CN112805294A - 用于核酸递送的叔氨基脂化的阳离子肽 - Google Patents

用于核酸递送的叔氨基脂化的阳离子肽 Download PDF

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CN112805294A
CN112805294A CN201980062070.0A CN201980062070A CN112805294A CN 112805294 A CN112805294 A CN 112805294A CN 201980062070 A CN201980062070 A CN 201980062070A CN 112805294 A CN112805294 A CN 112805294A
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cationic peptide
integer
independently
lipid
pegylated
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C·J·麦金利
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Nutcracker Therapeutics Inc
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Nutcracker Therapeutics Inc
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
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    • A61K48/0041—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 the non-active part being polymeric
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    • C07K5/1002—Tetrapeptides with the first amino acid being neutral
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    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
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    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
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    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
CN201980062070.0A 2018-09-28 2019-09-27 用于核酸递送的叔氨基脂化的阳离子肽 Pending CN112805294A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862738717P 2018-09-28 2018-09-28
US62/738,717 2018-09-28
US201962885036P 2019-08-09 2019-08-09
US62/885,036 2019-08-09
PCT/US2019/053655 WO2020069442A1 (en) 2018-09-28 2019-09-27 Tertiary amino lipidated cationic peptides for nucleic acid delivery

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CN112805294A true CN112805294A (zh) 2021-05-14

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CN201980062070.0A Pending CN112805294A (zh) 2018-09-28 2019-09-27 用于核酸递送的叔氨基脂化的阳离子肽
CN201980062278.2A Expired - Fee Related CN112789031B (zh) 2018-09-28 2019-09-27 用于核酸递送的包含脂化的阳离子肽化合物的脂质纳米颗粒制剂

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US (4) US20210322575A1 (enExample)
EP (2) EP3856146A4 (enExample)
JP (2) JP7430713B2 (enExample)
KR (2) KR20210068093A (enExample)
CN (2) CN112805294A (enExample)
AU (2) AU2019347774A1 (enExample)
BR (2) BR112021004929A2 (enExample)
CA (2) CA3110914A1 (enExample)
IL (2) IL281135B2 (enExample)
MX (2) MX2021003421A (enExample)
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Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210322575A1 (en) * 2018-09-28 2021-10-21 Nutcracker Therapeutics, Inc. Lipid nanoparticle formulations comprising lipidated cationic peptide compounds for nucleic acid delivery
CN114555130A (zh) * 2019-08-09 2022-05-27 胡桃钳医疗公司 用于核酸递送的脂化阳离子肽-peg组合物
BR112022021380A2 (pt) * 2020-04-22 2022-12-06 Nutcracker Therapeutics Inc Nanopartículas de rna mensageiro para tratamento
NL2026825B1 (en) 2020-08-07 2022-04-05 Nutcracker Therapeutics Inc Multicomponent delivery system for polyanionic cargo compound delivery
US20230293706A1 (en) 2020-08-07 2023-09-21 Nutcracker Therapeutics, Inc. Multicomponent Delivery Systems for Polyanionic Cargo Compound Delivery
IL303434A (en) * 2020-12-07 2023-08-01 Tufts College Lipidoid preparations and methods of using them
WO2022235923A2 (en) * 2021-05-05 2022-11-10 Arcturus Therapeutics, Inc. Peptide-lipid conjugates
US20240307526A1 (en) 2021-08-06 2024-09-19 Nutcracker Therapeutics, Inc. Compositions comprising hydroxyethyl-capped cationic peptoids
NL2029057B1 (en) 2021-08-06 2023-02-21 Nutcracker Therapeutics Inc Compositions comprising hydroxyethyl-capped cationic peptoids
CN114778712B (zh) * 2022-03-21 2023-06-02 天津键凯科技有限公司 一种聚乙二醇脂质及含有该脂质的脂质纳米粒含量的检测方法
EP4584278A1 (en) 2022-09-09 2025-07-16 Nutcracker Therapeutics, Inc. 2-aminopropane-1,3-diol-capped cationic peptoids for nucleic acid delivery
CN121752579A (zh) 2023-06-29 2026-03-27 胡桃夹子治疗公司 乙基修饰的rna帽和使用方法
KR20250036032A (ko) * 2023-09-05 2025-03-13 한국생명공학연구원 지질 나노 입자를 이용한 핵산 전달 시스템
WO2025137277A1 (en) 2023-12-19 2025-06-26 Nutcracker Therapeutics, Inc. Degradable peptoids
US20250381150A1 (en) 2024-03-08 2025-12-18 Genzyme Corporation Lipid nanoparticles
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof
WO2026142951A1 (en) 2024-12-23 2026-07-02 Genzyme Corporation Pharmaceutical system

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6191115B1 (en) * 1998-08-12 2001-02-20 Abbott Laboratories C-terminus modified heptapeptide LHRH analogs
US20060019912A1 (en) * 2003-12-19 2006-01-26 Chiron Corporation Cell transfecting formulations of small interfering RNA related compositions and methods of making and use
US20110230397A1 (en) * 2006-08-09 2011-09-22 Maria Vincenza Carriero Peptides having pharmacological activity for treating disorders associated with altered cell migration, such as cancer
US20140142044A1 (en) * 2011-04-12 2014-05-22 The United States of America, as represented by the Secretary, Department of Health & Human Servic Peptide mimetic ligands of polo-like kinase 1 polo box domain and methods of use

