JP7578590B2 - 第ｉｘ因子を発現するための組成物及び方法 - Google Patents

第ｉｘ因子を発現するための組成物及び方法 Download PDF

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JP7578590B2
JP7578590B2 JP2021521112A JP2021521112A JP7578590B2 JP 7578590 B2 JP7578590 B2 JP 7578590B2 JP 2021521112 A JP2021521112 A JP 2021521112A JP 2021521112 A JP2021521112 A JP 2021521112A JP 7578590 B2 JP7578590 B2 JP 7578590B2
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factor
nucleic acid
sequence
acid construct
rna
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JP2022512731A5 (enExample
JP2022512731A (ja
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ダグラス フィン，ジョナサン
フアン，ホン－レン
ロイ，モイトリ
ライ，ケーディー
サットラー，レイチェル
カイラトサウス，クリストス
ワン，チェン
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Regeneron Pharmaceuticals Inc
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JP2021521112A 2018-10-18 2019-10-18 第ｉｘ因子を発現するための組成物及び方法 Active JP7578590B2 (ja)

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US201862747509P 2018-10-18 2018-10-18
US62/747,509 2018-10-18
US201962829009P 2019-04-03 2019-04-03
US62/829,009 2019-04-03
US201962829621P 2019-04-04 2019-04-04
US62/829,621 2019-04-04
US201962840352P 2019-04-29 2019-04-29
US62/840,352 2019-04-29
PCT/US2019/057090 WO2020082046A2 (en) 2018-10-18 2019-10-18 Compositions and methods for expressing factor ix

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WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
KR20250075694A (ko) 2022-09-28 2025-05-28 리제너론 파마슈티칼스 인코포레이티드 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체
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IL326018A (en) 2023-07-28 2026-03-01 Regeneron Pharma Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease
AR133384A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida
AU2024315073A1 (en) 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
EP4771049A1 (en) 2023-08-28 2026-07-08 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
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
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WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
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