 4
- 239000013598 vector Substances 0.000 claims description 4
- 239000003981 vehicle Substances 0.000 claims description 4
- 101001072736 Homo sapiens Glycine-tRNA ligase Proteins 0.000 claims description 2
- 239000003795 chemical substances by application Substances 0.000 claims description 2
- 238000000034 method Methods 0.000 description 10
- 230000035772 mutation Effects 0.000 description 2
- 239000003814 drug Substances 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024107253A JP2024133570A (ja) | 2018-08-29 | 2024-07-03 | 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862724604P | 2018-08-29 | 2018-08-29 | |
| US62/724,604 | 2018-08-29 | ||
| PCT/US2019/048832 WO2020047268A1 (en) | 2018-08-29 | 2019-08-29 | Products and methods for inhibition of expression of mutant gars protein |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024107253A Division JP2024133570A (ja) | 2018-08-29 | 2024-07-03 | 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021534794A JP2021534794A (ja) | 2021-12-16 |
| JP2021534794A5 JP2021534794A5 (https=) | 2022-08-31 |
| JPWO2020047268A5 true JPWO2020047268A5 (https=) | 2022-08-31 |
Family
ID=67953864
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021510957A Pending JP2021534794A (ja) | 2018-08-29 | 2019-08-29 | 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法 |
| JP2024107253A Pending JP2024133570A (ja) | 2018-08-29 | 2024-07-03 | 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024107253A Pending JP2024133570A (ja) | 2018-08-29 | 2024-07-03 | 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20210324417A1 (https=) |
| EP (1) | EP3844284A1 (https=) |
| JP (2) | JP2021534794A (https=) |
| AU (1) | AU2019328270B2 (https=) |
| CA (1) | CA3110665A1 (https=) |
| WO (1) | WO2020047268A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL299762A (en) * | 2020-07-13 | 2023-03-01 | Univ Pennsylvania | Useful preparations for the treatment of Charcot-Marietot disease |
| AU2021390471A1 (en) * | 2020-12-01 | 2023-06-29 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of peripheral myelin protein-22 |
| CA3234702A1 (en) * | 2021-10-07 | 2023-04-13 | Research Institute At Nationwide Children's Hospital | Products and methods for myelin protein zero silencing and treating cmt1b disease |
| IT202200009395A1 (it) * | 2022-05-09 | 2022-08-09 | Giam Pharma Int Sarl | Attività antitumorale del meso-(p-acetamidophenyl)-calix[4]pirrolo mediante l’Inibizione dell’attività enzimatica della proteina Glicil-tRNA Sintasi 1 (GARS1) |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US8258286B2 (en) * | 2007-04-26 | 2012-09-04 | University Of Iowa Research Foundation | Reduction of off-target RNA interference toxicity |
| EP2152874A2 (en) * | 2007-04-26 | 2010-02-17 | University of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| EP2593125B1 (en) * | 2010-07-12 | 2017-11-01 | aTyr Pharma, Inc. | Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glycyl-trna synthetases |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| US11377656B2 (en) * | 2016-03-10 | 2022-07-05 | Novartis Ag | Chemically modified messenger RNA's |
-
2019
- 2019-08-29 JP JP2021510957A patent/JP2021534794A/ja active Pending
- 2019-08-29 WO PCT/US2019/048832 patent/WO2020047268A1/en not_active Ceased
- 2019-08-29 EP EP19768951.6A patent/EP3844284A1/en active Pending
- 2019-08-29 AU AU2019328270A patent/AU2019328270B2/en active Active
- 2019-08-29 US US17/271,377 patent/US20210324417A1/en active Pending
- 2019-08-29 CA CA3110665A patent/CA3110665A1/en active Pending
-
2024
- 2024-07-03 JP JP2024107253A patent/JP2024133570A/ja active Pending