BR112021004929A2 - peptídeos catiônicos terciários amino-lipidados para entrega de ácido nucleico - Google Patents

peptídeos catiônicos terciários amino-lipidados para entrega de ácido nucleico Download PDF

Info

Publication number
BR112021004929A2
BR112021004929A2 BR112021004929-0A BR112021004929A BR112021004929A2 BR 112021004929 A2 BR112021004929 A2 BR 112021004929A2 BR 112021004929 A BR112021004929 A BR 112021004929A BR 112021004929 A2 BR112021004929 A2 BR 112021004929A2
Authority
BR
Brazil
Prior art keywords
amino
lipidated
pegylated
tertiary
cationic peptide
Prior art date
Application number
BR112021004929-0A
Other languages
English (en)
Portuguese (pt)
Inventor
Colin James MCKINLAY
Original Assignee
Nutcracker 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 Nutcracker Therapeutics, Inc. filed Critical Nutcracker Therapeutics, Inc.
Publication of BR112021004929A2 publication Critical patent/BR112021004929A2/pt

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2/00—Peptides of undefined number of amino acids; Derivatives thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/10—Tetrapeptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/10—Tetrapeptides
    • C07K5/1002—Tetrapeptides with the first amino acid being neutral
    • C07K5/1005—Tetrapeptides with the first amino acid being neutral and aliphatic
    • C07K5/1008—Tetrapeptides with the first amino acid being neutral and aliphatic the side chain containing 0 or 1 carbon atoms, i.e. Gly, Ala
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • 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
    • B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Optics & Photonics (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Nanotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Inorganic Chemistry (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • 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)
BR112021004929-0A 2018-09-28 2019-09-27 peptídeos catiônicos terciários amino-lipidados para entrega de ácido nucleico BR112021004929A2 (pt)

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

Publications (1)

Publication Number Publication Date
BR112021004929A2 true BR112021004929A2 (pt) 2021-06-01

Family

ID=69952323

Family Applications (2)

Application Number Title Priority Date Filing Date
BR112021004929-0A BR112021004929A2 (pt) 2018-09-28 2019-09-27 peptídeos catiônicos terciários amino-lipidados para entrega de ácido nucleico
BR112021005815-0A BR112021005815A2 (pt) 2018-09-28 2019-09-27 formulações de nanoparticulas lipídicas compreendendo compostos peptídicos catiônicos lipidados para liberação de ácido nucleico

Family Applications After (1)

Application Number Title Priority Date Filing Date
BR112021005815-0A BR112021005815A2 (pt) 2018-09-28 2019-09-27 formulações de nanoparticulas lipídicas compreendendo compostos peptídicos catiônicos lipidados para liberação de ácido nucleico

Country Status (14)

Country Link
US (4) US20210322575A1 (https=)
EP (2) EP3856757A4 (https=)
JP (2) JP7418419B2 (https=)
KR (2) KR20210068093A (https=)
CN (2) CN112789031B (https=)
AU (2) AU2019351268A1 (https=)
BR (2) BR112021004929A2 (https=)
CA (2) CA3110914A1 (https=)
IL (2) IL281135B2 (https=)
MX (2) MX2021003420A (https=)
MY (1) MY204458A (https=)
SG (2) SG11202102164PA (https=)
WO (2) WO2020069442A1 (https=)
ZA (2) ZA202101376B (https=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3856757A4 (en) * 2018-09-28 2022-06-29 Nutcracker Therapeutics, Inc. Tertiary amino lipidated cationic peptides for nucleic acid delivery
AU2020329156A1 (en) * 2019-08-09 2022-03-03 Nutcracker Therapeutics, Inc. Lipidated cationic peptide-PEG compositions for nucleic acid delivery
TW202206083A (zh) * 2020-04-22 2022-02-16 美商胡桃夾子治療公司 mRNA治療奈米粒子
WO2022032058A1 (en) 2020-08-07 2022-02-10 Nutcracker Therapeutics, Inc. Multicomponent delivery systems for polyanionic cargo compound delivery
NL2026825B1 (en) 2020-08-07 2022-04-05 Nutcracker Therapeutics Inc Multicomponent delivery system for polyanionic cargo compound delivery
IL303434A (en) * 2020-12-07 2023-08-01 Tufts College Lipidoid preparations and methods of using them
US20220378702A1 (en) * 2021-05-05 2022-12-01 Arcturus Therapeutics, Inc. Peptide-lipid conjugates
NL2029057B1 (en) 2021-08-06 2023-02-21 Nutcracker Therapeutics Inc Compositions comprising hydroxyethyl-capped cationic peptoids
CA3227513A1 (en) 2021-08-06 2023-02-09 Colin James MCKINLAY 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
JP2025531758A (ja) 2022-09-09 2025-09-25 ナッツクラッカー セラピューティクス， インコーポレイテッド 核酸送達のための２－アミノプロパン－１，３－ジオールでキャップされたカチオン性ペプトイド
US20250026779A1 (en) 2023-06-29 2025-01-23 Nutcracker Therapeutics, Inc. Ethyl modified rna caps and methods of use
CN121793991A (zh) * 2023-09-05 2026-04-03 韩国生命工学研究院 使用脂质纳米颗粒的核酸递送系统
WO2025137277A1 (en) 2023-12-19 2025-06-26 Nutcracker Therapeutics, Inc. Degradable peptoids
TW202543585A (zh) 2024-03-08 2025-11-16 美商健臻公司 脂質奈米粒子
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

Family Cites Families (47)

* 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
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6191115B1 (en) * 1998-08-12 2001-02-20 Abbott Laboratories C-terminus modified heptapeptide LHRH analogs
ATE388226T1 (de) * 1999-08-27 2008-03-15 Novartis Vaccines & Diagnostic Chimerische antisense-oligonukleotide und zelltransfektions-zusammensetzungen davon
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
CA2532228C (en) * 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP1709195B1 (en) * 2003-12-19 2014-01-22 Novartis Vaccines and Diagnostics, Inc. Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
ITMI20061607A1 (it) * 2006-08-09 2008-02-10 Maria Vincenza Carriero Peptidi con attivita farmacologica
CN106267902B (zh) 2007-06-04 2019-08-20 高压生物科学公司 分子的压力提高的提取和分配
CA2721333C (en) 2008-04-15 2020-12-01 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
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
US20110305769A1 (en) * 2008-11-17 2011-12-15 Enzon Pharmaceuticals, Inc. Branched cationic lipids for nucleic acids delivery system
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
US20110053829A1 (en) * 2009-09-03 2011-03-03 Curevac Gmbh Disulfide-linked polyethyleneglycol/peptide conjugates for the transfection of nucleic acids
CN107028886A (zh) 2009-11-04 2017-08-11 不列颠哥伦比亚大学 含有核酸的脂质粒子及相关的方法
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation 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
DK2631291T3 (da) 2010-10-22 2019-06-11 Olix Pharmaceuticals Inc Nukleinsyremolekyler, der inducerer rna-interferens, og anvendelser deraf
EP2686342A2 (en) * 2011-04-12 2014-01-22 The Government of The United States of America, as represented by The Secretary, Department of Health and Human Services Peptide mimetic ligands of polo-like kinase 1 polo box domain and methods of use
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
US9579338B2 (en) 2011-11-04 2017-02-28 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
AU2013240051B2 (en) 2012-03-27 2017-11-30 Sirna Therapeutics, Inc. Diether based biodegradable cationic lipids for siRNA delivery
KR101480055B1 (ko) 2012-04-04 2015-01-09 주식회사 삼양바이오팜 음이온성 약물 전달체의 제조 방법
EP2858679B2 (en) 2012-06-08 2024-06-05 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
WO2013192310A1 (en) 2012-06-19 2013-12-27 Massachusetts Institute Of Technology Mass production and size control of nanoparticles through controlled microvortices
TW201408625A (zh) 2012-07-06 2014-03-01 Kyowa Hakko Kirin Co Ltd 陽離子性脂質
WO2014089239A1 (en) 2012-12-07 2014-06-12 Alnylam Pharmaceuticals, Inc. Improved nucleic acid lipid particle formulations
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
KR102354389B1 (ko) 2013-08-21 2022-01-20 큐어백 아게 Rna―암호화된 단백질의 발현을 증가시키는 방법
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
EP3155116A4 (en) 2014-06-10 2017-12-27 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
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
HUE053990T2 (hu) 2015-07-22 2021-08-30 Nitto Denko Corp Nanorészecske liofilizált formák készítményei és eljárások
EP4011451A1 (en) 2015-10-22 2022-06-15 ModernaTX, Inc. Metapneumovirus mrna vaccines
US10758588B2 (en) * 2015-12-07 2020-09-01 Purdue Research Foundation Inhibitors for proliferating cell nuclear antigen and uses
BR112018008344A2 (pt) 2015-12-13 2018-10-30 Nitto Denko Corp molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição
WO2018064755A1 (en) * 2016-10-03 2018-04-12 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
CA3043033A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Improved ice-based lipid nanoparticle formulation for delivery of mrna
WO2018107061A1 (en) 2016-12-09 2018-06-14 Board Of Regents, The University Of Texas System Hybrid exosomal-polymeric (hexpo) nano-platform for delivery of rnai therapeutics
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
EP3856757A4 (en) * 2018-09-28 2022-06-29 Nutcracker Therapeutics, Inc. Tertiary amino lipidated cationic peptides for nucleic acid delivery

Also Published As

Publication number Publication date
EP3856146A1 (en) 2021-08-04
CN112789031A (zh) 2021-05-11
US11446394B2 (en) 2022-09-20
MY204458A (en) 2024-08-29
JP7418419B2 (ja) 2024-01-19
IL281135A (en) 2021-04-29
JP7430713B2 (ja) 2024-02-13
US20210371458A1 (en) 2021-12-02
AU2019347774A1 (en) 2021-03-25
US20210369867A1 (en) 2021-12-02
IL281135B2 (en) 2024-12-01
AU2019351268A1 (en) 2021-03-18
BR112021005815A2 (pt) 2021-06-29
US20210324002A1 (en) 2021-10-21
KR20210068093A (ko) 2021-06-08
JP2022501408A (ja) 2022-01-06
EP3856757A4 (en) 2022-06-29
WO2020069442A1 (en) 2020-04-02
CN112805294A (zh) 2021-05-14
CA3110914A1 (en) 2020-04-02
CN112789031B (zh) 2023-03-24
ZA202101465B (en) 2024-06-26
EP3856146A4 (en) 2022-07-06
ZA202101376B (en) 2024-06-26
MX2021003421A (es) 2021-06-15
SG11202102163SA (en) 2021-04-29
JP2022502451A (ja) 2022-01-11
CA3111476A1 (en) 2020-04-02
SG11202102164PA (en) 2021-04-29
US20210322575A1 (en) 2021-10-21
WO2020069445A1 (en) 2020-04-02
US11701434B2 (en) 2023-07-18
KR20210068092A (ko) 2021-06-08
IL281653A (en) 2021-05-31
EP3856757A1 (en) 2021-08-04
IL281135B1 (en) 2024-08-01
MX2021003420A (es) 2021-07-16

Similar Documents

Publication Publication Date Title
BR112021004929A2 (pt) peptídeos catiônicos terciários amino-lipidados para entrega de ácido nucleico
JP7657773B2 (ja) 核酸送達のための脂質化カチオン性ペプチド－ｐｅｇ組成物
US20110212028A1 (en) Chemically modified cell-penetrating peptides for improved delivery of gene modulating compounds
CN102027043A (zh) 生物可降解的交联支链聚(亚烷基亚胺)
SK63198A3 (en) Lipopolyamines as transfection agents and pharmaceutical uses thereof
US20130011448A1 (en) System for improved delivery of gene modulating compounds
JP2025538575A (ja) 核酸送達用シクロアルカン系脂質化合物及びそれを含む脂質ナノ粒子
KR20240159310A (ko) 신규한 이온화지질을 이용한 지질나노입자 조성물
HK40050965A (en) Tertiary amino lipidated cationic peptides for nucleic acid delivery
HK40050963B (en) Lipid nanoparticle formulations comprising lipidated cationic peptide compounds for nucleic acid delivery
HK40050963A (en) Lipid nanoparticle formulations comprising lipidated cationic peptide compounds for nucleic acid delivery
WO2026090175A2 (en) Propargyl amino-ionizable lipid compounds, lipid nanoparticles (lnps), and methods of making and using same for gene editing

Legal Events

Date Code Title Description
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

Free format text: REFERENTE A 6A ANUIDADE.

B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: EM VIRTUDE DO ARQUIVAMENTO PUBLICADO NA RPI 2846 DE 22-07-2025 E CONSIDERANDO AUSENCIA DE MANIFESTACAO DENTRO DOS PRAZOS LEGAIS, INFORMO QUE CABE SER MANTIDO O ARQUIVAMENTO DO PEDIDO DE PATENTE, CONFORME O DISPOSTO NO ARTIGO 12, DA RESOLUCAO 113/2013.