JPWO2020132455A5 - - Google Patents
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- Publication number
- JPWO2020132455A5 JPWO2020132455A5 JP2021535718A JP2021535718A JPWO2020132455A5 JP WO2020132455 A5 JPWO2020132455 A5 JP WO2020132455A5 JP 2021535718 A JP2021535718 A JP 2021535718A JP 2021535718 A JP2021535718 A JP 2021535718A JP WO2020132455 A5 JPWO2020132455 A5 JP WO2020132455A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- pharmaceutical composition
- sequence
- mirna
- mirna target
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 24
- 229920001239 microRNA Polymers 0.000 claims 22
- 108020004388 MicroRNAs Proteins 0.000 claims 21
- 239000002679 microRNA Substances 0.000 claims 21
- 229920001405 Coding region Polymers 0.000 claims 7
- 210000003594 Ganglia, Spinal Anatomy 0.000 claims 5
- 210000004027 cells Anatomy 0.000 claims 5
- 239000003814 drug Substances 0.000 claims 5
- 239000002773 nucleotide Substances 0.000 claims 5
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- 229920001320 Leader sequence (mRNA) Polymers 0.000 claims 4
- 229920000401 Three prime untranslated region Polymers 0.000 claims 4
- 230000003612 virological Effects 0.000 claims 4
- 210000003169 Central Nervous System Anatomy 0.000 claims 3
- 238000007913 intrathecal administration Methods 0.000 claims 3
- 238000004519 manufacturing process Methods 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 150000007523 nucleic acids Chemical group 0.000 claims 3
- 210000000234 Capsid Anatomy 0.000 claims 2
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 2
- 230000000977 initiatory Effects 0.000 claims 2
- 238000000185 intracerebroventricular Methods 0.000 claims 2
- 238000001990 intravenous administration Methods 0.000 claims 2
- 210000002569 neurons Anatomy 0.000 claims 2
- 230000001105 regulatory Effects 0.000 claims 2
- 229920000160 (ribonucleotides)n+m Polymers 0.000 claims 1
- 210000001130 Astrocytes Anatomy 0.000 claims 1
- 229920001661 Chitosan Polymers 0.000 claims 1
- 210000001153 Interneurons Anatomy 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 108020004999 Messenger RNA Proteins 0.000 claims 1
- 210000000274 Microglia Anatomy 0.000 claims 1
- 229920002957 Naked DNA Polymers 0.000 claims 1
- 210000004248 Oligodendroglia Anatomy 0.000 claims 1
- 241000125945 Protoparvovirus Species 0.000 claims 1
- 210000000449 Purkinje Cells Anatomy 0.000 claims 1
- 210000000278 Spinal Cord Anatomy 0.000 claims 1
- 230000003376 axonal Effects 0.000 claims 1
- 210000003008 brain-resident macrophage Anatomy 0.000 claims 1
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims 1
- 230000002708 enhancing Effects 0.000 claims 1
- 239000012537 formulation buffer Substances 0.000 claims 1
- 238000001476 gene delivery Methods 0.000 claims 1
- 238000001415 gene therapy Methods 0.000 claims 1
- 210000004565 granule cell Anatomy 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 239000010954 inorganic particle Substances 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 229920002106 messenger RNA Polymers 0.000 claims 1
- 230000000051 modifying Effects 0.000 claims 1
- 230000004770 neurodegeneration Effects 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 210000001176 projection neuron Anatomy 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862783956P | 2018-12-21 | 2018-12-21 | |
| US62/783,956 | 2018-12-21 | ||
| US201962924970P | 2019-10-23 | 2019-10-23 | |
| US62/924,970 | 2019-10-23 | ||
| US201962934915P | 2019-11-13 | 2019-11-13 | |
| US62/934,915 | 2019-11-13 | ||
| PCT/US2019/067872 WO2020132455A1 (fr) | 2018-12-21 | 2019-12-20 | Compositions pour la réduction spécifique de drg de l'expression de transgène |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022517174A JP2022517174A (ja) | 2022-03-07 |
| JPWO2020132455A5 true JPWO2020132455A5 (fr) | 2022-12-27 |
Family
ID=71100588
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021535718A Pending JP2022517174A (ja) | 2018-12-21 | 2019-12-20 | 導入遺伝子発現のｄｒｇ特異的低減のための組成物 |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US20210077553A1 (fr) |
