JP2023534037A5 - - Google Patents
Info
- Publication number
- JP2023534037A5 JP2023534037A5 JP2023502675A JP2023502675A JP2023534037A5 JP 2023534037 A5 JP2023534037 A5 JP 2023534037A5 JP 2023502675 A JP2023502675 A JP 2023502675A JP 2023502675 A JP2023502675 A JP 2023502675A JP 2023534037 A5 JP2023534037 A5 JP 2023534037A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- mirna
- vector
- hmfn2
- coding sequence
- 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 (2)
| Publication Number | Publication Date |
|---|---|
| JP2023534037A JP2023534037A (ja) | 2023-08-07 |
| JP2023534037A5 true JP2023534037A5 (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 |
| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| 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 |
| WO2024261139A1 (en) | 2023-06-21 | 2024-12-26 | 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 |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| 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 |
| 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130037977A1 (en) | 2010-04-08 | 2013-02-14 | Paul A. Burke | Preparation of Lipid Nanoparticles |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| EP4331620A3 (en) | 2012-12-07 | 2024-12-04 | Translate Bio, Inc. | Lipidic nanoparticles for mrna delivery |
| AU2014348212C1 (en) | 2013-11-18 | 2018-11-29 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | 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 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| WO2018168961A1 (ja) | 2017-03-16 | 2018-09-20 | 株式会社デンソー | 自己位置推定装置 |
| EP3768695A4 (en) | 2018-02-27 | 2022-04-06 | The Trustees of the University of Pennsylvania | NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
| 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 |
| WO2020047268A1 (en) * | 2018-08-29 | 2020-03-05 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of mutant gars protein |
| 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 WO PCT/US2021/041406 patent/WO2022015715A1/en not_active Ceased
- 2021-07-13 BR BR112023000578A patent/BR112023000578A2/pt unknown
- 2021-07-13 EP EP21754870.0A patent/EP4179097A1/en active Pending
- 2021-07-13 CA CA3185281A patent/CA3185281A1/en active Pending
- 2021-07-13 KR KR1020237003861A patent/KR20230038503A/ko active Pending
- 2021-07-13 JP JP2023502675A patent/JP2023534037A/ja active Pending
- 2021-07-13 MX MX2023000658A patent/MX2023000658A/es unknown
- 2021-07-13 AU AU2021309645A patent/AU2021309645A1/en active Pending
- 2021-07-13 CN CN202180062652.6A patent/CN116438311A/zh active Pending
- 2021-07-13 TW TW110125641A patent/TW202216244A/zh unknown
- 2021-07-13 US US18/005,504 patent/US20230270884A1/en 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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