JP2023545433A5 - - Google Patents
Info
- Publication number
- JP2023545433A5 JP2023545433A5 JP2023521706A JP2023521706A JP2023545433A5 JP 2023545433 A5 JP2023545433 A5 JP 2023545433A5 JP 2023521706 A JP2023521706 A JP 2023521706A JP 2023521706 A JP2023521706 A JP 2023521706A JP 2023545433 A5 JP2023545433 A5 JP 2023545433A5
- Authority
- JP
- Japan
- Prior art keywords
- expression cassette
- sequence
- hgla
- raav
- seq
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063089850P | 2020-10-09 | 2020-10-09 | |
| US63/089,850 | 2020-10-09 | ||
| US202163146286P | 2021-02-05 | 2021-02-05 | |
| US63/146,286 | 2021-02-05 | ||
| US202163186092P | 2021-05-08 | 2021-05-08 | |
| US63/186,092 | 2021-05-08 | ||
| PCT/US2021/054145 WO2022076803A1 (en) | 2020-10-09 | 2021-10-08 | Compositions and methods for treatment of fabry disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023545433A JP2023545433A (ja) | 2023-10-30 |
| JP2023545433A5 true JP2023545433A5 (enExample) | 2024-10-10 |
Family
ID=81125461
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023521706A Pending JP2023545433A (ja) | 2020-10-09 | 2021-10-08 | ファブリー病の治療のための組成物及び方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230365955A1 (enExample) |
| EP (1) | EP4225906A1 (enExample) |
| JP (1) | JP2023545433A (enExample) |
| KR (1) | KR20230118075A (enExample) |
| AU (1) | AU2021356684A1 (enExample) |
| CA (1) | CA3193833A1 (enExample) |
| TW (1) | TW202229560A (enExample) |
| WO (1) | WO2022076803A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR124981A1 (es) * | 2021-02-26 | 2023-05-24 | Takeda Pharmaceuticals Co | Composición y métodos para el tratamiento de la enfermedad de fabry |
| US20240408157A1 (en) * | 2021-12-02 | 2024-12-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| CA3261151A1 (en) * | 2022-07-08 | 2024-01-11 | Cornell University | PHARMACEUTICAL METHODS AND COMPOSITIONS FOR THE TREATMENT AND PREVENTION OF ALZHEIMER'S DISEASE |
| KR20250052387A (ko) * | 2022-08-25 | 2025-04-18 | 다케다 야쿠힌 고교 가부시키가이샤 | 파브리병의 치료에서의 사용을 위한 조성물 |
| 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 |
| WO2025007046A1 (en) | 2023-06-29 | 2025-01-02 | The Trustees Of The University Of Pennsylvania | Mutant aav with central nervous system targeting motifs and compositions containing same |
| KR20260114566A (ko) | 2023-11-14 | 2026-07-22 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도 |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
| US6136597A (en) | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| US7419829B2 (en) | 2000-10-06 | 2008-09-02 | Oxford Biomedica (Uk) Limited | Vector system |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| WO2005012877A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for antibody engineering |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2010053572A2 (en) | 2008-11-07 | 2010-05-14 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| JP5868319B2 (ja) | 2009-09-03 | 2016-02-24 | エボニック レーム ゲゼルシャフト ミット ベシュレンクテル ハフツングＥｖｏｎｉｋ Ｒｏｅｈｍ ＧｍｂＨ | １つ以上の生物学的活性物質、調合補助物質および磁化可能粒子を包含する経口剤形 |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| KR20220025112A (ko) | 2011-06-08 | 2022-03-03 | 샤이어 휴먼 지네틱 테라피즈 인크. | Mrna 전달을 위한 지질 나노입자 조성물 및 방법 |
| EA037448B1 (ru) | 2012-06-08 | 2021-03-30 | Этрис Гмбх | Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция |
| BR112015021036A2 (pt) | 2013-03-15 | 2017-10-10 | Univ Pennsylvania | composições e métodos para tratar mps1 |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| WO2016049230A1 (en) | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US20170051267A1 (en) * | 2015-08-20 | 2017-02-23 | Research Foundation Of The City University Of New York | Novel alpha-galactosidase a derivatives |
| EP3387138B2 (en) | 2015-12-11 | 2026-05-27 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| KR102617502B1 (ko) | 2016-09-12 | 2023-12-26 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
| RS65241B1 (sr) | 2017-02-28 | 2024-03-29 | Univ Pennsylvania | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
| IL319002A (en) | 2018-06-14 | 2025-04-01 | Regenxbio Inc | Anion exchange chromatography for recombinant AAV production |
| JP7577650B2 (ja) * | 2018-10-10 | 2024-11-05 | アミカス セラピューティックス インコーポレイテッド | ジスルフィド結合により安定したポリペプチド組成物及びその使用方法 |
| MX2021007600A (es) * | 2018-12-21 | 2021-08-11 | Univ Pennsylvania | Composiciones para la reduccion de la expresion transgenica especifica de drg. |
| CN113423419A (zh) * | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
| WO2021158915A1 (en) | 2020-02-06 | 2021-08-12 | Precision Biosciences, Inc. | Recombinant adeno-associated virus compositions and methods for producing and using the same |
| EP3868886A1 (en) | 2020-02-21 | 2021-08-25 | Bia Separations D.O.O. | A method for separation or depletion of empty aav capsids from full aav capsids |
| CA3175905A1 (en) * | 2020-04-27 | 2021-11-04 | David H. Kirn | Codon optimized gla genes and uses thereof |
-
2021
- 2021-10-08 AU AU2021356684A patent/AU2021356684A1/en not_active Abandoned
- 2021-10-08 CA CA3193833A patent/CA3193833A1/en active Pending
- 2021-10-08 US US18/246,872 patent/US20230365955A1/en active Pending
- 2021-10-08 JP JP2023521706A patent/JP2023545433A/ja active Pending
- 2021-10-08 WO PCT/US2021/054145 patent/WO2022076803A1/en not_active Ceased
- 2021-10-08 EP EP21805710.7A patent/EP4225906A1/en active Pending
- 2021-10-08 TW TW110137528A patent/TW202229560A/zh unknown
- 2021-10-08 KR KR1020237015206A patent/KR20230118075A/ko not_active Withdrawn
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