TWI904069B - 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法 - Google Patents

用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法

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Publication number
TWI904069B
TWI904069B TW107134336A TW107134336A TWI904069B TW I904069 B TWI904069 B TW I904069B TW 107134336 A TW107134336 A TW 107134336A TW 107134336 A TW107134336 A TW 107134336A TW I904069 B TWI904069 B TW I904069B
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Taiwan
Prior art keywords
seq
sequence
rna
ttr
combination
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TW107134336A
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English (en)
Chinese (zh)
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TW201932479A (zh
Inventor
阿提 馬亨德拉 布拉科許 康裘莉亞
書布 歐德
潔西卡 林 西賽
雷納德 麥克 雷斯卡里
瓦特 史瑞普斯
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美商英特利亞醫療公司
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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
    • 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
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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TW107134336A 2017-09-29 2018-09-28 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法 TWI904069B (zh)

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US201762566236P 2017-09-29 2017-09-29
US62/566,236 2017-09-29
US201862671902P 2018-05-15 2018-05-15
US62/671,902 2018-05-15

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TW201932479A TW201932479A (zh) 2019-08-16
TWI904069B true TWI904069B (zh) 2025-11-11

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US (4) US20200248180A1 (https=)
EP (1) EP3688161A1 (https=)
JP (3) JP2021500864A (https=)
KR (1) KR20200058509A (https=)
CN (2) CN118530993A (https=)
AU (2) AU2018338787C1 (https=)
BR (1) BR112020005287A2 (https=)
CA (1) CA3077251A1 (https=)
CO (1) CO2020005116A2 (https=)
IL (3) IL311170B1 (https=)
MX (2) MX2020003608A (https=)
MY (1) MY203849A (https=)
PH (1) PH12020550364A1 (https=)
SG (1) SG11202002565YA (https=)
TW (1) TWI904069B (https=)
WO (1) WO2019067872A1 (https=)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
MX394999B (es) 2017-09-29 2025-03-24 Regeneron Pharma Animales no humanos que comprenden un locus ttr humanizado y metodos de uso
JP7543259B2 (ja) 2018-10-18 2024-09-02 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
AU2020248337A1 (en) * 2019-03-28 2021-11-04 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof
EP3946598A1 (en) * 2019-03-28 2022-02-09 Intellia Therapeutics, Inc. Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent
BR112021019224A2 (pt) * 2019-03-28 2021-11-30 Intellia Therapeutics Inc Polinucleotídeos, composições e métodos para expressão de polipeptídeo
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
MX2022006950A (es) * 2019-12-11 2022-11-07 Intellia Therapeutics Inc Arn guía modificados para edición de genes.
EP4237005A4 (en) * 2020-10-28 2024-10-09 Novo Nordisk A/S ANTI-TRANSTHYRETIN ANTIBODIES AND METHODS OF USE THEREOF
CR20230305A (es) 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
CN112795595A (zh) * 2020-12-24 2021-05-14 中山大学 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统
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
US20250127927A1 (en) * 2021-08-23 2025-04-24 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
CN116064598B (zh) * 2021-10-08 2024-03-12 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
WO2023185697A2 (en) * 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
TW202417017A (zh) * 2022-06-30 2024-05-01 香港商正基基因科技有限公司 具有化學修飾的導引rna
CN118853659A (zh) * 2023-04-14 2024-10-29 深圳瑞吉生物科技有限公司 提高mRNA翻译蛋白的输出量的核酸及其应用
WO2024259135A1 (en) 2023-06-13 2024-12-19 Intellia Therapeutics, Inc. Assays for analysis of ribonucleic acid (rna) molecules
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015183026A1 (ko) * 2014-05-28 2015-12-03 주식회사 툴젠 불활성화된 표적 특이적 뉴클레아제를 이용한 표적 dna의 분리 방법
WO2017053431A2 (en) * 2015-09-21 2017-03-30 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
WO2017077386A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a

Family Cites Families (31)

* 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
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
US6974667B2 (en) 2000-06-14 2005-12-13 Gene Logic, Inc. Gene expression profiles in liver cancer
US7244764B2 (en) * 2003-06-23 2007-07-17 Neurochem (International) Limited Methods and compositions for treating amyloid-related diseases
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
WO2010017509A1 (en) 2008-08-07 2010-02-11 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
EA036772B1 (ru) 2008-10-20 2020-12-18 Элнилэм Фармасьютикалз, Инк. Композиции и способы для ингибирования экспрессии транстиретина
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
RS56011B1 (sr) 2010-04-29 2017-09-29 Ionis Pharmaceuticals Inc Modulacija ekspresije transtiretina
HUE048622T2 (hu) 2011-11-18 2020-08-28 Alnylam Pharmaceuticals Inc RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
DK2931897T3 (en) * 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
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
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
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
KR20160019553A (ko) * 2013-06-17 2016-02-19 더 브로드 인스티튜트, 인코퍼레이티드 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
KR102386101B1 (ko) * 2014-06-26 2022-04-14 리제너론 파마슈티칼스 인코포레이티드 표적화된 유전자 변형을 위한 방법 및 그 조성물, 및 사용 방법
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'
CA3006618A1 (en) * 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
US11083799B2 (en) * 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
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
AU2017374044B2 (en) 2016-12-08 2023-11-30 Intellia Therapeutics, Inc. Modified guide RNAs

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015183026A1 (ko) * 2014-05-28 2015-12-03 주식회사 툴젠 불활성화된 표적 특이적 뉴클레아제를 이용한 표적 dna의 분리 방법
WO2017053431A2 (en) * 2015-09-21 2017-03-30 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
WO2017077386A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
期刊 Anders, Carolin, Martin Jinek, In vitro enzymology of Cas9, Methods in enzymology, vol. 546, 2014/11/12, pages 1-20;網路文獻 David V. Morrissey, "Robust In Vivo Editing of Hepatocyte Target DNA Mediated by Lipid Nanoparticle Delivery of CRISPR/Cas9 Components", /www.intelliatx.com/, 2017/05/13, chrome-extension://efaidnbmnnnibpcajpcglclefindmkaj/https://ir.intelliatx.com/static-files/c4880575-fbdb-44a3-ad2d-1ce551cec00b *
網路文獻 David V. Morrissey, "Robust In Vivo Editing of Hepatocyte Target DNA Mediated by Lipid Nanoparticle Delivery of CRISPR/Cas9 Components", /www.intelliatx.com/, 2017/05/13, chrome-extension://efaidnbmnnnibpcajpcglclefindmkaj/https://ir.intelliatx.com/static-files/c4880575-fbdb-44a3-ad2d-1ce551cec00b

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