AU2009305639B2 - Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics - Google Patents

Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics Download PDF

Info

Publication number
AU2009305639B2
AU2009305639B2 AU2009305639A AU2009305639A AU2009305639B2 AU 2009305639 B2 AU2009305639 B2 AU 2009305639B2 AU 2009305639 A AU2009305639 A AU 2009305639A AU 2009305639 A AU2009305639 A AU 2009305639A AU 2009305639 B2 AU2009305639 B2 AU 2009305639B2
Authority
AU
Australia
Prior art keywords
alkyl
rna
amino acid
composition
nucleic acid
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.)
Ceased
Application number
AU2009305639A
Other languages
English (en)
Other versions
AU2009305639A1 (en
Inventor
Roger C. Adami
Jaya S. Giyanani
Pierrot Harvie
Michael E. Houston
Rachel E. Johns
Barry A. Polisky
Michael V. Templin
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.)
Marina Biotech Inc
Original Assignee
Marina Biotech 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 Marina Biotech Inc filed Critical Marina Biotech Inc
Publication of AU2009305639A1 publication Critical patent/AU2009305639A1/en
Application granted granted Critical
Publication of AU2009305639B2 publication Critical patent/AU2009305639B2/en
Priority to AU2016222371A priority Critical patent/AU2016222371A1/en
Priority to AU2018204605A priority patent/AU2018204605A1/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • A61K9/1277—Preparation processes; Proliposomes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/10—Drugs for disorders of the urinary system of the bladder
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Dispersion Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Oncology (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Pain & Pain Management (AREA)
  • Immunology (AREA)
AU2009305639A 2008-10-16 2009-10-16 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics Ceased AU2009305639B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2016222371A AU2016222371A1 (en) 2008-10-16 2016-08-31 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
AU2018204605A AU2018204605A1 (en) 2008-10-16 2018-06-25 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US10606208P 2008-10-16 2008-10-16
US61/106,062 2008-10-16
US16737909P 2009-04-07 2009-04-07
US61/167,379 2009-04-07
PCT/US2009/060930 WO2010045512A2 (en) 2008-10-16 2009-10-16 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2016222371A Division AU2016222371A1 (en) 2008-10-16 2016-08-31 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics

Publications (2)

Publication Number Publication Date
AU2009305639A1 AU2009305639A1 (en) 2010-04-22
AU2009305639B2 true AU2009305639B2 (en) 2016-06-23

Family

ID=41560381

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2009305639A Ceased AU2009305639B2 (en) 2008-10-16 2009-10-16 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
AU2016222371A Abandoned AU2016222371A1 (en) 2008-10-16 2016-08-31 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
AU2018204605A Abandoned AU2018204605A1 (en) 2008-10-16 2018-06-25 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2016222371A Abandoned AU2016222371A1 (en) 2008-10-16 2016-08-31 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
AU2018204605A Abandoned AU2018204605A1 (en) 2008-10-16 2018-06-25 Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics

Country Status (12)

Country Link
US (3) US20100112042A1 (enExample)
EP (2) EP2349210B1 (enExample)
JP (2) JP2012505913A (enExample)
KR (1) KR20110071017A (enExample)
CN (2) CN102231979B (enExample)
AU (3) AU2009305639B2 (enExample)
BR (1) BRPI0920171A2 (enExample)
CA (1) CA2739046A1 (enExample)
IL (1) IL212329A0 (enExample)
SG (1) SG196818A1 (enExample)
WO (1) WO2010045512A2 (enExample)
ZA (1) ZA201102319B (enExample)

