JP2022529782A5 - - Google Patents
Info
- Publication number
- JP2022529782A5 JP2022529782A5 JP2021562000A JP2021562000A JP2022529782A5 JP 2022529782 A5 JP2022529782 A5 JP 2022529782A5 JP 2021562000 A JP2021562000 A JP 2021562000A JP 2021562000 A JP2021562000 A JP 2021562000A JP 2022529782 A5 JP2022529782 A5 JP 2022529782A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- isolated nucleic
- vector
- raav
- promoter
- 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.)
- Ceased
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962836493P | 2019-04-19 | 2019-04-19 | |
| US62/836,493 | 2019-04-19 | ||
| PCT/US2020/028504 WO2020214809A2 (en) | 2019-04-19 | 2020-04-16 | Gene therapies for stargardt disease (abca4) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022529782A JP2022529782A (ja) | 2022-06-24 |
| JP2022529782A5 true JP2022529782A5 (enExample) | 2023-04-25 |
Family
ID=72838359
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021562000A Ceased JP2022529782A (ja) | 2019-04-19 | 2020-04-16 | シュタルガルト病（ａｂｃａ４）の遺伝子治療 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US12516097B2 (enExample) |
| EP (1) | EP3955973A4 (enExample) |
| JP (1) | JP2022529782A (enExample) |
| CN (1) | CN114126665B (enExample) |
| AU (1) | AU2020257392A1 (enExample) |
| CA (1) | CA3137135A1 (enExample) |
| WO (1) | WO2020214809A2 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3137135A1 (en) | 2019-04-19 | 2020-10-22 | University Of Massachusetts | Gene therapies for stargardt disease (abca4) |
| KR20220128607A (ko) | 2019-09-18 | 2022-09-21 | 인터갈락틱 테라퓨틱스, 인크. | 합성 dna 벡터 및 사용 방법 |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| CA3205822A1 (en) * | 2021-01-22 | 2022-07-28 | Marcelo M. NOCIARI | Compositions and methods for the identification of compounds that protect against lipofuscin cytotoxicity |
| CA3211687A1 (en) * | 2021-03-19 | 2022-09-22 | Ozan ALKAN | Non-viral dna vectors and uses thereof for expressing pfic therapeutics |
| WO2022232041A1 (en) * | 2021-04-26 | 2022-11-03 | University Of Massachusetts | Gene therapies for stargardt disease (abca4) |
| US20240425877A1 (en) * | 2021-05-07 | 2024-12-26 | Ucl Business Ltd | Abca4 genome editing |
| CN115074369B (zh) * | 2022-06-24 | 2023-10-20 | 成都金唯科生物科技有限公司 | 表达全长abca4基因的腺相关病毒载体及应用 |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
| JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
| JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
| 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. |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US20090214478A1 (en) | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| WO2009134418A2 (en) | 2008-04-30 | 2009-11-05 | Fox Chase Cancer Center | Assay for identifying agents that modulate epigenetic silencing, and agents identified thereby |
| EP3800191B1 (en) * | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| CA3005474A1 (en) * | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| JP7659291B2 (ja) * | 2016-12-13 | 2025-04-09 | スティヒティング ラドバウド ユニヴェルシテール メディッシュ セントルム | スターガルト病の治療のためのアンチセンスオリゴヌクレオチド |
| CA3054735A1 (en) | 2017-02-27 | 2018-08-30 | Case Western Reserve University | Visual cycle modulators |
| CA3137135A1 (en) | 2019-04-19 | 2020-10-22 | University Of Massachusetts | Gene therapies for stargardt disease (abca4) |
| WO2022232041A1 (en) | 2021-04-26 | 2022-11-03 | University Of Massachusetts | Gene therapies for stargardt disease (abca4) |
-
2020
- 2020-04-16 CA CA3137135A patent/CA3137135A1/en active Pending
- 2020-04-16 CN CN202080044221.2A patent/CN114126665B/zh active Active
- 2020-04-16 AU AU2020257392A patent/AU2020257392A1/en not_active Abandoned
- 2020-04-16 EP EP20791568.7A patent/EP3955973A4/en active Pending
- 2020-04-16 US US17/604,556 patent/US12516097B2/en active Active
- 2020-04-16 JP JP2021562000A patent/JP2022529782A/ja not_active Ceased
- 2020-04-16 WO PCT/US2020/028504 patent/WO2020214809A2/en not_active Ceased
-
2025
- 2025-10-02 US US19/347,786 patent/US20260022154A1/en active Pending
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