JP2021531752A5 - - Google Patents
Info
- Publication number
- JP2021531752A5 JP2021531752A5 JP2021500664A JP2021500664A JP2021531752A5 JP 2021531752 A5 JP2021531752 A5 JP 2021531752A5 JP 2021500664 A JP2021500664 A JP 2021500664A JP 2021500664 A JP2021500664 A JP 2021500664A JP 2021531752 A5 JP2021531752 A5 JP 2021531752A5
- Authority
- JP
- Japan
- Prior art keywords
- identity
- sequence
- seq
- lamp
- expression
- 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024114749A JP2024150588A (ja) | 2018-07-12 | 2024-07-18 | ダノン病治療用遺伝子療法ベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862697302P | 2018-07-12 | 2018-07-12 | |
| US62/697,302 | 2018-07-12 | ||
| PCT/US2019/041465 WO2020014523A1 (en) | 2018-07-12 | 2019-07-11 | Gene therapy vectors for treatment of danon disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024114749A Division JP2024150588A (ja) | 2018-07-12 | 2024-07-18 | ダノン病治療用遺伝子療法ベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021531752A JP2021531752A (ja) | 2021-11-25 |
| JP2021531752A5 true JP2021531752A5 (enExample) | 2022-07-14 |
| JP7586810B2 JP7586810B2 (ja) | 2024-11-19 |
Family
ID=69141704
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021500664A Active JP7586810B2 (ja) | 2018-07-12 | 2019-07-11 | ダノン病治療用遺伝子療法ベクター |
| JP2024114749A Pending JP2024150588A (ja) | 2018-07-12 | 2024-07-18 | ダノン病治療用遺伝子療法ベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024114749A Pending JP2024150588A (ja) | 2018-07-12 | 2024-07-18 | ダノン病治療用遺伝子療法ベクター |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US10703797B2 (enExample) |
| EP (1) | EP3820537A4 (enExample) |
| JP (2) | JP7586810B2 (enExample) |
| KR (1) | KR102918544B1 (enExample) |
| CN (1) | CN112512596B (enExample) |
| AU (1) | AU2019299970B2 (enExample) |
| BR (1) | BR112021000509A2 (enExample) |
| CA (1) | CA3106010A1 (enExample) |
| IL (1) | IL279918A (enExample) |
| MX (1) | MX2021000443A (enExample) |
| SG (1) | SG11202100022SA (enExample) |
| WO (1) | WO2020014523A1 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102673203B1 (ko) | 2016-04-20 | 2024-06-10 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | Pklr의 유전자 발현 증강을 위한 조성물 및 방법 |
| AU2018352583B2 (en) | 2017-10-16 | 2025-04-17 | Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. | Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency |
| US12539316B2 (en) | 2018-04-11 | 2026-02-03 | Spacecraft Seven, Llc | Compositions and methods for stem cell transplant |
| IL278213B2 (en) | 2018-04-27 | 2026-04-01 | Rocket Pharmaceuticals Ltd | A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd) |
| IL280465B2 (en) | 2018-07-30 | 2025-03-01 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Methods for gene modification of hematopoietic cells |
| US20220143215A1 (en) * | 2019-02-12 | 2022-05-12 | Spacecraft Seven, Llc | Gene therapy vectors for treatment of danon disease |
| WO2021142267A1 (en) | 2020-01-08 | 2021-07-15 | Scarpmap Llc | Methods and computing system for processing ultrasound image to determine health of subdermal tissue |
| WO2022017630A1 (en) * | 2020-07-23 | 2022-01-27 | Ucl Business Ltd | GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF |
| WO2022026516A1 (en) * | 2020-07-29 | 2022-02-03 | The Board Of Regents Of The University Of Texas System | Transgene cassettes, aav vectors, and aav viral vectors for expression of human codon-optimized cstb |
| WO2022032226A1 (en) | 2020-08-07 | 2022-02-10 | Spacecraft Seven, Llc | Plakophilin-2 (pkp2) gene therapy using aav vector |
| EP4217010A4 (en) * | 2020-09-28 | 2025-01-08 | Tacit Therapeutics, Inc. | TRANS-SPLICING SYSTEM FOR TISSUE-SPECIFIC REPLACEMENT OF RNA SEQUENCES |
| JP2023548746A (ja) * | 2020-10-13 | 2023-11-21 | クリヤ セラピューティクス インコーポレイテッド | サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用 |
| WO2022087321A1 (en) * | 2020-10-21 | 2022-04-28 | Duke University | Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy |
| MX2023005901A (es) * | 2020-11-20 | 2023-08-29 | Baylor College Medicine | Supresión de señalización hippo en el nicho de células madre promueve la regeneración de músculo esquelético. |
| CN116887868A (zh) * | 2020-12-07 | 2023-10-13 | 太空飞船七有限责任公司 | 达农病的治疗 |
| BR112023021037A2 (pt) * | 2021-04-12 | 2023-12-19 | Univ California | Terapia gênica para cardiomiopatia ventricular direita arritmogênica |
| TW202317768A (zh) | 2021-07-08 | 2023-05-01 | 美商特納亞治療股份有限公司 | 用於基因療法的優化表現卡匣 |
| JP2024533163A (ja) | 2021-09-03 | 2024-09-12 | タシト・セラピューティクス・インコーポレーテッド | スプライソソーム構成要素のリクルートを介したｒｎａ編集 |
| KR102577502B1 (ko) * | 2021-09-08 | 2023-09-13 | 주식회사 꿈랩 | 생체분자의 안정적 발현 및 전달을 위한 플라스미드 플랫폼 |
| WO2023038479A1 (ko) * | 2021-09-08 | 2023-03-16 | 주식회사 꿈랩 | 생체분자의 안정적 발현 및 전달을 위한 플라스미드 플랫폼 |
| WO2025217543A1 (en) * | 2024-04-12 | 2025-10-16 | AskBio Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding lamp2b |