Families Citing this family (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006138380A2 (en) 2005-06-15 2006-12-28 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
JP5475643B2 (ja) 2007-05-04 2014-04-16 マリーナ バイオテック，インコーポレイテッド アミノ酸脂質およびその使用
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
WO2010059829A2 (en) * 2008-11-19 2010-05-27 Mdrna, Inc. Compositions and methods for triggered release rna therapeutics
EP2496260A1 (en) 2009-11-04 2012-09-12 Marina Biotech, Inc. Activity generating delivery molecules
EP3403647A1 (en) 2009-12-01 2018-11-21 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
WO2012027675A2 (en) 2010-08-26 2012-03-01 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
WO2012112730A2 (en) * 2011-02-15 2012-08-23 Merrimack Pharmaceuticals, Inc. Compositions and methods for delivering nucleic acid to a cell
US9238716B2 (en) 2011-03-28 2016-01-19 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
US20140206753A1 (en) 2011-06-08 2014-07-24 Shire Human Genetic Therapies, Inc. Lipid nanoparticle compositions and methods for mrna delivery
EP3533873A1 (en) 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
PE20181541A1 (es) 2011-10-27 2018-09-26 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco
CA2853689C (en) * 2011-11-04 2020-06-30 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
WO2013173638A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
EP2850188A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING THE EXPRESSION OF THE HEMOGLOBIN GENE FAMILIES
CN104583401A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节atp2a2表达的组合物和方法
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
JP2015518713A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド Ｕｔｒｎ発現を調節するための組成物及び方法
JP2015518712A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド Ｍｅｃｐ２発現を調節するための組成物及び方法
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
WO2014043544A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
CA2904151C (en) 2013-03-14 2023-09-12 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
CN103172701B (zh) * 2013-03-18 2015-09-30 中国科学院过程工程研究所 一种新型穿膜肽及其应用
HUE040425T2 (hu) 2013-04-30 2019-03-28 Delta Fly Pharma Inc Liposzóma topikális adagoláshoz és alkalmazása
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
WO2015048020A2 (en) * 2013-09-24 2015-04-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for the manufacture of lipid nanoparticles
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
WO2015061491A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
DK4023249T3 (da) 2014-04-23 2025-01-13 Modernatx Inc Nukleinsyrevacciner
MX373952B (es) 2014-04-25 2020-07-13 Shire Human Genetic Therapies Métodos de purificación de arn mensajero.
CN106659731A (zh) 2014-05-30 2017-05-10 夏尔人类遗传性治疗公司 用于递送核酸的可生物降解脂质
UA121863C2 (uk) 2014-06-24 2020-08-10 Транслейт Байо, Інк. Стереохімічно збагачені композиції для доставки нуклеїнових кислот
US9840479B2 (en) 2014-07-02 2017-12-12 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
AU2015337909B2 (en) * 2014-10-30 2018-12-13 Delta-Fly Pharma, Inc. New production method of lipoplex for local administration and antitumor drug using lipoplex
JP6373749B2 (ja) 2014-12-19 2018-08-15 富士フイルム株式会社 リポソームの製造方法及びリポソーム製造装置
RS64331B1 (sr) 2015-06-19 2023-08-31 Massachusetts Inst Technology Alkenil supstituisani 2,5-piperazindioni i njihova primena u sastavima za isporuku agensa u organizam ili ćeliju subjekta
US11564893B2 (en) * 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
MA46316A (fr) 2015-10-22 2021-03-24 Modernatx Inc Vaccin contre le cytomégalovirus humain
US9901649B2 (en) 2016-05-03 2018-02-27 International Business Machines Corporation Block copolymers for therapeutic drug delivery
WO2018075980A1 (en) 2016-10-21 2018-04-26 Modernatx, Inc. Human cytomegalovirus vaccine
KR101797168B1 (ko) * 2016-11-10 2017-11-13 주식회사 바이오셀트란 신규의 프로테인 트랜스덕션 도메인을 함유하는 구형 구조체
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
CN110603330B (zh) * 2017-04-28 2023-09-05 协和麒麟株式会社 寡核苷酸衍生物或其盐
MA49138A (fr) 2017-05-16 2020-03-25 Translate Bio Inc Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr
US11786607B2 (en) 2017-06-15 2023-10-17 Modernatx, Inc. RNA formulations
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
BR112020021037A2 (pt) * 2018-04-25 2021-01-19 Ethris Gmbh Composição, composição sólida, processo para a preparação de uma composição, método para preservar uma formulação de nano ou micropartícula de um agente terapeuticamente ativo, uso de um composto selecionado de alcanos c3-c5 substituídos com um ou dois grupos hidróxi, e, dispositivo
WO2020041793A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
WO2020154594A2 (en) * 2019-01-25 2020-07-30 Agenovir Corporation Compositions and methods for treatment of human papillomavirus
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
WO2021127394A2 (en) * 2019-12-20 2021-06-24 Translate Bio, Inc. Rectal delivery of messenger rna
EP3915544A1 (en) * 2020-05-25 2021-12-01 Leon-Nanodrugs GmbH Method for producing a liposome dispersion
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
EP4412591A1 (en) * 2021-10-06 2024-08-14 leon-nanodrugs GmbH Method for preparing lipid nanoparticles
CN114569703B (zh) * 2022-02-25 2023-06-13 纽柏抗衰老国际医生集团(广州)有限公司 一种助睡眠的脂质体包裹复合氨基酸的组合物及制备方法
CN116370437A (zh) * 2023-05-26 2023-07-04 北京因诺惟康医药科技有限公司 包含胆固醇琥珀酸单酯的核酸脂质纳米颗粒组合物及其用途
WO2025207519A1 (en) * 2024-03-23 2025-10-02 Texas Tech University System Biocompatible and biodegradable polymer nanodiscs as lipoprotein-mimicking nanocarriers with high stability and long circulation time for drug delivery

