KR20220004648A - Ttr 가이드 rna, 및 rna-가이드 dna 결합제를 암호화하는 폴리뉴클레오티드를 포함하는 조성물 및 방법 - Google Patents

Ttr 가이드 rna, 및 rna-가이드 dna 결합제를 암호화하는 폴리뉴클레오티드를 포함하는 조성물 및 방법 Download PDF

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KR20220004648A
KR20220004648A KR1020217035004A KR20217035004A KR20220004648A KR 20220004648 A KR20220004648 A KR 20220004648A KR 1020217035004 A KR1020217035004 A KR 1020217035004A KR 20217035004 A KR20217035004 A KR 20217035004A KR 20220004648 A KR20220004648 A KR 20220004648A
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lipid
composition
seq
rna
ttr
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Korean (ko)
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용 창
세스 씨. 알렉산더
크리스티 엠. 우드
아티 마헨드라 프라카쉬 칸졸리아
쇼부 오다테
제시카 린 시쳐
레이날드 마이클 레스카뷰
월터 스트랩스
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인텔리아 테라퓨틱스, 인크.
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KR1020217035004A 2019-03-28 2020-03-27 Ttr 가이드 rna, 및 rna-가이드 dna 결합제를 암호화하는 폴리뉴클레오티드를 포함하는 조성물 및 방법 Pending KR20220004648A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962825637P 2019-03-28 2019-03-28
US62/825,637 2019-03-28
PCT/US2020/025513 WO2020198697A1 (en) 2019-03-28 2020-03-27 Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent

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KR20220004648A true KR20220004648A (ko) 2022-01-11

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KR1020217035004A Pending KR20220004648A (ko) 2019-03-28 2020-03-27 Ttr 가이드 rna, 및 rna-가이드 dna 결합제를 암호화하는 폴리뉴클레오티드를 포함하는 조성물 및 방법

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CN (1) CN113874076A (enExample)
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Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4144378A1 (en) * 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
MX2023006056A (es) 2020-11-25 2023-06-06 Akagera Medicines Inc Nanoparticulas lipidicas para suministro de acidos nucleicos y metodos de uso de las mismas.
US20220396782A1 (en) * 2021-06-04 2022-12-15 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof
CA3224995A1 (en) * 2021-06-22 2022-12-29 Intellia Therapeutics, Inc. Methods for in vivo editing of a liver gene
WO2023185697A2 (en) 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
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
WO2024061296A2 (en) 2022-09-22 2024-03-28 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
EP4627083A1 (en) * 2022-11-30 2025-10-08 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof
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 (23)

* Cited by examiner, † Cited by third party
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
DK0618925T4 (da) 1991-12-24 2012-07-09 Isis Pharmaceuticals Inc Antisense-oligonukleotider
WO1995032305A1 (en) 1994-05-19 1995-11-30 Dako A/S Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
EP2931898B1 (en) * 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP4481048A3 (en) 2012-12-17 2025-02-26 President and Fellows of Harvard College Rna-guided human genome engineering
WO2014136086A1 (en) 2013-03-08 2014-09-12 Novartis Ag Lipids and lipid compositions for the delivery of active agents
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
US10342761B2 (en) 2014-07-16 2019-07-09 Novartis Ag Method of encapsulating a nucleic acid in a lipid nanoparticle host
US9944912B2 (en) 2015-03-03 2018-04-17 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
CN107849562B (zh) * 2015-07-28 2021-10-26 丹尼斯科美国公司 基因组编辑系统及使用方法
HUE055458T2 (hu) 2015-09-21 2021-11-29 Trilink Biotechnologies Llc Kompozíciók és módszerek 5'-Cap RNS-ek szintézisére
WO2017173054A1 (en) 2016-03-30 2017-10-05 Intellia Therapeutics, Inc. Lipid nanoparticle formulations for crispr/cas components
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
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
CN111417728A (zh) * 2017-09-29 2020-07-14 因特利亚治疗公司 用于ttr基因编辑及治疗attr淀粉样变性的组合物及方法
CN111405912B (zh) * 2017-09-29 2025-02-14 因特利亚治疗公司 用于基因组编辑的多核苷酸、组合物及方法
EP3802828A4 (en) * 2018-06-08 2022-10-26 Intellia Therapeutics, Inc. RNA MODIFIED GUIDES FOR GENE EDITING
JP2022502055A (ja) * 2018-09-28 2022-01-11 インテリア セラピューティクス，インコーポレイテッド 乳酸デヒドロゲナーゼ（ｌｄｈａ）遺伝子編集のための組成物及び方法

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