AU2020248337A1 - Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof - Google Patents

Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof Download PDF

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Publication number
AU2020248337A1
AU2020248337A1 AU2020248337A AU2020248337A AU2020248337A1 AU 2020248337 A1 AU2020248337 A1 AU 2020248337A1 AU 2020248337 A AU2020248337 A AU 2020248337A AU 2020248337 A AU2020248337 A AU 2020248337A AU 2020248337 A1 AU2020248337 A1 AU 2020248337A1
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composition
lipid
seq
administered
subject
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Inventor
Seth C. Alexander
Yong Chang
Arti Mahendra Prakash KANJOLIA
Reynald Michael LESCARBEAU
Shobu ODATE
Jessica Lynn SEITZER
Walter Strapps
Kristy M. WOOD
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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AU2020248337A 2019-03-28 2020-03-27 Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof Pending AU2020248337A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962825676P 2019-03-28 2019-03-28
US201962825637P 2019-03-28 2019-03-28
US62/825,637 2019-03-28
US62/825,676 2019-03-28
PCT/US2020/025533 WO2020198706A1 (en) 2019-03-28 2020-03-27 Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof

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AU2020248337A1 true AU2020248337A1 (en) 2021-11-04

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US (1) US20230035659A1 (enExample)
EP (1) EP3946285A1 (enExample)
JP (2) JP7636338B2 (enExample)
KR (1) KR20220004984A (enExample)
CN (1) CN113874004A (enExample)
AU (1) AU2020248337A1 (enExample)
CA (1) CA3134544A1 (enExample)
CO (1) CO2021014562A2 (enExample)
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CN117098541A (zh) 2020-11-25 2023-11-21 阿卡格拉医药公司 用于递送核酸的脂质纳米粒及相关使用方法
EP4259792A1 (en) * 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
EP4322920A1 (en) * 2021-04-17 2024-02-21 Intellia Therapeutics, Inc. Lipid nanoparticle compositions
CA3220738A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
WO2022271780A1 (en) * 2021-06-22 2022-12-29 Intellia Therapeutics, Inc. Methods for in vivo editing of a liver gene
WO2023230587A2 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
KR20260094824A (ko) * 2023-10-25 2026-06-23 씨에스피씨 종콰이 팔마씨우티컬 테크놀로지 (스자좡) 컴퍼니 리미티드 고효율, 저오프타겟 유전자 편집 도구
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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