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004002453A1 (en) * 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Method and apparatus for producing liposomes
US20050064026A1 (en) * 2003-09-05 2005-03-24 Boehringer Ingelheim Pharma Gmbh & Co. Kg Method for preparing homogenous liposomes and lipoplexes
WO2007107304A2 (en) * 2006-03-17 2007-09-27 Novosom Ag An efficient method for loading amphoteric liposomes with nucleic acid active substances
US20080317839A1 (en) * 2007-05-04 2008-12-25 Nastech Pharmaceutical Company Inc. Amino acid lipids and uses thereof

Family Cites Families (86)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3272873D1 (en) 1981-06-04 1986-10-02 Pharmasol Corp Pressurized container with dispensing pump
US5208036A (en) 1985-01-07 1993-05-04 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
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
CH671514A5 (enExample) * 1986-12-03 1989-09-15 Induchem Ag
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
AU630076B2 (en) 1987-09-21 1992-10-22 Gen-Probe Incorporated Non-nucleotide linking reagents for nucleotide probes
US5563250A (en) 1987-12-02 1996-10-08 Neorx Corporation Cleavable conjugates for the delivery and release of agents in native form
WO1991003162A1 (en) 1989-08-31 1991-03-21 City Of Hope Chimeric dna-rna catalytic sequences
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5962219A (en) 1990-06-11 1999-10-05 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: chemi-selex
DE69123979T2 (de) 1990-10-12 1997-04-30 Max Planck Gesellschaft Abgeänderte ribozyme
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5505931A (en) 1993-03-04 1996-04-09 The Dow Chemical Company Acid cleavable compounds, their preparation and use as bifunctional acid-labile crosslinking agents
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
WO1995006731A2 (en) 1993-09-02 1995-03-09 Ribozyme Pharmaceuticals, Inc. Non-nucleotide containing enzymatic nucleic acid
EP0725788B1 (en) 1993-10-27 1998-12-16 Ribozyme Pharmaceuticals, Inc. 2'-amido and 2'-peptido modified oligonucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US6447796B1 (en) 1994-05-16 2002-09-10 The United States Of America As Represented By The Secretary Of The Army Sustained release hydrophobic bioactive PLGA microspheres
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
DE69527206T2 (de) 1994-09-30 2003-02-27 Inex Pharmaceuticals Corp., Vancouver Mittel zum einbringen polyanionischer materialien in zellen
US5681940A (en) 1994-11-02 1997-10-28 Icn Pharmaceuticals Sugar modified nucleosides and oligonucleotides
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
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
DE69634084T2 (de) * 1995-06-07 2005-12-08 Inex Pharmaceuticals Corp. Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
WO1996040961A1 (en) 1995-06-07 1996-12-19 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5705385A (en) * 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
ATE373672T1 (de) 1996-01-16 2007-10-15 Sirna Therapeutics Inc Synthese von methoxynukleoside und enzymatische nukleisäure moleküle
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6509320B1 (en) 1996-10-16 2003-01-21 Icn Pharmaceuticals, Inc. Purine L-nucleosides, analogs and uses thereof
DE69735382T2 (de) 1996-11-04 2006-11-30 Qiagen Gmbh Kationische reagenzien zür transfektion
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
US6001311A (en) 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
US5965542A (en) * 1997-03-18 1999-10-12 Inex Pharmaceuticals Corp. Use of temperature to control the size of cationic liposome/plasmid DNA complexes
US6835395B1 (en) * 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
US6395713B1 (en) 1997-07-23 2002-05-28 Ribozyme Pharmaceuticals, Inc. Compositions for the delivery of negatively charged molecules
US6110745A (en) * 1997-07-24 2000-08-29 Inex Pharmaceuticals Corp. Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
US6734171B1 (en) * 1997-10-10 2004-05-11 Inex Pharmaceuticals Corp. Methods for encapsulating nucleic acids in lipid bilayers
EP1038016A2 (en) 1997-12-16 2000-09-27 Valentis Inc. Needle-free injection of formulated nucleic acid molecules
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
