JPWO2022015715A5 - - Google Patents
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- Publication number
- JPWO2022015715A5 JPWO2022015715A5 JP2023502675A JP2023502675A JPWO2022015715A5 JP WO2022015715 A5 JPWO2022015715 A5 JP WO2022015715A5 JP 2023502675 A JP2023502675 A JP 2023502675A JP 2023502675 A JP2023502675 A JP 2023502675A JP WO2022015715 A5 JPWO2022015715 A5 JP WO2022015715A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- mirna
- coding sequence
- sequence
- vector
- 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063051336P | 2020-07-13 | 2020-07-13 | |
| US63/051,336 | 2020-07-13 | ||
| US202163173045P | 2021-04-09 | 2021-04-09 | |
| US63/173,045 | 2021-04-09 | ||
| PCT/US2021/041406 WO2022015715A1 (en) | 2020-07-13 | 2021-07-13 | Compositions useful for treatment of charcot-marie-tooth disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023534037A JP2023534037A (ja) | 2023-08-07 |
| JP2023534037A5 JP2023534037A5 (https=) | 2024-07-23 |
| JPWO2022015715A5 true JPWO2022015715A5 (https=) | 2024-07-23 |
Family
ID=77317406
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023502675A Pending JP2023534037A (ja) | 2020-07-13 | 2021-07-13 | シャルコー・マリー・トゥース病の治療に有用な組成物 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20230270884A1 (https=) |
| EP (1) | EP4179097A1 (https=) |
| JP (1) | JP2023534037A (https=) |
| KR (1) | KR20230038503A (https=) |
| CN (1) | CN116438311A (https=) |
| AU (1) | AU2021309645A1 (https=) |
| BR (1) | BR112023000578A2 (https=) |
| CA (1) | CA3185281A1 (https=) |
| CO (1) | CO2023001500A2 (https=) |
| IL (1) | IL299762A (https=) |
| MX (1) | MX2023000658A (https=) |
| TW (1) | TW202216244A (https=) |
| WO (1) | WO2022015715A1 (https=) |
| ZA (1) | ZA202300505B (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201907882D0 (en) | 2019-06-03 | 2019-07-17 | Cyprus Foundation For Muscular Dystrophy Res | Methods |
| WO2023049846A1 (en) * | 2021-09-24 | 2023-03-30 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| KR20250135916A (ko) | 2022-12-17 | 2025-09-15 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물 |
| WO2024155966A1 (en) * | 2023-01-20 | 2024-07-25 | Loma Linda University Health | Methods and compositions for treatment of niemann-pick disease type c and charcot-marie-tooth disease |
| EP4731642A1 (en) | 2023-06-21 | 2026-04-29 | Fundació Institut de Recerca Biomèdica (IRB Barcelona) | Variants of mfn2 and their use in the treatment/prevention of diseases associated with alterations in endoplasmatic reticulum function |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| 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 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| ES2646630T3 (es) | 2008-11-07 | 2017-12-14 | Massachusetts Institute Of Technology | Lipidoides aminoalcohólicos y usos de los mismos |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2558074B1 (en) | 2010-04-08 | 2018-06-06 | The Trustees of Princeton University | Preparation of lipid nanoparticles |
| WO2012075040A2 (en) | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| PL3586861T3 (pl) | 2011-06-08 | 2022-05-23 | Translate Bio, Inc. | Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EA037448B1 (ru) | 2012-06-08 | 2021-03-30 | Этрис Гмбх | Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция |
| EP4331620A3 (en) | 2012-12-07 | 2024-12-04 | Translate Bio, Inc. | Lipidic nanoparticles for mrna delivery |
| US9593077B2 (en) | 2013-11-18 | 2017-03-14 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | 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 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| WO2018168961A1 (ja) | 2017-03-16 | 2018-09-20 | 株式会社デンソー | 自己位置推定装置 |
| MX2020008932A (es) | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| IL276859B2 (en) | 2018-02-27 | 2025-12-01 | Univ Pennsylvania | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
| JP2021534794A (ja) * | 2018-08-29 | 2021-12-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 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 |
| WO2020132455A1 (en) | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
-
2021
- 2021-07-13 IL IL299762A patent/IL299762A/en unknown
- 2021-07-13 BR BR112023000578A patent/BR112023000578A2/pt unknown
- 2021-07-13 CN CN202180062652.6A patent/CN116438311A/zh active Pending
- 2021-07-13 TW TW110125641A patent/TW202216244A/zh unknown
- 2021-07-13 CA CA3185281A patent/CA3185281A1/en active Pending
- 2021-07-13 MX MX2023000658A patent/MX2023000658A/es unknown
- 2021-07-13 AU AU2021309645A patent/AU2021309645A1/en active Pending
- 2021-07-13 EP EP21754870.0A patent/EP4179097A1/en active Pending
- 2021-07-13 KR KR1020237003861A patent/KR20230038503A/ko active Pending
- 2021-07-13 US US18/005,504 patent/US20230270884A1/en active Pending
- 2021-07-13 WO PCT/US2021/041406 patent/WO2022015715A1/en not_active Ceased
- 2021-07-13 JP JP2023502675A patent/JP2023534037A/ja active Pending
-
2023
- 2023-01-11 ZA ZA2023/00505A patent/ZA202300505B/en unknown
- 2023-02-10 CO CONC2023/0001500A patent/CO2023001500A2/es unknown
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