JP7472121B2 - アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 - Google Patents

アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 Download PDF

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JP7472121B2
JP7472121B2 JP2021521406A JP2021521406A JP7472121B2 JP 7472121 B2 JP7472121 B2 JP 7472121B2 JP 2021521406 A JP2021521406 A JP 2021521406A JP 2021521406 A JP2021521406 A JP 2021521406A JP 7472121 B2 JP7472121 B2 JP 7472121B2
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rna
sequence
composition
nucleic acid
seq
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JP2022505402A (ja
JP2022505402A5 (https=
Inventor
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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Regeneron Pharmaceuticals Inc
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
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    • A01K2207/15—Humanized animals
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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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JP2021521406A 2018-10-18 2019-10-18 アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法 Active JP7472121B2 (ja)

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JP2024063210A JP7820434B2 (ja) 2018-10-18 2024-04-10 アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法

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US201862747402P 2018-10-18 2018-10-18
US62/747,402 2018-10-18
US201962840346P 2019-04-29 2019-04-29
US62/840,346 2019-04-29
PCT/US2019/057086 WO2020082042A2 (en) 2018-10-18 2019-10-18 Compositions and methods for transgene expression from an albumin locus

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JP2024063210A Active JP7820434B2 (ja) 2018-10-18 2024-04-10 アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法

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US (2) US20200270617A1 (https=)
EP (1) EP3867381A2 (https=)
JP (2) JP7472121B2 (https=)
KR (2) KR102949204B1 (https=)
CN (1) CN114207130A (https=)
AU (1) AU2019361203B2 (https=)
BR (1) BR112021007343A2 (https=)
CA (1) CA3116918A1 (https=)
CO (1) CO2021006363A2 (https=)
IL (1) IL282236A (https=)
MX (1) MX2021004278A (https=)
MY (1) MY208564A (https=)
PH (1) PH12021550844A1 (https=)
SA (1) SA521421766B1 (https=)
SG (1) SG11202103733SA (https=)
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JP2025521154A (ja) 2022-05-31 2025-07-08 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法
CN120659627A (zh) 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法
KR20250075694A (ko) 2022-09-28 2025-05-28 리제너론 파마슈티칼스 인코포레이티드 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체
EP4612184A1 (en) 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
KR20250116795A (ko) 2022-11-14 2025-08-01 리제너론 파마슈티칼스 인코포레이티드 별아교세포로의 섬유아세포 성장 인자 수용체 3-매개된 전달을 위한 조성물 및 방법
KR20260032927A (ko) 2023-06-30 2026-03-10 리제너론 파마슈티칼스 인코포레이티드 상동성 지향 복구를 증가시키는 방법 및 조성물
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
CN121693566A (zh) 2023-07-28 2026-03-17 瑞泽恩制药公司 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达
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
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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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Citations (10)

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