CA3236235A1 - Lipid nanoparticle compositions for delivering circular polynucleotides - Google Patents

Lipid nanoparticle compositions for delivering circular polynucleotides Download PDF

Info

Publication number
CA3236235A1
CA3236235A1 CA3236235A CA3236235A CA3236235A1 CA 3236235 A1 CA3236235 A1 CA 3236235A1 CA 3236235 A CA3236235 A CA 3236235A CA 3236235 A CA3236235 A CA 3236235A CA 3236235 A1 CA3236235 A1 CA 3236235A1
Authority
CA
Canada
Prior art keywords
lipid
pharmaceutical composition
peg
ionizable lipid
ionizable
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3236235A
Other languages
English (en)
French (fr)
Inventor
Allen T. HORHOTA
JungHoon YANG
Kevin KAUFFMAN
Thomas Barnes
Robert Alexander WESSELHOEFT
Amy M. BECKER
Gregory MOTZ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Orna Therapeutics Inc
Original Assignee
Orna 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 Orna Therapeutics Inc filed Critical Orna Therapeutics Inc
Publication of CA3236235A1 publication Critical patent/CA3236235A1/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five 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
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • 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
    • A61K47/06—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
    • A61K47/50—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
    • 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
    • A61K48/0033—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 non-polymeric
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C219/06—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/22—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated the carbon skeleton being further substituted by oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/24—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having more than one carboxyl group bound to the carbon skeleton, e.g. aspartic acid

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Dispersion Chemistry (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3236235A 2021-11-08 2022-11-08 Lipid nanoparticle compositions for delivering circular polynucleotides Pending CA3236235A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163277055P 2021-11-08 2021-11-08
US63/277,055 2021-11-08
PCT/US2022/049313 WO2023081526A1 (en) 2021-11-08 2022-11-08 Lipid nanoparticle compositions for delivering circular polynucleotides

Publications (1)

Publication Number Publication Date
CA3236235A1 true CA3236235A1 (en) 2023-05-11

Family

ID=84537778

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3236235A Pending CA3236235A1 (en) 2021-11-08 2022-11-08 Lipid nanoparticle compositions for delivering circular polynucleotides

Country Status (11)

Country Link
US (4) US12415776B2 (https=)
EP (1) EP4430024A1 (https=)
JP (1) JP2024542155A (https=)
KR (1) KR20240107139A (https=)
CN (1) CN118541346A (https=)
AU (1) AU2022383068A1 (https=)
CA (1) CA3236235A1 (https=)
IL (1) IL312490A (https=)
MX (1) MX2024005521A (https=)
TW (1) TW202334080A (https=)
WO (1) WO2023081526A1 (https=)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118317944A (zh) * 2021-09-14 2024-07-09 雷纳嘉德医疗管理公司 非环状脂质及其使用方法
TW202334080A (zh) 2021-11-08 2023-09-01 美商歐納醫療公司 用於遞送環狀聚核苷酸之脂質奈米粒子組合物
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
WO2024102762A1 (en) * 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102730A1 (en) * 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
AU2023398825A1 (en) 2022-12-15 2025-06-26 Orna Therapeutics, Inc. Circular rna compositions and methods
EP4705481A2 (en) 2023-05-05 2026-03-11 Orna Therapeutics, Inc. Circular rna compositions and methods
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025007148A1 (en) 2023-06-30 2025-01-02 Orna Therapeutics, Inc. Polymer lipid nanoparticle compositions for delivering circular polynucleotides
KR20260046133A (ko) * 2023-07-19 2026-04-06 젠에디트바이오 리미티드 지질 및 지질 나노입자
WO2025045142A1 (en) * 2023-08-29 2025-03-06 Shanghai Circode Biomed Co., Ltd. Circular rna encoding vegf polypeptides, formulations, and methods of uses
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025087303A1 (en) * 2023-10-24 2025-05-01 Mote Therapeutics Inc. Lipid nanoparticle with non-covalent bifunctional binders for active targeting
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
CN117658839A (zh) * 2023-11-22 2024-03-08 武汉大学 一种含羟基可电离脂质及其制备方法与应用
WO2025117969A1 (en) 2023-12-01 2025-06-05 Orna Therapeutics, Inc. Process for manufacturing lipid nanoparticles
WO2025120230A1 (en) 2023-12-07 2025-06-12 T-Curx Gmbh Method of producing leukocytes expressing artificial antigen receptors
TW202547451A (zh) * 2023-12-13 2025-12-16 美商泰德治療公司 可電離陽離子脂質和脂質奈米顆粒及其合成方法和用途
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
WO2025140249A1 (en) * 2023-12-26 2025-07-03 Suzhou Abogen Biosciences Co., Ltd. A circular rna and relevant constructs and a method using the same
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026006593A1 (en) 2024-06-28 2026-01-02 Orna Therapeutics, Inc. Synthetic internal ribosome entry sites
WO2026072709A1 (en) 2024-09-25 2026-04-02 Orna Therapeutics, Inc. Chimeric antigen receptors targeting bcma