| EP (1) | EP3908326A4 (fr) |
| JP (1) | JP2022517174A (fr) |
| KR (1) | KR20210107037A (fr) |
| CN (1) | CN113646005A (fr) |
| AU (1) | AU2019401314A1 (fr) |
| BR (1) | BR112021011143A2 (fr) |
| CA (1) | CA3123600A1 (fr) |
| CL (1) | CL2021001624A1 (fr) |
| CO (1) | CO2021008538A2 (fr) |
| IL (1) | IL284185A (fr) |
| JO (1) | JOP20210160A1 (fr) |
| MX (1) | MX2021007600A (fr) |
| PE (1) | PE20211581A1 (fr) |
| PH (1) | PH12021551341A1 (fr) |
| SG (1) | SG11202105907QA (fr) |
| WO (1) | WO2020132455A1 (fr) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112022020753A2 (pt) | 2020-04-15 | 2022-12-20 | Voyager Therapeutics Inc | Compostos de ligação a tau |
| JP2023526310A (ja) * | 2020-05-12 | 2023-06-21 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 導入遺伝子発現のｄｒｇ特異的低減のための組成物 |
| EP4149955A1 (fr) | 2020-05-13 | 2023-03-22 | Voyager Therapeutics, Inc. | Redirection de tropisme de capsides de vaa |
| WO2021247995A2 (fr) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de la douleur neuropathique |
| WO2021257668A1 (fr) | 2020-06-17 | 2021-12-23 | The Trustees Of The University Of Pennsylvania | Compositions et méthodes pour le traitement de patients de thérapie génique |
| IL299762A (en) | 2020-07-13 | 2023-03-01 | Univ Pennsylvania | Useful preparations for the treatment of Charcot-Marietot disease |
| MX2023000815A (es) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta. |
| WO2022026410A2 (fr) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions et méthodes pour le traitement de la maladie de niemann-pick de type c1 |
| CA3195553A1 (fr) | 2020-10-18 | 2022-04-21 | Qiang Wang | Vecteur de virus adeno-associe(aav) ameliore et ses utilisations |
| WO2022094078A1 (fr) | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions utiles dans le traitement du syndrome de rett |
| WO2022119890A1 (fr) | 2020-12-01 | 2022-06-09 | The Trustees Of The University Of Pennsylvania | Compositions et leurs utilisations pour le traitement du syndrome d'angelman |
| WO2022187548A1 (fr) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Expression régulée de protéines virales |
| US20240175050A1 (en) * | 2021-03-19 | 2024-05-30 | Mayo Foundation For Medical Education And Research | Methods and materials for treating propionic acidemia |
| US20230034817A1 (en) | 2021-04-12 | 2023-02-02 | Affinia Therapeutics Inc. | Recombinant aav for treatment of neural disease |
| WO2023113806A1 (fr) | 2021-12-16 | 2023-06-22 | Affinia Therapeutics, Inc. | Vaa recombinant pour le traitement d'une maladie neurale |
| AR125406A1 (es) | 2021-04-23 | 2023-07-12 | Univ Pennsylvania | Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen |
| AR125467A1 (es) | 2021-04-27 | 2023-07-19 | Univ Pennsylvania | Cápsides de virus adenoasociados derivados de porcinos y usos de los estos |
| WO2023044483A2 (fr) | 2021-09-20 | 2023-03-23 | Voyager Therapeutics, Inc. | Compositions et procédés pour le traitement du cancer positif her2 |
| TW202325845A (zh) | 2021-10-02 | 2023-07-01 | 賓州大學委員會 | 新穎aav衣殼及含其之組成物 |
| IL312241A (en) * | 2021-10-18 | 2024-06-01 | Univ Pennsylvania | Compositions useful in the treatment of CDKL5 deficiency disorder (CDD) |
| WO2023087019A2 (fr) * | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions pour la réduction spécifique de drg de l'expression de transgènes |
| WO2023091949A2 (fr) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de troubles neurologiques liés à un déficit en bêta glucosylcéramidase |
| WO2023092004A1 (fr) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions et méthodes pour le traitement de troubles liés à tau |
| WO2023091948A1 (fr) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Variants de capsides d'aav et leurs utilisations |
| WO2023133574A1 (fr) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions et méthodes utiles pour le traitement de troubles médiés par c9orf72 |
| WO2023147374A2 (fr) | 2022-01-25 | 2023-08-03 | Voyager Therapeutics, Inc. | Système d'expression de baculovirus |
