KR20230016175A - Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법 - Google Patents

Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법 Download PDF

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KR20230016175A
KR20230016175A KR1020227038926A KR20227038926A KR20230016175A KR 20230016175 A KR20230016175 A KR 20230016175A KR 1020227038926 A KR1020227038926 A KR 1020227038926A KR 20227038926 A KR20227038926 A KR 20227038926A KR 20230016175 A KR20230016175 A KR 20230016175A
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composition
editing
guide rna
administered
angptl3
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알렉산드라 체드윅
칼란토타틸 지 라지브
앨런 로드
크리스토퍼 쳉
캐롤라인 레이스
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버브 테라퓨틱스, 인크.
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
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    • C07K2319/00—Fusion polypeptide
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KR1020227038926A 2020-04-09 2021-04-09 Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법 Pending KR20230016175A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063007803P 2020-04-09 2020-04-09
US202063007797P 2020-04-09 2020-04-09
US63/007,803 2020-04-09
US63/007,797 2020-04-09
US202063045033P 2020-06-26 2020-06-26
US202063045032P 2020-06-26 2020-06-26
US63/045,032 2020-06-26
US63/045,033 2020-06-26
US202163136087P 2021-01-11 2021-01-11
US63/136,087 2021-01-11
PCT/US2021/026729 WO2021207710A2 (en) 2020-04-09 2021-04-09 Base editing of angptl3 and methods of using same for treatment of disease

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KR20230016175A true KR20230016175A (ko) 2023-02-01

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KR1020227038926A Pending KR20230016175A (ko) 2020-04-09 2021-04-09 Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법
KR1020227038947A Pending KR20230016176A (ko) 2020-04-09 2021-04-09 Pcsk9의 염기 편집 및 질환 치료를 위한 이의 사용 방법

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KR1020227038947A Pending KR20230016176A (ko) 2020-04-09 2021-04-09 Pcsk9의 염기 편집 및 질환 치료를 위한 이의 사용 방법

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US (5) US12029795B2 (enExample)
EP (3) EP4133071A4 (enExample)
JP (4) JP7457832B2 (enExample)
KR (2) KR20230016175A (enExample)
CN (2) CN116096429A (enExample)
AU (4) AU2021252991B2 (enExample)
BR (2) BR112022020407A2 (enExample)
CA (2) CA3179863A1 (enExample)
GB (8) GB2632564B (enExample)
IL (2) IL297193A (enExample)
MX (1) MX2022012683A (enExample)
PH (2) PH12022553015A1 (enExample)
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CN119351474A (zh) 2017-02-22 2025-01-24 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
GB2632564B (en) 2020-04-09 2025-06-18 Verve Therapeutics Inc Base editing of angptl3 and methods of using same for treatment of disease
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
US20240424138A1 (en) * 2021-10-21 2024-12-26 Prime Medicine, Inc. Genome editing compositions and method for treatment of retinitis pigmentosa
US12037616B2 (en) * 2022-03-01 2024-07-16 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (ANGPTL3) related conditions
WO2023180904A1 (en) * 2022-03-21 2023-09-28 Crispr Therapeutics Ag Methods and compositions for treating lipoprotein-related diseases
JP2025522311A (ja) 2022-05-25 2025-07-15 アカゲラ・メディスンズ，インコーポレイテッド 核酸を送達するための脂質ナノ粒子およびその使用方法
CN119948162A (zh) * 2022-09-22 2025-05-06 锐正基因(苏州)有限公司 用于治疗高胆固醇血症和/或心血管疾病的组合物和方法
KR20250124819A (ko) * 2022-12-21 2025-08-20 인텔리아 테라퓨틱스, 인크. 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법
WO2024152058A1 (en) * 2023-01-13 2024-07-18 The Trustees Of The University Of Pennsylvania Compositions and methods for the treatment of hereditary tyrosinemia type 1
WO2024175550A1 (en) * 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024197065A2 (en) * 2023-03-20 2024-09-26 Verve Therapeutics, Inc. In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease
CN118813611B (zh) * 2023-04-19 2025-09-16 清华大学 一种基于rna核酶的dear核酸操纵系统及其应用
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions
WO2025045247A1 (en) * 2023-08-31 2025-03-06 Geneditbio Limited Nucleic acids encoding crispr-associated proteins and uses thereof
WO2025054363A1 (en) * 2023-09-05 2025-03-13 Cornell University Minigene cassettes, recombinant polynucleotides, and methods of their use
WO2025064656A1 (en) * 2023-09-19 2025-03-27 Beam Therapeutics Inc. Compositions and methods for altering a nucleobase in a lipoprotein(a) polynucleotide
CN117587013B (zh) * 2023-11-27 2024-06-14 湖南师范大学 一种血管发育异常的斑马鱼模型的构建方法
CN117568313B (zh) * 2024-01-15 2024-04-26 上海贝斯昂科生物科技有限公司 基因编辑组合物及其用途
CN118956837B (zh) * 2024-02-26 2025-09-23 深圳信立泰药业股份有限公司 用于pcsk9基因修饰或编辑的组合物及其使用方法
WO2025184520A1 (en) * 2024-02-29 2025-09-04 Intellia Therapeutics, Inc. Compositions and methods for angiopoietin like 3 (angptl3) editing
JP2025139413A (ja) * 2024-03-12 2025-09-26 サミー株式会社 遊技機
CN120130446A (zh) * 2025-03-10 2025-06-13 苏州系统医学研究所 一种制备疾病动物模型的方法及其应用

Family Cites Families (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001034768A2 (en) 1999-11-09 2001-05-17 Human Genome Sciences, Inc. 15 human secreted proteins
EP1027033B1 (en) 1997-05-14 2009-07-22 The University Of British Columbia High efficiency encapsulation of nucleic acids in lipid vesicles
EP1067182A3 (en) 1999-07-08 2001-11-21 Helix Research Institute Secretory protein or membrane protein
US20030119038A1 (en) 1999-09-09 2003-06-26 Bingham Brendan William NARC1, novel subtilase-like homologs
US7029895B2 (en) 1999-09-27 2006-04-18 Millennium Pharmaceuticals, Inc. 27411, a novel human PGP synthase
CA2388617A1 (en) 1999-10-22 2001-05-03 Millennium Pharmaceuticals, Inc. Nucleic acid molecules derived from rat brain and programmed cell death models
AU785007B2 (en) 1999-11-24 2006-08-24 Mcs Micro Carrier Systems Gmbh Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells
WO2001057081A2 (en) 2000-02-07 2001-08-09 Millennium Pharmaceuticals, Inc. Narc-1, subtilase-like homologs
AU2001261024A1 (en) 2000-04-12 2001-10-30 Delta Biotechnology Limited Albumin fusion proteins
JP2004515217A (ja) 2000-06-16 2004-05-27 インサイト・ゲノミックス・インコーポレイテッド プロテアーゼ
AU2001280471A1 (en) 2000-08-11 2002-02-25 Eli Lilly And Company Novel secreted proteins and their uses
AU2002227280A1 (en) 2000-12-08 2002-06-18 Incyte Genomics, Inc. Protein modification and maintenance molecules
DE10109897A1 (de) 2001-02-21 2002-11-07 Novosom Ag Fakultativ kationische Liposomen und Verwendung dieser
EP1423134A2 (en) 2001-03-21 2004-06-02 Human Genome Sciences Human secreted proteins
US7858117B2 (en) 2002-02-21 2010-12-28 Novosom Ag Amphoteric liposomes and their use
EP1440981A3 (en) 2003-01-21 2005-11-23 Research Association for Biotechnology Full-length human cdna
EP1471152A1 (en) 2003-04-25 2004-10-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Mutations in the human PCSK9 gene associated to hypercholesterolemia
DE102004054730A1 (de) 2004-03-28 2006-05-11 Novosom Ag Serumstabile amphotere Liposomen
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
US20070087045A1 (en) 2005-10-14 2007-04-19 Industrial Technology Research Institute Lipid carrier and method of preparing the same
US20100040610A1 (en) 2006-11-07 2010-02-18 Ayesha Sitlani Antagonists of pcsk9
TWI445716B (zh) * 2008-09-12 2014-07-21 Rinat Neuroscience Corp Ｐｃｓｋ９拮抗劑類
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
KR102271292B1 (ko) * 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
FR3012370B1 (fr) 2013-10-30 2017-04-14 Michelin & Cie Armature de sommet pour pneumatique d'avion
ES2774968T3 (es) 2013-12-19 2020-07-23 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
KR102298476B1 (ko) * 2013-12-24 2021-09-03 아이오니스 파마수티컬즈, 인코포레이티드 안지오포이에틴-유사 3 발현의 조절
PT3766916T (pt) 2014-06-25 2022-11-28 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
CA2964796C (en) * 2014-10-17 2022-01-11 The Penn State Research Foundation Methods and compositions for multiplex rna guided genome editing and other rna technologies
MA41123A (fr) * 2014-12-02 2017-10-10 Oncomed Pharm Inc Polythérapie pour le traitement du cancer
WO2017004279A2 (en) * 2015-06-29 2017-01-05 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
AU2016285852B2 (en) 2015-06-29 2020-12-17 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
CA2989884A1 (en) 2015-07-09 2017-01-12 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
KR102405695B1 (ko) * 2015-08-31 2022-06-03 엘지디스플레이 주식회사 유기 발광 표시 장치 및 그 제조 방법
LT3368507T (lt) 2015-10-28 2023-03-10 Acuitas Therapeutics Inc. Nauji lipidai ir lipidų nanodalelių kompozicijos, skirtos nukleorūgščių tiekimui
EP3373979A1 (en) * 2015-11-12 2018-09-19 Pfizer Inc Tissue-specific genome engineering using crispr-cas9
CA3006618A1 (en) * 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
JP7080172B2 (ja) 2015-12-10 2022-06-03 モデルナティエックス インコーポレイテッド 治療薬の送達のための組成物及び方法
WO2017136794A1 (en) * 2016-02-03 2017-08-10 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
BR112018069795A2 (pt) 2016-03-30 2019-01-29 Intellia Therapeutics, Inc. formulações de nanopartículas lipídicas para componentes de crispr/cas
US10767175B2 (en) * 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
US10927383B2 (en) * 2016-06-30 2021-02-23 Ethris Gmbh Cas9 mRNAs
WO2018119514A1 (en) 2016-12-28 2018-07-05 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
GB2573062A (en) * 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
SG10202106058WA (en) * 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
KR102569848B1 (ko) * 2016-12-23 2023-08-25 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
EA201991804A1 (ru) * 2017-02-01 2020-02-07 МОДЕРНАТиЭкс, ИНК. ИММУНОМОДУЛИРУЮЩИЕ ТЕРАПЕВТИЧЕСКИЕ КОМПОЗИЦИИ мРНК, КОДИРУЮЩЕЙ ПЕПТИДЫ С АКТИВИРУЮЩЕЙ ОНКОГЕННОЙ МУТАЦИЕЙ
TW201839136A (zh) * 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
JP7277052B2 (ja) 2017-02-22 2023-05-18 クリスパー セラピューティクス アーゲー プロタンパク質転換酵素サブチリシン／ケキシン９型（ｐｃｓｋ９）関連障害の処置のための組成物および方法
CN119351474A (zh) * 2017-02-22 2025-01-24 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
WO2018183808A1 (en) * 2017-03-31 2018-10-04 Agenovir Corporation Antiviral therapeutic
WO2018191719A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipid delivery of therapeutic agents to adipose tissue
EP3645054A4 (en) 2017-06-26 2021-03-31 The Broad Institute, Inc. CRISPR / CAS-ADENINE DESAMINASE COMPOSITIONS, TARGETED NUCLEIC ACID EDITING SYSTEMS AND METHODS
JP2020534795A (ja) * 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
EP3437650A1 (en) 2017-07-31 2019-02-06 Accanis Biotech F&E GmbH & Co KG Treatment of local skin hypotrophy conditions
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
AR113154A1 (es) 2017-09-29 2020-01-29 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para edición del genoma
KR20250042147A (ko) 2017-09-29 2025-03-26 인텔리아 테라퓨틱스, 인크. 제제
KR102832035B1 (ko) 2017-09-29 2025-07-09 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
WO2019213183A1 (en) 2018-04-30 2019-11-07 The Trustees Of The University Of Pennsylvania In utero crispr-mediated therapeutic editing of genes
CN112601816B (zh) 2018-05-11 2024-12-17 比姆医疗股份有限公司 使用可编程碱基编辑器系统遏止病原性突变的方法
US12157760B2 (en) * 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US20210301274A1 (en) * 2018-09-07 2021-09-30 Beam Therapeutics Inc. Compositions and Methods for Delivering a Nucleobase Editing System
IL322436A (en) 2018-09-21 2025-09-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
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
WO2021056302A1 (en) * 2019-09-26 2021-04-01 Syngenta Crop Protection Ag Methods and compositions for dna base editing
IL296133A (en) * 2020-03-04 2022-11-01 Verve Therapeutics Inc Preparations and methods for administering targeted RNA
GB2632564B (en) 2020-04-09 2025-06-18 Verve Therapeutics Inc Base editing of angptl3 and methods of using same for treatment of disease
WO2022060871A1 (en) * 2020-09-15 2022-03-24 Verve Therapeutics, Inc. Lipid formulations for gene editing

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