Family Cites Families (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US4861719A (en) | 1986-04-25 | 1989-08-29 | Fred Hutchinson Cancer Research Center | DNA constructs for retrovirus packaging cell lines |
| US5278056A (en) | 1988-02-05 | 1994-01-11 | The Trustees Of Columbia University In The City Of New York | Retroviral packaging cell lines and process of using same |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| AU8200191A (en) | 1990-07-09 | 1992-02-04 | United States of America, as represented by the Secretary, U.S. Department of Commerce, The | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| ATE237694T1 (de) | 1991-08-20 | 2003-05-15 | Us Gov Health & Human Serv | Adenovirus vermittelter gentransfer in den gastrointestinaltrakt |
| JP3866760B2 (ja) | 1993-02-22 | 2007-01-10 | ザ ロックフェラー ユニバーシティ | 一過性トランスフェクションによる高力価のヘルパーフリーのレトロウイルスの生産 |
| FR2712812B1 (fr) | 1993-11-23 | 1996-02-09 | Centre Nat Rech Scient | Composition pour la production de produits thérapeutiques in vivo. |
| IL116816A (en) | 1995-01-20 | 2003-05-29 | Rhone Poulenc Rorer Sa | Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| US6004797A (en) | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
| US6346415B1 (en) | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| US20020065236A1 (en) * | 1998-09-09 | 2002-05-30 | Yew Nelson S. | CpG reduced plasmids and viral vectors |
| 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 |
| US7858367B2 (en) | 2002-04-30 | 2010-12-28 | Duke University | Viral vectors and methods for producing and using the same |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| JP2005535318A (ja) | 2002-08-12 | 2005-11-24 | ユニヴェルシテ ドゥ シェルブルック | プレメッセンジャーｒｎａのスプライス部位選択の再プログラミング方法 |
| AU2003295881A1 (en) * | 2002-11-25 | 2004-06-18 | Exelixis, Inc. | Lamps as modifiers of the p53 pathway and methods of use |
| 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 |
| CA2641070C (en) | 2006-02-06 | 2017-01-03 | Ramot At Tel-Aviv University Ltd. | Enzyme replacement therapy for treating lysosomal storage diseases |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US8324367B2 (en) | 2006-11-03 | 2012-12-04 | Medtronic, Inc. | Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity |
| PL2158322T3 (pl) | 2007-06-06 | 2017-10-31 | Genzyme Corp | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| US9169494B2 (en) | 2010-01-12 | 2015-10-27 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| CA2799944A1 (en) * | 2010-05-20 | 2011-11-24 | University Of Rochester | Methods and compositions related to modulating autophagy |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| US10648001B2 (en) * | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| AU2013332061A1 (en) | 2012-10-17 | 2015-05-07 | Fondazione Telethon | Gene therapy for glycogen storage diseases |
| US10781459B2 (en) | 2014-06-20 | 2020-09-22 | University Of Florida Research Foundation, Incorporated | Methods of packaging multiple adeno-associated virus vectors |
| WO2016200543A2 (en) | 2015-05-13 | 2016-12-15 | The Trustees Of The University Of Pennsylvania | Aav-mediated expression of anti-inluenza antibodies and methods of use thereof |
| EP3405215B1 (en) | 2016-01-19 | 2022-06-15 | The Regents of the University of California | Methods for the treatment of danon disease and other disorders of autophagy |
| IL320371A (en) * | 2016-09-08 | 2025-06-01 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Gene therapy for patients with fanconi anemia |
| EP3519569B1 (en) | 2016-09-30 | 2020-12-02 | Esteve Pharmaceuticals, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| AU2018236353B2 (en) | 2017-03-15 | 2023-12-21 | The Regents Of The University Of California | Methods of treating lysosomal disorders |
| US20220143215A1 (en) * | 2019-02-12 | 2022-05-12 | Spacecraft Seven, Llc | Gene therapy vectors for treatment of danon disease |
-
2019
- 2019-07-11 EP EP19835189.2A patent/EP3820537A4/en active Pending
- 2019-07-11 CN CN201980049274.0A patent/CN112512596B/zh active Active
- 2019-07-11 MX MX2021000443A patent/MX2021000443A/es unknown
- 2019-07-11 JP JP2021500664A patent/JP7586810B2/ja active Active
- 2019-07-11 KR KR1020217003946A patent/KR102918544B1/ko active Active
- 2019-07-11 AU AU2019299970A patent/AU2019299970B2/en active Active
- 2019-07-11 CA CA3106010A patent/CA3106010A1/en active Pending
- 2019-07-11 SG SG11202100022SA patent/SG11202100022SA/en unknown
- 2019-07-11 WO PCT/US2019/041465 patent/WO2020014523A1/en not_active Ceased
- 2019-07-11 BR BR112021000509-9A patent/BR112021000509A2/pt unknown
- 2019-12-27 US US16/729,002 patent/US10703797B2/en active Active
-
2021
- 2021-01-03 IL IL279918A patent/IL279918A/en unknown
- 2021-01-08 US US17/144,933 patent/US20210381003A1/en not_active Abandoned
-
2024
- 2024-07-18 JP JP2024114749A patent/JP2024150588A/ja active Pending
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