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
JP4652648B2 (ja) 1999-08-27 2011-03-16 ノバルティス バクシンズ アンド ダイアグノスティックス，インコーポレーテッド キメラアンチセンスオリゴヌクレオチド、およびその細胞トランスフェクション処方物
JP2004535388A (ja) * 2001-04-30 2004-11-25 ターゲティッド ジェネティクス コーポレイション 脂質含有薬物送達複合体およびそれらの生成方法
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
AU2004257373B2 (en) * 2003-07-16 2011-03-24 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
US9458190B2 (en) 2007-06-04 2016-10-04 Pressure Biosciences, Inc. Extraction and partitioning of molecules
EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. Novel lipid formulations for nucleic acid delivery
WO2009132131A1 (en) * 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
EP3699172A3 (en) 2008-11-10 2020-11-18 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
EP2350296A4 (en) * 2008-11-17 2013-04-03 Enzon Pharmaceuticals Inc BRANCHED CATIONIC LIPIDS FOR THE SYSTEM FOR INTRODUCING NUCLEIC ACIDS
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US20110053829A1 (en) * 2009-09-03 2011-03-03 Curevac Gmbh Disulfide-linked polyethyleneglycol/peptide conjugates for the transfection of nucleic acids
RU2573409C2 (ru) 2009-11-04 2016-01-20 Дзе Юниверсити Оф Бритиш Коламбиа Содержащие нуклеиновые кислоты липидные частицы и относящиеся к ним способы
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases
DK2575767T3 (en) 2010-06-04 2017-03-13 Sirna Therapeutics Inc HOWEVER UNKNOWN LOW MOLECULAR CATIONIC LIPIDS TO PROCESS OIGONUCLEOTIDES
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
CN103328633B (zh) 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
CA2853689C (en) 2011-11-04 2020-06-30 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
CA2867323C (en) 2012-03-27 2020-07-07 Sirna Therapeutics, Inc. Diether based biodegradable cationic lipids for sirna delivery
ES2836279T3 (es) 2012-04-04 2021-06-24 Samyang Biopharmaceuticals Método de preparación de una composición para administrar un fármaco aniónico
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
WO2013192310A1 (en) 2012-06-19 2013-12-27 Massachusetts Institute Of Technology Mass production and size control of nanoparticles through controlled microvortices
TW201726599A (zh) 2012-07-06 2017-08-01 協和醱酵麒麟有限公司 陽離子性脂質
AU2013355258A1 (en) 2012-12-07 2015-06-11 Alnylam Pharmaceuticals, Inc. Improved nucleic acid lipid particle formulations
US10266559B2 (en) 2013-03-14 2019-04-23 Translate Bio, Inc. Ribonucleic acids with 4′-thio-modified nucleotides and related methods
SG10201801428RA (en) 2013-08-21 2018-03-28 Curevac Ag Method for increasing expression of rna-encoded proteins
HRP20220070T1 (hr) 2014-04-23 2022-04-01 Modernatx, Inc. Cjepiva nukleinske kiseline
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
ES2555160B1 (es) 2014-06-24 2016-10-25 Aptus Biotech, S.L. Aptámeros específicos de TLR-4 y usos de los mismos
EP3865576A1 (en) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
CN113679689B (zh) 2015-07-22 2023-09-01 日东电工株式会社 用于纳米粒冷冻干燥物形式的组合物及方法
DE20164728T1 (de) 2015-10-22 2021-09-30 Modernatx, Inc. Impfstoffe gegen atemwegsvirus
WO2017100154A1 (en) * 2015-12-07 2017-06-15 Purdue Research Foundation Inhibitors for proliferating cell nuclear antigen and uses
AU2016371624B2 (en) 2015-12-13 2020-08-27 Nitto Denko Corporation siRNA structures for high activity and reduced off target
WO2018064755A1 (en) * 2016-10-03 2018-04-12 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
CN116549671A (zh) 2016-11-10 2023-08-08 川斯勒佰尔公司 用于递送mrna的改进的基于ice的脂质纳米颗粒制剂
US12564558B2 (en) 2016-12-09 2026-03-03 Board Of Regents, The University Of Texas System Hybrid exosomal-polymeric (hexpo) nano-platform for delivery of RNAi therapeutics
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
US20210322575A1 (en) * 2018-09-28 2021-10-21 Nutcracker Therapeutics, Inc. Lipid nanoparticle formulations comprising lipidated cationic peptide compounds for nucleic acid delivery

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6191115B1 (en) * 1998-08-12 2001-02-20 Abbott Laboratories C-terminus modified heptapeptide LHRH analogs
US20060019912A1 (en) * 2003-12-19 2006-01-26 Chiron Corporation Cell transfecting formulations of small interfering RNA related compositions and methods of making and use
US20110230397A1 (en) * 2006-08-09 2011-09-22 Maria Vincenza Carriero Peptides having pharmacological activity for treating disorders associated with altered cell migration, such as cancer
US20140142044A1 (en) * 2011-04-12 2014-05-22 The United States of America, as represented by the Secretary, Department of Health & Human Servic Peptide mimetic ligands of polo-like kinase 1 polo box domain and methods of use

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