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

Publications (1)

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CO2021014562A2 true CO2021014562A2 (es) 2021-11-19

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US (1) US20230035659A1 (https=)
EP (1) EP3946285A1 (https=)
JP (2) JP7636338B2 (https=)
KR (1) KR20220004984A (https=)
CN (1) CN113874004A (https=)
AU (1) AU2020248337A1 (https=)
CA (1) CA3134544A1 (https=)
CO (1) CO2021014562A2 (https=)
MX (1) MX2021011690A (https=)
WO (1) WO2020198706A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
CR20230305A (es) * 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
BR112023021477A2 (pt) * 2021-04-17 2024-01-30 Intellia Therapeutics Inc Composições de nanopartículas lipídicas
WO2022246266A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
KR20240038705A (ko) * 2021-06-22 2024-03-25 인텔리아 테라퓨틱스, 인크. 간 유전자의 생체 내 편집 방법
EP4531819A2 (en) 2022-05-25 2025-04-09 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
CN120265763A (zh) * 2023-10-25 2025-07-04 上海津曼特生物科技有限公司 靶向ttr的基因编辑组合物
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

Family Cites Families (22)

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Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
KR940703846A (ko) 1991-12-24 1994-12-12 비. 린네 파샬 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides)
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
AU2010223967B2 (en) * 2009-03-12 2015-07-30 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
CA2895155C (en) 2012-12-17 2021-07-06 President And Fellows Of Harvard College Rna-guided human genome engineering
EP3608308B1 (en) 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
MX2016007325A (es) * 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
EP3169309B1 (en) 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
ES2964690T3 (es) 2015-09-21 2024-04-09 Trilink Biotechnologies Llc Método para sintetizar ARN con caperuza 5'
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018067447A1 (en) 2016-10-03 2018-04-12 Itellia Therapeutics, Inc. Improved methods for identifying double strand break sites
PT3688162T (pt) 2017-09-29 2024-04-23 Intellia Therapeutics Inc Formulações
AR113154A1 (es) * 2017-09-29 2020-01-29 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para edición del genoma
EP3688161A1 (en) * 2017-09-29 2020-08-05 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis

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