JPWO2023039440A5 - - Google Patents

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Publication number
JPWO2023039440A5
JPWO2023039440A5 JP2024515069A JP2024515069A JPWO2023039440A5 JP WO2023039440 A5 JPWO2023039440 A5 JP WO2023039440A5 JP 2024515069 A JP2024515069 A JP 2024515069A JP 2024515069 A JP2024515069 A JP 2024515069A JP WO2023039440 A5 JPWO2023039440 A5 JP WO2023039440A5
Authority
JP
Japan
Prior art keywords
sequence
mutation
grna
domain
optionally
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
JP2024515069A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024533313A (ja
JP2024533313A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/076063 external-priority patent/WO2023039440A2/en
Publication of JP2024533313A publication Critical patent/JP2024533313A/ja
Publication of JP2024533313A5 publication Critical patent/JP2024533313A5/ja
Publication of JPWO2023039440A5 publication Critical patent/JPWO2023039440A5/ja
Pending legal-status Critical Current

Links

JP2024515069A 2021-09-08 2022-09-07 Hbbを調節する組成物及び方法 Pending JP2024533313A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163241994P 2021-09-08 2021-09-08
US63/241,994 2021-09-08
US202163250143P 2021-09-29 2021-09-29
US63/250,143 2021-09-29
US202263303900P 2022-01-27 2022-01-27
US63/303,900 2022-01-27
PCT/US2022/076063 WO2023039440A2 (en) 2021-09-08 2022-09-07 Hbb-modulating compositions and methods

Publications (3)

Publication Number Publication Date
JP2024533313A JP2024533313A (ja) 2024-09-12
JP2024533313A5 JP2024533313A5 (https=) 2025-09-17
JPWO2023039440A5 true JPWO2023039440A5 (https=) 2025-09-17

Family

ID=85506910

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024515069A Pending JP2024533313A (ja) 2021-09-08 2022-09-07 Hbbを調節する組成物及び方法

Country Status (7)

Country Link
US (1) US20240252682A1 (https=)
EP (1) EP4399307A4 (https=)
JP (1) JP2024533313A (https=)
AU (1) AU2022342169A1 (https=)
CA (1) CA3231679A1 (https=)
TW (1) TW202323524A (https=)
WO (1) WO2023039440A2 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CN113286880A (zh) 2018-08-28 2021-08-20 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
CA3174483A1 (en) 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Improved methods and compositions for modulating a genome
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
KR20240099164A (ko) 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
EP4684023A2 (en) * 2023-03-21 2026-01-28 Tessera Therapeutics, Inc. Modified template guide rna molecules
WO2024206125A1 (en) * 2023-03-24 2024-10-03 The Broad Institute, Inc. Use of prime editing for treating sickle cell disease
WO2025059607A1 (en) * 2023-09-13 2025-03-20 Tessera Therapeutics, Inc. Lipid nanoparticles for delivery of therapeutic payloads to cells
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3445388T3 (da) * 2016-04-18 2024-06-03 Vertex Pharma Materialer og fremgangsmåder til behandling af hæmoglobinopatier
BR112019000057A2 (pt) * 2016-07-05 2019-04-02 The Johns Hopkins University composições e métodos com base em crispr/cas9 para o tratamento de degeneração de retina
JP7657726B2 (ja) * 2019-03-19 2025-04-07 ザ ブロード インスティテュート,インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
CN114667149A (zh) * 2019-09-09 2022-06-24 比姆医疗股份有限公司 新型核碱基编辑器及其使用方法
US12435330B2 (en) * 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA

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