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3854480A (en) 1969-04-01 1974-12-17 Alza Corp Drug-delivery system
US3832253A (en) 1973-03-21 1974-08-27 Baxter Laboratories Inc Method of making an inflatable balloon catheter
US4450150A (en) 1973-05-17 1984-05-22 Arthur D. Little, Inc. Biodegradable, implantable drug delivery depots, and method for preparing and using the same
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4667014A (en) 1983-03-07 1987-05-19 Syntex (U.S.A.) Inc. Nonapeptide and decapeptide analogs of LHRH, useful as LHRH antagonists
US4452775A (en) 1982-12-03 1984-06-05 Syntex (U.S.A.) Inc. Cholesterol matrix delivery system for sustained release of macromolecules
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
CA1200416A (en) 1983-05-13 1986-02-11 Societe Des Produits Nestle S.A. Food process
US5019369A (en) 1984-10-22 1991-05-28 Vestar, Inc. Method of targeting tumors in humans
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
IN165717B (https=) 1986-08-07 1989-12-23 Battelle Memorial Institute
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
JPH04167172A (ja) 1990-10-31 1992-06-15 Nec Corp ベクトルプロセッサ
US6319494B1 (en) 1990-12-14 2001-11-20 Cell Genesys, Inc. Chimeric chains for receptor-associated signal transduction pathways
IL104570A0 (en) 1992-03-18 1993-05-13 Yeda Res & Dev Chimeric genes and cells transformed therewith
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US5948902A (en) 1997-11-20 1999-09-07 South Alabama Medical Science Foundation Antisense oligonucleotides to human serine/threonine protein phosphatase genes
FR2824431A1 (fr) 2001-05-03 2002-11-08 Mitsubishi Electric Inf Tech Methode et dispositif de reception de signal
MXPA05000511A (es) 2001-07-12 2005-09-30 Jefferson Foote Anticuepros super humanizados.
EP1412065A2 (en) 2001-07-27 2004-04-28 President And Fellows Of Harvard College Laminar mixing apparatus and methods
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
US20040014194A1 (en) 2002-03-27 2004-01-22 Schering Corporation Beta-secretase crystals and methods for preparing and using the same
WO2006069782A2 (en) 2004-12-27 2006-07-06 Silence Therapeutics Ag. Lipid complexes coated with peg and their use
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
ATE475669T1 (de) 2004-06-29 2010-08-15 Immunocore Ltd Einen modifizierten t-zellen-rezeptor exprimierende zellen
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
EP2046266A4 (en) 2006-07-21 2009-11-04 Massachusetts Inst Technology ENDMODICIFIED POLY (BETA AMINO ESTER) AND ITS USE
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
CA2689042A1 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
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
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
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
US8722082B2 (en) 2008-11-10 2014-05-13 Tekmira Pharmaceuticals Corporation Lipids and compositions for the delivery of therapeutics
US8734853B2 (en) 2008-11-17 2014-05-27 University Of North Texas Health Science Center At Fort Worth HDL particles for delivery of nucleic acids
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
ES2579936T3 (es) 2009-08-20 2016-08-17 Sirna Therapeutics, Inc. Nuevos lípidos catiónicos con diversos grupos de cabeza para el suministro oligonucleotídico
US9222086B2 (en) 2009-09-23 2015-12-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
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
WO2011066651A1 (en) 2009-12-01 2011-06-09 Protiva Biotherapeutics, Inc. Snalp formulations containing antioxidants
EP3296398A1 (en) 2009-12-07 2018-03-21 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
EP2525781A1 (en) 2010-01-22 2012-11-28 Schering Corporation Novel cationic lipids for oligonucleotide delivery
US20130037977A1 (en) 2010-04-08 2013-02-14 Paul A. Burke Preparation of Lipid Nanoparticles
US20130156845A1 (en) 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
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
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2012024526A2 (en) 2010-08-20 2012-02-23 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
LT4226941T (lt) 2010-08-31 2025-01-10 Glaxosmithkline Biologicals Sa Pegilintos liposomos, skirtos imunogeną koduojančios rnr pristatymui
CA2809858C (en) 2010-09-20 2019-11-12 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
CA2811430A1 (en) 2010-09-30 2012-04-05 Merck Sharp & Dohme Corp. Low molecular weight cationic lipids for oligonucleotide delivery
EP3485913A1 (en) 2010-10-21 2019-05-22 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
WO2012162210A1 (en) 2011-05-26 2012-11-29 Merck Sharp & Dohme Corp. Ring constrained cationic lipids for oligonucleotide delivery
ES2795110T3 (es) 2011-06-08 2020-11-20 Translate Bio Inc Lípidos escindibles
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
EP4115875A1 (en) 2011-07-06 2023-01-11 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
MX366055B (es) 2011-08-31 2019-06-26 Novartis Ag Liposomas pegilados para admistracion de acido ribonucleico (arn) que codifica para inmunogeno.
EP3456317B1 (en) 2011-09-27 2025-09-24 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
EP3485875A1 (en) 2011-11-04 2019-05-22 Nitto Denko Corporation Single use system for sterelily producing lipid-nucleic acid particles
WO2013073480A1 (ja) 2011-11-18 2013-05-23 日油株式会社 細胞内動態を改善したカチオン性脂質
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
AU2012347605B2 (en) 2011-12-07 2017-09-21 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
JP6182457B2 (ja) 2011-12-12 2017-08-16 協和発酵キリン株式会社 カチオン性脂質を含有するドラックデリバリーシステムのための脂質ナノ粒子
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
AU2013240051B2 (en) 2012-03-27 2017-11-30 Sirna Therapeutics, Inc. Diether based biodegradable cationic lipids for siRNA delivery
EP2719422B1 (fr) * 2012-10-12 2015-02-18 Sorin CRM SAS Microsonde multipolaire de détection/stimulation implantable
ES2859923T3 (es) 2013-02-28 2021-10-04 Univ Tufts Compuestos de disulfuro para el suministro de agentes farmacéuticos
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
JP6620093B2 (ja) 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
US10426737B2 (en) 2013-12-19 2019-10-01 Novartis Ag Lipids and lipid compositions for the delivery of active agents
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
JP6637988B2 (ja) 2014-11-18 2020-01-29 アークトゥルス セラピューティクス， インコーポレイテッド Ｒｎａ送達のためのイオン化可能カチオン性脂質
US11669953B2 (en) 2015-01-30 2023-06-06 Hitachi High-Tech Corporation Pattern matching device and computer program for pattern matching
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
JP6948313B6 (ja) 2015-09-17 2022-01-14 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物および組成物
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
US9834510B2 (en) 2015-12-30 2017-12-05 Arcturus Therapeutics, Inc. Aromatic ionizable cationic lipid
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
US20180020547A1 (en) 2016-07-13 2018-01-18 Alcatel-Lucent Canada Inc. Underlying recessed component placement
EP4186888B1 (en) 2017-03-15 2025-11-26 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
WO2019081383A1 (en) 2017-10-25 2019-05-02 Universität Zürich EUCARYOTE 4 INITIATION FACTOR RECRUITMENT APTAMERS FOR ENHANCED TRANSLATION
TWI894123B (zh) 2017-12-27 2025-08-21 日商武田藥品工業有限公司 含核酸之脂質奈米粒子及其用途
US20190314291A1 (en) 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
MX2020007944A (es) 2018-02-01 2020-11-24 Tufts College Nanocomplejos similares a los lípidos y sus usos.
WO2020061367A1 (en) * 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2020257611A1 (en) * 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
US20210142281A1 (en) * 2019-11-12 2021-05-13 Airspace Technologies, Inc. Logistical Management System
AU2021205337A1 (en) * 2020-01-10 2022-07-21 Modernatx, Inc. Methods of making tolerogenic dendritic cells
JP7716112B2 (ja) 2020-03-02 2025-07-31 トラスティーズ オブ タフツ カレッジ 免疫細胞へのｉｎ ｖｉｖｏでのｍＲＮＡ送達のためのイミダゾール系合成リピドイド
US12302753B2 (en) * 2020-07-20 2025-05-13 Beijing Summer Sprout Technology Co., Ltd. Organic electroluminescent materials and devices
EP4262819A4 (en) * 2020-12-21 2024-12-04 Beam Therapeutics Inc. NANOMATERIALS WITH ESTER-BOUND ACETALS
US11895119B2 (en) * 2021-01-31 2024-02-06 Salesforce, Inc. Systems, methods, and apparatuses for pre-configured per-tenant isolation in a multi-tenant cloud based computing environment
KR20240013087A (ko) * 2021-05-24 2024-01-30 쑤저우 아보젠 바이오사이언시스 컴퍼니 리미티드 지질 화합물 및 지질 나노입자 조성물
TW202334080A (zh) 2021-11-08 2023-09-01 美商歐納醫療公司 用於遞送環狀聚核苷酸之脂質奈米粒子組合物

