JP2023526310A5 - - Google Patents

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Publication number
JP2023526310A5
JP2023526310A5 JP2022569265A JP2022569265A JP2023526310A5 JP 2023526310 A5 JP2023526310 A5 JP 2023526310A5 JP 2022569265 A JP2022569265 A JP 2022569265A JP 2022569265 A JP2022569265 A JP 2022569265A JP 2023526310 A5 JP2023526310 A5 JP 2023526310A5
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JP
Japan
Prior art keywords
mir
target sequences
sequence
raav
composition according
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
JP2022569265A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023526310A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/032003 external-priority patent/WO2021231579A1/en
Publication of JP2023526310A publication Critical patent/JP2023526310A/ja
Publication of JP2023526310A5 publication Critical patent/JP2023526310A5/ja
Pending legal-status Critical Current

Links

JP2022569265A 2020-05-12 2021-05-12 導入遺伝子発現のｄｒｇ特異的低減のための組成物 Pending JP2023526310A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063023593P 2020-05-12 2020-05-12
US63/023,593 2020-05-12
US202063038488P 2020-06-12 2020-06-12
US63/038,488 2020-06-12
US202063043562P 2020-06-24 2020-06-24
US63/043,562 2020-06-24
US202063079299P 2020-09-16 2020-09-16
US63/079,299 2020-09-16
US202163152042P 2021-02-22 2021-02-22
US63/152,042 2021-02-22
PCT/US2021/032003 WO2021231579A1 (en) 2020-05-12 2021-05-12 Compositions for drg-specific reduction of transgene expression

Publications (2)

Publication Number Publication Date
JP2023526310A JP2023526310A (ja) 2023-06-21
JP2023526310A5 true JP2023526310A5 (enExample) 2024-05-22

Family

ID=76532231

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022569265A Pending JP2023526310A (ja) 2020-05-12 2021-05-12 導入遺伝子発現のｄｒｇ特異的低減のための組成物

Country Status (12)

Country Link
US (1) US20230304034A1 (enExample)
EP (1) EP4162059A1 (enExample)
JP (1) JP2023526310A (enExample)
KR (1) KR20230010670A (enExample)
CN (1) CN115803064A (enExample)
AU (1) AU2021270447A1 (enExample)
BR (1) BR112022022212A2 (enExample)
CA (1) CA3177407A1 (enExample)
CO (1) CO2022017959A2 (enExample)
IL (1) IL298138A (enExample)
MX (1) MX2022014258A (enExample)
WO (1) WO2021231579A1 (enExample)

Families Citing this family (9)

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CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
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WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
EP4504797A1 (en) 2022-04-06 2025-02-12 The Trustees of The University of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
AU2023303882A1 (en) * 2022-07-08 2025-01-23 Sensorion REGULATORY SEQUENCES COMPRISING MicroRNA TARGET SITES
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy

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CA3012195A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
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IL316926A (en) 2017-05-11 2025-01-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis
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CN111989396A (zh) 2017-11-30 2020-11-24 宾夕法尼亚州大学信托人 用于iiib型粘多糖贮积病的基因疗法
AU2018375164B2 (en) 2017-11-30 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for Mucopolysaccharidosis IIIA
WO2020132455A1 (en) * 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CA3133889A1 (en) * 2019-04-24 2020-10-29 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
KR20220020261A (ko) * 2019-05-03 2022-02-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이염성 백질이영양증의 치료에 유용한 조성물
WO2021081217A1 (en) * 2019-10-23 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression

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