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 (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109641063B (zh) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | 用于增强pklr的基因表达的组合物和方法 |
| SG11202002263TA (en) | 2017-10-16 | 2020-04-29 | Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
| JP7576754B2 (ja) | 2018-04-11 | 2024-11-01 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | 幹細胞移植のための組成物および方法 |
| AU2019261646A1 (en) | 2018-04-27 | 2020-11-12 | Spacecraft Seven, Llc | Gene therapy for CNS degeneration |
| CN120324641A (zh) * | 2019-02-12 | 2025-07-18 | 太空飞船七有限责任公司 | 用于治疗达农病的基因治疗载体 |
| EP4087489B1 (en) | 2020-01-08 | 2025-05-14 | Vitruvia Holdings Inc. | 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 |
| IL300263A (en) | 2020-08-07 | 2023-03-01 | Spacecraft Seven Llc | Plakophilin-2 (PKP2) gene therapy using an AAV vector |
| EP4217010A4 (en) * | 2020-09-28 | 2025-01-08 | Tacit Therapeutics, Inc. | TRANS-SPLICING SYSTEM FOR TISSUE-SPECIFIC REPLACEMENT OF RNA SEQUENCES |
| EP4228610A1 (en) * | 2020-10-13 | 2023-08-23 | Kriya Therapeutics, Inc. | Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer |
| EP4232152A4 (en) * | 2020-10-21 | 2025-04-23 | Duke University | Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy |
| JP2023550151A (ja) * | 2020-11-20 | 2023-11-30 | ベイラー カレッジ オブ メディスン | 幹細胞ニッチにおけるｈｉｐｐｏシグナル伝達を抑制することによる骨格筋再生の促進 |
| KR20230129431A (ko) * | 2020-12-07 | 2023-09-08 | 스페이스크래프트 세븐, 엘엘씨 | 다농병의 치료 |
| MX2024000175A (es) * | 2021-07-08 | 2024-03-05 | Tenaya Therapeutics Inc | Casetes de expresión optimizada para genoterapia. |
| IL311101A (en) | 2021-09-03 | 2024-04-01 | Tacit Therapeutics Inc | RNA editing through recruitment of SPLICEOSOME components |
| US20240382619A1 (en) * | 2021-09-08 | 2024-11-21 | Kkoomlab Inc. | Plasmid platform for stable expression and delivery of biomolecules |
| EP4400590A4 (en) * | 2021-09-08 | 2025-10-29 | Kkoomlab Inc | PLASMIC PLATFORM FOR THE STABLE EXPRESSION AND ADMINISTRATION OF BIOMOLECULES |
| TW202603176A (zh) * | 2024-04-12 | 2026-01-16 | 美商阿斯克生物公司 | 以密碼子最佳化核酸編碼ｌａｍｐ２ｂ的治療性腺相關病毒 |
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 |
| WO1993003769A1 (en) | 1991-08-20 | 1993-03-04 | THE UNITED STATES OF AMERICA, represented by THE SECRETARY, DEPARTEMENT OF HEALTH AND HUMAN SERVICES | Adenovirus mediated transfer of genes to the gastrointestinal tract |
| DE69434860T2 (de) | 1993-02-22 | 2007-03-15 | The Rockefeller University | Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion |
| 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 |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| AU2003223775A1 (en) | 2002-04-30 | 2003-11-17 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| WO2004015106A1 (en) | 2002-08-12 | 2004-02-19 | UNIVERSITé DE SHERBROOKE | Methods to reprogram splice site selection in pre-messenger rnas |
| WO2004048537A2 (en) * | 2002-11-25 | 2004-06-10 | Exelixis, Inc. | Lamps as modifiers of the p53 pathway and methods of use |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| WO2007091250A2 (en) | 2006-02-06 | 2007-08-16 | The Medical Research And Infrastructure Fund Of The Tel-Aviv Sourasky Medical Center | Enzyme replacement therapy for treating lysosomal storage diseases |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford 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 |
| PT3252161T (pt) | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
| ES2683695T3 (es) | 2010-01-12 | 2018-09-27 | The University Of North Carolina At Chapel Hill | Repeticiones terminales invertidas restrictivas para vectores virales |
| US20130184223A1 (en) * | 2010-05-20 | 2013-07-18 | University Of Rochester | Methods and compositions related to modulating autophagy |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| US10648001B2 (en) * | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| US20150273016A1 (en) | 2012-10-17 | 2015-10-01 | Fondazione Telethon | Gene therapy for glycogen storage diseases |
| WO2015196179A1 (en) | 2014-06-20 | 2015-12-23 | University Of Florida Research Foundation, Inc. | Methods of packaging multiple adeno-associated virus vectors |
| EA201792500A1 (ru) * | 2015-05-13 | 2018-04-30 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Aav-опосредованная экспрессия антител против гриппа и способы их использования |
| PL3405215T3 (pl) | 2016-01-19 | 2022-10-31 | The Regents Of The University Of California | Sposoby leczenia choroby danona i innych zaburzeń autofagii |
| MX2019002699A (es) * | 2016-09-08 | 2019-12-16 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Terapia genica para pacientes con anemia de fanconi. |
| WO2018060097A1 (en) | 2016-09-30 | 2018-04-05 | Laboratorios Del Dr. Esteve, S. A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| WO2018170239A1 (en) | 2017-03-15 | 2018-09-20 | The Regents Of The University Of California | Methods of treating lysosomal disorders |
| CN120324641A (zh) * | 2019-02-12 | 2025-07-18 | 太空飞船七有限责任公司 | 用于治疗达农病的基因治疗载体 |
-
2019
- 2019-07-11 WO PCT/US2019/041465 patent/WO2020014523A1/en not_active Ceased
- 2019-07-11 CN CN201980049274.0A patent/CN112512596B/zh active Active
- 2019-07-11 SG SG11202100022SA patent/SG11202100022SA/en unknown
- 2019-07-11 KR KR1020217003946A patent/KR102918544B1/ko active Active
- 2019-07-11 MX MX2021000443A patent/MX2021000443A/es unknown
- 2019-07-11 CA CA3106010A patent/CA3106010A1/en active Pending
- 2019-07-11 AU AU2019299970A patent/AU2019299970B2/en active Active
- 2019-07-11 EP EP19835189.2A patent/EP3820537A4/en active Pending
- 2019-07-11 BR BR112021000509-9A patent/BR112021000509A2/pt unknown
- 2019-07-11 JP JP2021500664A patent/JP7586810B2/ja active Active
- 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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