EP2138191A1 (en) 1998-01-05 2009-12-30 University Of Washington Enhanced transport using membrane disruptive agents
US6426086B1 (en) 1998-02-03 2002-07-30 The Regents Of The University Of California pH-sensitive, serum-stable liposomes
EP1071753A2 (en) 1998-04-20 2001-01-31 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
SI20474A (sl) 1998-05-26 2001-08-31 Icn Pharmaceuticals, Inc. Novi nukleozidi, ki imajo bicikličen sladkorni del
EP1159441B8 (en) 1999-03-10 2008-10-29 Marie Curie Cancer Care Delivery of nucleic acids and proteins to cells
US6849272B1 (en) 1999-04-21 2005-02-01 Massachusetts Institute Of Technology Endosomolytic agents and cell delivery systems
US6083482A (en) 1999-05-11 2000-07-04 Icn Pharmaceuticals, Inc. Conformationally locked nucleosides and oligonucleotides
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
EP1102785B1 (en) 1999-06-07 2013-02-13 Arrowhead Research Corporation COMPOSITIONS FOR DRUG DELIVERY USING pH SENSITIVE MOLECULES
AU771706B2 (en) 1999-07-15 2004-04-01 University Of British Columbia, The Methods and apparatus for preparation of lipid vesicles
US7094423B1 (en) * 1999-07-15 2006-08-22 Inex Pharmaceuticals Corp. Methods for preparation of lipid-encapsulated therapeutic agents
US6200599B1 (en) 1999-10-07 2001-03-13 The Regents Of The University Of California Ortho ester lipids
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
EP1203614A1 (de) * 2000-11-03 2002-05-08 Polymun Scientific Immunbiologische Forschung GmbH Verfahren und Vorrichtung zur Herstellung von Lipidvesikeln
US20020130430A1 (en) 2000-12-29 2002-09-19 Castor Trevor Percival Methods for making polymer microspheres/nanospheres and encapsulating therapeutic proteins and other products
US6897196B1 (en) 2001-02-07 2005-05-24 The Regents Of The University Of California pH sensitive lipids based on ortho ester linkers, composition and method
DE10109897A1 (de) * 2001-02-21 2002-11-07 Novosom Ag Fakultativ kationische Liposomen und Verwendung dieser
JP2004525138A (ja) 2001-03-26 2004-08-19 アルザ・コーポレーシヨン 治療剤の改良された細胞内送達のためのリポソーム組成物
WO2002087541A1 (en) * 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US7060498B1 (en) 2001-11-28 2006-06-13 Genta Salus Llc Polycationic water soluble copolymer and method for transferring polyanionic macromolecules across biological barriers
US7141540B2 (en) 2001-11-30 2006-11-28 Genta Salus Llc Cyclodextrin grafted biocompatible amphilphilic polymer and methods of preparation and use thereof
JP2005517433A (ja) 2002-02-20 2005-06-16 サーナ・セラピューティクス・インコーポレイテッド 短干渉核酸（ｓｉＮＡ）を用いるＴＮＦおよびＴＮＦレセプタースーパーファミリー遺伝子発現のＲＮＡ干渉媒介性阻害
US7591994B2 (en) 2002-12-13 2009-09-22 Immunomedics, Inc. Camptothecin-binding moiety conjugates
AU2003237864B2 (en) * 2002-05-15 2008-12-18 California Pacific Medical Center Delivery of nucleic acid-like compounds
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
EP1730280B1 (en) * 2004-03-26 2018-10-24 Curis, Inc. Rna interference modulators of hedgehog signaling and uses thereof
US7514530B2 (en) * 2004-04-26 2009-04-07 Centre National De La Recherche Scientifique Peptide carrier for delivering siRNA into mammalian cells
US20060040882A1 (en) * 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
EP1748760B1 (en) * 2004-05-24 2009-12-23 Polymun Scientific Immunbiologische Forschung GmbH Superloaded liposomes for drug delivery
US20070213293A1 (en) 2005-04-08 2007-09-13 Nastech Pharmaceutical Company Inc. Rnai therapeutic for respiratory virus infection
US20070213257A1 (en) * 2005-08-12 2007-09-13 Nastech Pharmaceutical Company Inc. Compositions and methods for complexes of nucleic acids and peptides
US7579318B2 (en) * 2005-12-06 2009-08-25 Centre De La Recherche De La Scientifique Cell penetrating peptides for intracellular delivery of molecules
US20080213350A1 (en) * 2007-02-20 2008-09-04 Texas Tech University System Encapsulation of nucleic acids in liposomes
US20080299659A1 (en) 2007-03-02 2008-12-04 Nastech Pharmaceutical Company Inc. Nucleic acid compounds for inhibiting apob gene expression and uses thereof
BRPI0811170B8 (pt) 2007-05-22 2021-05-25 Arcturus Therapeutics Inc oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila
JP5697450B2 (ja) * 2007-10-02 2015-04-08 マリーナ バイオテック，インコーポレイテッド 核酸の送達のためのリポペプチド
US11625808B2 (en) 2020-07-30 2023-04-11 Arm Limited Graphics processing systems

