CO2021014562A2 - Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr que comprenden un corticosteroide o el uso de este - Google Patents

Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr que comprenden un corticosteroide o el uso de este

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Publication number
CO2021014562A2
CO2021014562A2 CONC2021/0014562A CO2021014562A CO2021014562A2 CO 2021014562 A2 CO2021014562 A2 CO 2021014562A2 CO 2021014562 A CO2021014562 A CO 2021014562A CO 2021014562 A2 CO2021014562 A2 CO 2021014562A2
Authority
CO
Colombia
Prior art keywords
corticosteroid
methods
compositions
treatment
ttr gene
Prior art date
Application number
CONC2021/0014562A
Other languages
English (en)
Spanish (es)
Inventor
Walter Strapps
Shobu Odate
Reynald Michael Lescarbeau
Arti Mahendra Prakash Kanjolia
Jessica Lynn Seitzer
Seth C Alexander
Yong Chang
Kristy M Wood
Original Assignee
Intellia Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of CO2021014562A2 publication Critical patent/CO2021014562A2/es

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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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    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
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CONC2021/0014562A 2019-03-28 2021-10-28 Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr que comprenden un corticosteroide o el uso de este CO2021014562A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962825676P 2019-03-28 2019-03-28
US201962825637P 2019-03-28 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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CO2021014562A2 true CO2021014562A2 (es) 2021-11-19

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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)
MX (1) MX2021011690A (enExample)
WO (1) WO2020198706A1 (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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