Also Published As

Publication number Publication date
EP4430024A1 (en) 2024-09-18
US12077485B2 (en) 2024-09-03
US20240190808A1 (en) 2024-06-13
US20240116852A1 (en) 2024-04-11
US12415776B2 (en) 2025-09-16
US20240189340A1 (en) 2024-06-13
JP2024542155A (ja) 2024-11-13
AU2022383068A1 (en) 2024-05-02
IL312490A (en) 2024-07-01
US20240158336A1 (en) 2024-05-16
TW202334080A (zh) 2023-09-01
CN118541346A (zh) 2024-08-23
WO2023081526A1 (en) 2023-05-11
US12180138B2 (en) 2024-12-31
US12384741B2 (en) 2025-08-12
MX2024005521A (es) 2024-05-21
KR20240107139A (ko) 2024-07-08

Similar Documents

Publication Publication Date Title
US12077485B2 (en) Lipid nanoparticle compositions for delivering circular polynucleotides
US20250283073A1 (en) Circular RNA Compositions and Methods
US20250268828A1 (en) Lipid Nanoparticle Compositions for Delivering Circular Polynucleotides
WO2021189059A2 (en) Circular rna compositions and methods
US12297285B2 (en) Circular RNA encoding chimeric antigen receptors targeting BCMA
US20250304994A1 (en) Circular RNA Compositions and Methods
WO2024102762A1 (en) Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024205657A2 (en) Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102730A1 (en) Lipids and nanoparticle compositions for delivering polynucleotides
HK40115968A (zh) 用於递送环状多核苷酸的脂质纳米颗粒组合物

Legal Events

Date Code Title Description
P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS

Effective date: 20241010

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD

Year of fee payment: 2

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241030

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241030