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004002453A1 (en) * 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Method and apparatus for producing liposomes
US20050064026A1 (en) * 2003-09-05 2005-03-24 Boehringer Ingelheim Pharma Gmbh & Co. Kg Method for preparing homogenous liposomes and lipoplexes
WO2007107304A2 (en) * 2006-03-17 2007-09-27 Novosom Ag An efficient method for loading amphoteric liposomes with nucleic acid active substances
US20080317839A1 (en) * 2007-05-04 2008-12-25 Nastech Pharmaceutical Company Inc. Amino acid lipids and uses thereof

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MAITANI, Y. et al.; Journal Of Liposome Research 2001, vol. 11, no. 1, 2001, pages 115-125 *
SKALKO, N.. et al.; European Journal Of Pharmaceutical Sciences, vol. 4, no. 6, 1996 , pages 359-366 *

Also Published As

Publication number Publication date
ZA201102319B (en) 2012-09-26
BRPI0920171A2 (pt) 2016-07-12
JP6126072B2 (ja) 2017-05-10
EP2349210A2 (en) 2011-08-03
EP2349210B1 (en) 2015-03-18
CN104382853A (zh) 2015-03-04
EP2902013A1 (en) 2015-08-05
CA2739046A1 (en) 2010-04-22
AU2018204605A1 (en) 2018-07-12
JP2012505913A (ja) 2012-03-08
AU2016222371A1 (en) 2016-09-15
JP2015096529A (ja) 2015-05-21
US20130039970A1 (en) 2013-02-14
WO2010045512A2 (en) 2010-04-22
US20100112042A1 (en) 2010-05-06
US20150209283A1 (en) 2015-07-30
AU2009305639A1 (en) 2010-04-22
CN102231979B (zh) 2014-10-29
CN102231979A (zh) 2011-11-02
IL212329A0 (en) 2011-06-30
KR20110071017A (ko) 2011-06-27
WO2010045512A3 (en) 2011-01-27
SG196818A1 (en) 2014-02-13

Similar Documents

Publication Publication Date Title
AU2009305639B2 (en) Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
AU2008247488B2 (en) Amino acid lipids and uses thereof
US9821067B2 (en) Lipopeptides for delivery of nucleic acids
AU2014202674B2 (en) Amino acid lipids and uses thereof
AU2014262213B2 (en) Amino acid lipids and uses thereof
HK40002101A (en) Amino acid lipids and uses thereof
HK1158059A (en) Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
AU2015202154A1 (en) Lipopeptides for delivery of nucleic acids

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired