CA2739046A1 - Process and compositions for liposomal and effective delivery of nucleic acids - Google Patents

Process and compositions for liposomal and effective delivery of nucleic acids Download PDF

Info

Publication number
CA2739046A1
CA2739046A1 CA2739046A CA2739046A CA2739046A1 CA 2739046 A1 CA2739046 A1 CA 2739046A1 CA 2739046 A CA2739046 A CA 2739046A CA 2739046 A CA2739046 A CA 2739046A CA 2739046 A1 CA2739046 A1 CA 2739046A1
Authority
CA
Canada
Prior art keywords
composition
agent
nucleic acid
peptide
disease
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.)
Abandoned
Application number
CA2739046A
Other languages
English (en)
French (fr)
Inventor
Barry A. Polisky
Roger C. Adami
Michael V. Templin
Pierrot Harvie
Rachel E. Johns
Jaya S. Giyanani
Michael E. Houston
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 CA2739046A1 publication Critical patent/CA2739046A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1277Preparation processes; Proliposomes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/10Drugs for disorders of the urinary system of the bladder
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Dispersion Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Communicable Diseases (AREA)
  • Virology (AREA)
CA2739046A 2008-10-16 2009-10-16 Process and compositions for liposomal and effective delivery of nucleic acids Abandoned CA2739046A1 (en)

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

Publications (1)

Publication Number Publication Date
CA2739046A1 true CA2739046A1 (en) 2010-04-22

Family

ID=41560381

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2739046A Abandoned CA2739046A1 (en) 2008-10-16 2009-10-16 Process and compositions for liposomal and effective delivery of nucleic acids

Country Status (12)

Country Link
US (3) US20100112042A1 (cg-RX-API-DMAC7.html)
EP (2) EP2902013A1 (cg-RX-API-DMAC7.html)
JP (2) JP2012505913A (cg-RX-API-DMAC7.html)
KR (1) KR20110071017A (cg-RX-API-DMAC7.html)
CN (2) CN102231979B (cg-RX-API-DMAC7.html)
AU (3) AU2009305639B2 (cg-RX-API-DMAC7.html)
BR (1) BRPI0920171A2 (cg-RX-API-DMAC7.html)
CA (1) CA2739046A1 (cg-RX-API-DMAC7.html)
IL (1) IL212329A0 (cg-RX-API-DMAC7.html)
SG (1) SG196818A1 (cg-RX-API-DMAC7.html)
WO (1) WO2010045512A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201102319B (cg-RX-API-DMAC7.html)

Families Citing this family (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2006259415B2 (en) 2005-06-15 2012-08-30 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
SI2494993T1 (sl) 2007-05-04 2019-01-31 Marina Biotech, Inc. Aminokislinski lipidi in njihove uporabe
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
WO2010059829A2 (en) * 2008-11-19 2010-05-27 Mdrna, Inc. Compositions and methods for triggered release rna therapeutics
CN102665761A (zh) 2009-11-04 2012-09-12 玛瑞纳生物技术有限公司 活性产生递送分子
US20110244026A1 (en) 2009-12-01 2011-10-06 Braydon Charles Guild Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
EP2609135A4 (en) 2010-08-26 2015-05-20 Massachusetts Inst Technology POLY (BETA AMINO ALCOHOLS), THEIR PREPARATION AND USES THEREOF
JP6054308B2 (ja) * 2011-02-15 2016-12-27 メリマック ファーマシューティカルズ, インコーポレイテッドMerrimack Pharmaceuticals, Inc. 核酸を細胞へ送達する組成物及び方法
PL2691443T3 (pl) 2011-03-28 2021-08-30 Massachusetts Institute Of Technology Sprzężone lipomery i ich zastosowania
LT2717893T (lt) 2011-06-08 2019-08-12 Translate Bio, Inc. Lipidų nanodalelių kompozicijos ir mrnr pristatymo būdai
AU2012308320C1 (en) * 2011-09-14 2018-08-23 Translate Bio Ma, Inc. Multimeric oligonucleotide compounds
EP4074694A1 (en) 2011-10-27 2022-10-19 Massachusetts Institute Of Technology Amino acid-, peptide- an polypeptide-lipids, isomers, compositions, an uses thereof
SI2773326T1 (sl) * 2011-11-04 2019-04-30 Nitto Denko Corporation Metoda za sterilno proizvodnjo delcev lipidno-nukleinske kisline
AU2013262700A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating hemoglobin gene family expression
CA2873794A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating smn gene family expression
WO2013173598A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating atp2a2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EP2850185A4 (en) 2012-05-16 2015-12-30 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING UTRN GENE EXPRESSION
US20150152410A1 (en) 2012-05-16 2015-06-04 Rana Therapeutics, Inc. Compositions and methods for modulating mecp2 expression
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
AU2014236305B2 (en) 2013-03-14 2019-01-17 Ethris Gmbh CFTR mRNA compositions and related methods and uses
MX2015012865A (es) 2013-03-14 2016-07-21 Shire Human Genetic Therapies Metodos para purificacion de arn mensajero.
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
US9745583B2 (en) 2013-04-30 2017-08-29 Delta-Fly Pharma, Inc. Liposome for topical administration and application thereof
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
CA2921161A1 (en) * 2013-09-24 2015-04-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for the manufacture of lipid nanoparticles
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
EP3871696B1 (en) 2013-10-22 2025-03-19 Translate Bio, Inc. Lipid formulations for delivery of messenger rna
ES2954366T3 (es) 2013-10-22 2023-11-21 Translate Bio Inc Terapia de ácido ribonucleico mensajero para la deficiencia de argininosuccinato sintetasa
RS66380B1 (sr) 2014-04-23 2025-02-28 Modernatx Inc Vakcine nukleinske kiseline
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
JP6599373B2 (ja) 2014-06-24 2019-10-30 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための立体化学的に濃縮した組成物
EP3164379A1 (en) 2014-07-02 2017-05-10 Massachusetts Institute of Technology Polyamine-fatty acid derived lipidoids and uses thereof
JP5934848B1 (ja) * 2014-10-30 2016-06-15 Delta−Fly Pharma株式会社 局所投与用リポプレックスの新規製造方法及び該リポプレックスを使用する抗腫瘍剤
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
SI3310764T1 (sl) 2015-06-19 2023-06-30 Massachusetts Institute Of Technology Z alkenilom substituirani 2,5-piperazindioni in njihova uporaba v sestavkih za dostavo sredstva subjektu ali celici
WO2017031232A1 (en) * 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス, インコーポレイテッド ヒトサイトメガロウイルスワクチン
US9901649B2 (en) 2016-05-03 2018-02-27 International Business Machines Corporation Block copolymers for therapeutic drug delivery
EP3528821A4 (en) 2016-10-21 2020-07-01 ModernaTX, Inc. HUMAN CYTOMEGALOVIRUS VACCINE
KR101797168B1 (ko) * 2016-11-10 2017-11-13 주식회사 바이오셀트란 신규의 프로테인 트랜스덕션 도메인을 함유하는 구형 구조체
US11253605B2 (en) 2017-02-27 2022-02-22 Translate Bio, Inc. Codon-optimized CFTR MRNA
AU2018259859B2 (en) * 2017-04-28 2024-02-15 Kyowa Kirin Co., Ltd. Oligonucleotide derivative or salt thereof
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
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
CA3073211A1 (en) * 2017-08-31 2019-03-07 Modernatx, Inc. Methods of making lipid nanoparticles
JP7256824B2 (ja) * 2018-04-25 2023-04-12 エスリス ゲーエムベーハー 粒子状製剤用の凍結保護剤
CN112930396B (zh) 2018-08-24 2024-05-24 川斯勒佰尔公司 用于纯化信使rna的方法
WO2020061457A1 (en) 2018-09-20 2020-03-26 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
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
JP7752615B2 (ja) * 2019-12-20 2025-10-10 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの直腸送達
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
US20240408034A1 (en) * 2021-10-06 2024-12-12 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

Family Cites Families (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR8207733A (pt) 1981-06-04 1983-05-10 Pharmasol Corp Recipiente pressurizado com bomba de suprimento
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
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
CH671514A5 (cg-RX-API-DMAC7.html) * 1986-12-03 1989-09-15 Induchem Ag
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
CA1339303C (en) 1987-09-21 1997-08-19 Lyle John Arnold Jr. 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
CA2039718C (en) 1989-08-31 2003-02-25 John J. Rossi 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
DE552178T1 (de) 1990-10-12 1994-02-03 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
AU4769893A (en) 1992-07-17 1994-02-14 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
PT748382E (pt) 1993-09-02 2003-03-31 Ribozyme Pharm Inc Acidos nucleicos enzimaticos contendo nao-nucleotidos
AU8088694A (en) 1993-10-27 1995-05-22 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
EP1179340A3 (en) 1994-09-30 2003-05-07 INEX Pharmaceutical Corp. Compositions for the introduction of polyanionic materials into cells
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
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)
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
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
JP4338106B2 (ja) 1995-06-07 2009-10-07 ライフ テクノロジーズ コーポレーション ペプチド増強カチオン脂質トランスフェクション
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
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
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
EP0886641A2 (en) 1996-01-16 1998-12-30 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
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
AU1922999A (en) 1997-12-16 1999-07-05 Baylor College Of Medicine 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
DE69941447D1 (de) 1998-01-05 2009-11-05 Univ Washington Erhöhter transport unter benutzung membranzerstörender stoffe
US6426086B1 (en) 1998-02-03 2002-07-30 The Regents Of The University Of California pH-sensitive, serum-stable liposomes
CA2326823A1 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
HRP20000751A2 (en) 1998-05-26 2001-12-31 Icn Pharmaceuticals Novel nucleosides having bicyclic sugar moiety
CA2365625A1 (en) 1999-03-10 2000-09-14 Phogen Limited Delivery of substances 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
DK1102785T3 (da) 1999-06-07 2013-05-13 Arrowhead Res Corp Sammensætninger til lægemiddeltilførsel ved anvendelse af pH-følsomme molekyler
AU769357B2 (en) 1999-07-15 2004-01-22 University Of British Columbia, The Methods for preparation of lipid-encapsulated therapeutic agents
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
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
NZ528523A (en) 2001-03-26 2006-03-31 Alza Corp Liposome composition for improved intracellular delivery of a therapeutic agent
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
US20030077829A1 (en) * 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
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
AU2003217550A1 (en) 2002-02-20 2003-09-09 Ribozyme Pharmaceuticals, Incorporated RNA INTERFERENCE MEDIATED INHIBITION OF TNF AND TNF RECEPTOR SUPERFAMILY GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US7591994B2 (en) 2002-12-13 2009-09-22 Immunomedics, Inc. Camptothecin-binding moiety conjugates
EP1509203B1 (en) * 2002-05-15 2016-04-13 California Pacific Medical Center Delivery of nucleic acid-like compounds
ES2354607T3 (es) * 2002-06-28 2011-03-16 Protiva Biotherapeutics Inc. Procedimiento y aparato para producir liposomas.
JP4951338B2 (ja) 2003-07-16 2012-06-13 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 脂質に封入された干渉rna
EP1512393A1 (de) * 2003-09-08 2005-03-09 BOEHRINGER INGELHEIM PHARMA GMBH & CO. KG Verfahren zur Herstellung von homogenen Liposomen und Lipoplexen
ES2707393T3 (es) * 2004-03-26 2019-04-03 Curis Inc Moduladores de interferencia de ARN de señalización de hedgehog y usos de los mismos
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
AU2005247091B2 (en) * 2004-05-24 2010-08-12 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
EP2004141A2 (en) * 2006-03-17 2008-12-24 Novosom AG An efficient method for loading amphoteric liposomes with nucleic acid active substances
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
SI2494993T1 (sl) * 2007-05-04 2019-01-31 Marina Biotech, Inc. Aminokislinski lipidi in njihove uporabe
NZ580712A (en) 2007-05-22 2011-12-22 Marina Biotech Inc Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers
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

Also Published As

Publication number Publication date
JP2012505913A (ja) 2012-03-08
BRPI0920171A2 (pt) 2016-07-12
SG196818A1 (en) 2014-02-13
CN102231979B (zh) 2014-10-29
CN102231979A (zh) 2011-11-02
AU2009305639B2 (en) 2016-06-23
EP2349210A2 (en) 2011-08-03
AU2009305639A1 (en) 2010-04-22
ZA201102319B (en) 2012-09-26
JP2015096529A (ja) 2015-05-21
US20130039970A1 (en) 2013-02-14
AU2018204605A1 (en) 2018-07-12
EP2349210B1 (en) 2015-03-18
US20100112042A1 (en) 2010-05-06
WO2010045512A3 (en) 2011-01-27
WO2010045512A2 (en) 2010-04-22
CN104382853A (zh) 2015-03-04
AU2016222371A1 (en) 2016-09-15
KR20110071017A (ko) 2011-06-27
JP6126072B2 (ja) 2017-05-10
US20150209283A1 (en) 2015-07-30
EP2902013A1 (en) 2015-08-05
IL212329A0 (en) 2011-06-30

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
EEER Examination request

Effective date: 20141003

FZDE Discontinued

Effective date: 20210226