| WO2023154693A1 (fr) | 2022-02-08 | 2023-08-17 | Voyager Therapeutics, Inc. | Variants de capside d'aav et leurs utilisations |
| WO2023196892A1 (fr) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Immunisation passive avec des anticorps neutralisants anti-aav pour empêcher la transduction hors cible de vecteurs aav administrés par voie intrathécale |
| WO2023196893A1 (fr) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions et méthodes de traitement d'un cancer du sein métastatique her2 positif et d'autres cancers |
| WO2023220695A2 (fr) | 2022-05-13 | 2023-11-16 | Voyager Therapeutics, Inc. | Compositions et procédés pour le traitement du cancer her2 positif |
| WO2023235791A1 (fr) | 2022-06-02 | 2023-12-07 | Voyager Therapeutics, Inc. | Variants de capside de vaa et leurs utilisations |
| WO2023240236A1 (fr) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale |
| WO2023250388A1 (fr) | 2022-06-22 | 2023-12-28 | Voyager Therapeutics, Inc. | Composés se liant à la protéine tau |
| WO2024006741A1 (fr) | 2022-06-28 | 2024-01-04 | Voyager Therapeutics, Inc. | Variants de capside d'aav et leurs utilisations |
| WO2024011112A1 (fr) | 2022-07-06 | 2024-01-11 | Voyager Therapeutics, Inc. | Variants de capside d'aav et leurs utilisations |
| WO2024008950A1 (fr) * | 2022-07-08 | 2024-01-11 | Ospedale San Raffaele S.R.L. | Cassettes transgéniques |
| WO2024030976A2 (fr) | 2022-08-03 | 2024-02-08 | Voyager Therapeutics, Inc. | Compositions et procédés permettant le franchissement de la barrière hémato-encéphalique |
| WO2024059739A1 (fr) | 2022-09-15 | 2024-03-21 | Voyager Therapeutics, Inc. | Composés de liaison à la protéine tau |
| WO2024073310A2 (fr) * | 2022-09-26 | 2024-04-04 | Encoded Therapeutics, Inc. | Éléments pour le dé-ciblage de l'expression génique dans le ganglion de la racine dorsale et/ou le foie |
| WO2024130067A2 (fr) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Vecteurs mutants aav recombinants avec motifs de ciblage spécifiques des muscles cardiaque et squelettique et compositions les contenant |
| WO2024130070A2 (fr) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Capsides aav recombinantes avec motifs de ciblage spécifiques du muscle cardiaque et squelettique et leurs utilisations |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006069584A2 (fr) * | 2004-12-29 | 2006-07-06 | Exiqon A/S | Nouvelles compositions d'oligonucleotides et sequences de sondes utiles pour la detection et l'analyse de microarn et de leurs marn cibles |
| KR20130114758A (ko) * | 2005-05-27 | 2013-10-17 | 오스페달레 산 라파엘 에스.알.엘. | Ｍｉ-ｒｎａ를 포함하는 유전자 벡터 |
| EP1966390A1 (fr) * | 2005-12-29 | 2008-09-10 | Exiqon A/S | Detection de l'origine tissulaire d'un cancer |
| DK2561073T3 (en) * | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
-
2019
- 2019-12-20 JO JOP/2021/0160A patent/JOP20210160A1/ar unknown
- 2019-12-20 AU AU2019401314A patent/AU2019401314A1/en active Pending
- 2019-12-20 KR KR1020217021804A patent/KR20210107037A/ko unknown
- 2019-12-20 SG SG11202105907QA patent/SG11202105907QA/en unknown
- 2019-12-20 EP EP19897715.9A patent/EP3908326A4/fr active Pending
- 2019-12-20 CA CA3123600A patent/CA3123600A1/fr active Pending
- 2019-12-20 PE PE2021000908A patent/PE20211581A1/es unknown
- 2019-12-20 CN CN201980092720.6A patent/CN113646005A/zh active Pending
- 2019-12-20 JP JP2021535718A patent/JP2022517174A/ja active Pending
- 2019-12-20 BR BR112021011143A patent/BR112021011143A2/pt unknown
- 2019-12-20 WO PCT/US2019/067872 patent/WO2020132455A1/fr active Application Filing
- 2019-12-20 MX MX2021007600A patent/MX2021007600A/es unknown
-
2020
- 2020-11-13 US US17/097,997 patent/US20210077553A1/en active Pending
-
2021
- 2021-06-07 PH PH12021551341A patent/PH12021551341A1/en unknown
- 2021-06-17 CL CL2021001624A patent/CL2021001624A1/es unknown
- 2021-06-20 IL IL284185A patent/IL284185A/en unknown
- 2021-06-29 CO CONC2021/0008538A patent/CO2021008538A2/es unknown
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