JP2022520232A5 - - Google Patents

Info

Publication number
JP2022520232A5
JP2022520232A5 JP2021546891A JP2021546891A JP2022520232A5 JP 2022520232 A5 JP2022520232 A5 JP 2022520232A5 JP 2021546891 A JP2021546891 A JP 2021546891A JP 2021546891 A JP2021546891 A JP 2021546891A JP 2022520232 A5 JP2022520232 A5 JP 2022520232A5
Authority
JP
Japan
Prior art keywords
gene therapy
therapy vector
raav gene
raav
administration
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
Application number
JP2021546891A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022520232A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/017987 external-priority patent/WO2020167996A1/en
Publication of JP2022520232A publication Critical patent/JP2022520232A/ja
Publication of JP2022520232A5 publication Critical patent/JP2022520232A5/ja
Priority to JP2025007633A priority Critical patent/JP2025063228A/ja
Pending legal-status Critical Current

Links

JP2021546891A 2019-02-12 2020-02-12 ダノン病の治療のための遺伝子療法ベクター Pending JP2022520232A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025007633A JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962804521P 2019-02-12 2019-02-12
US62/804,521 2019-02-12
US201962934928P 2019-11-13 2019-11-13
US62/934,928 2019-11-13
PCT/US2020/017987 WO2020167996A1 (en) 2019-02-12 2020-02-12 Gene therapy vectors for treatment of danon disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025007633A Division JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Publications (2)

Publication Number Publication Date
JP2022520232A JP2022520232A (ja) 2022-03-29
JP2022520232A5 true JP2022520232A5 (enExample) 2023-02-21

Family

ID=72043957

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021546891A Pending JP2022520232A (ja) 2019-02-12 2020-02-12 ダノン病の治療のための遺伝子療法ベクター
JP2025007633A Pending JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025007633A Pending JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Country Status (12)

Country Link
US (1) US20220143215A1 (enExample)
EP (1) EP3924371A4 (enExample)
JP (2) JP2022520232A (enExample)
KR (1) KR20210125999A (enExample)
CN (2) CN120324641A (enExample)
AU (3) AU2020221842A1 (enExample)
BR (1) BR112021015751A2 (enExample)
CA (1) CA3128514A1 (enExample)
IL (1) IL285238A (enExample)
MX (1) MX2021009696A (enExample)
SG (1) SG11202107744SA (enExample)
WO (1) WO2020167996A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
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)
EP3820537A4 (en) * 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
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
WO2022032226A1 (en) * 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
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
CN114874990A (zh) * 2021-02-05 2022-08-09 中国科学院苏州纳米技术与纳米仿生研究所 一种功能化外泌体及其制备方法和应用
US12609410B2 (en) 2021-09-23 2026-04-21 Lg Energy Solution, Ltd. High heat-resistant connector, and battery module, battery pack, and vehicle including the same
CN114457112A (zh) * 2022-02-07 2022-05-10 苏州市立医院 一种特异性神经靶向mr分子探针及其制备方法和应用
CN115975051B (zh) * 2022-11-17 2025-09-12 东南大学 靶向外泌体及制备方法、应用、药物和药物递送系统

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003038049A2 (en) * 2001-10-29 2003-05-08 Renovis, Inc. Method for isolating cell-type specific mrnas
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US9434928B2 (en) * 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
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
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
WO2019028273A1 (en) * 2017-08-02 2019-02-07 Dna2.0, Inc. Dba Atum DNA VECTORS AND ELEMENTS FOR SUSTAINED GENE EXPRESSION IN EUKARYOTIC CELLS
EP3820537A4 (en) * 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
WO2020033842A1 (en) * 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production

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