PL3221293T3 - Jonizowalny kationowy lipid do dostarczania rna - Google Patents

Jonizowalny kationowy lipid do dostarczania rna

Info

Publication number
PL3221293T3
PL3221293T3 PL15724466.6T PL15724466T PL3221293T3 PL 3221293 T3 PL3221293 T3 PL 3221293T3 PL 15724466 T PL15724466 T PL 15724466T PL 3221293 T3 PL3221293 T3 PL 3221293T3
Authority
PL
Poland
Prior art keywords
cationic lipid
rna delivery
ionizable cationic
ionizable
rna
Prior art date
Application number
PL15724466.6T
Other languages
English (en)
Polish (pl)
Inventor
Joseph E. Payne
Padmanabh Chivukula
Original Assignee
Arcturus 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
Priority claimed from US14/546,105 external-priority patent/US9593077B2/en
Priority claimed from US14/707,876 external-priority patent/US9365610B2/en
Application filed by Arcturus Therapeutics, Inc. filed Critical Arcturus Therapeutics, Inc.
Publication of PL3221293T3 publication Critical patent/PL3221293T3/pl

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02Monothiocarbamic acids; Derivatives thereof
    • C07C333/04Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C323/00Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
    • C07C323/50Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton
    • C07C323/51Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C323/60Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton with the carbon atom of at least one of the carboxyl groups bound to nitrogen atoms
    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0033Medicinal 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
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02Monothiocarbamic acids; Derivatives thereof
    • C07C333/10Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups being part of any of the groups, X being a hetero atom, Y being any atom, e.g., N-acyl-thiocarbamates
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J1/00Normal steroids containing carbon, hydrogen, halogen or oxygen, not substituted in position 17 beta by a carbon atom, e.g. estrane, androstane
    • C07J1/0003Androstane derivatives
    • C07J1/0007Androstane derivatives not substituted in position 17
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J31/00Normal steroids containing one or more sulfur atoms not belonging to a hetero ring
    • C07J31/006Normal steroids containing one or more sulfur atoms not belonging to a hetero ring not covered by C07J31/003
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J41/00Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0055Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0043Nose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/007Pulmonary tract; Aromatherapy
    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • 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
    • A61K9/12Aerosols; Foams
    • 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
    • A61K9/1272Non-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
    • 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
    • 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

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Otolaryngology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL15724466.6T 2014-11-18 2015-05-11 Jonizowalny kationowy lipid do dostarczania rna PL3221293T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US14/546,105 US9593077B2 (en) 2013-11-18 2014-11-18 Ionizable cationic lipid for RNA delivery
US14/707,796 US9567296B2 (en) 2013-11-18 2015-05-08 Ionizable cationic lipid for RNA delivery
US14/707,876 US9365610B2 (en) 2013-11-18 2015-05-08 Asymmetric ionizable cationic lipid for RNA delivery
PCT/US2015/030218 WO2016081029A1 (en) 2014-11-18 2015-05-11 Ionizable cationic lipid for rna delivery

Publications (1)

Publication Number Publication Date
PL3221293T3 true PL3221293T3 (pl) 2023-07-10

Family

ID=56014367

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15724466.6T PL3221293T3 (pl) 2014-11-18 2015-05-11 Jonizowalny kationowy lipid do dostarczania rna

Country Status (20)

Country Link
EP (1) EP3221293B1 (show.php)
JP (1) JP6637988B2 (show.php)
KR (1) KR102380363B1 (show.php)
CN (1) CN107207428A (show.php)
AU (1) AU2015350561B2 (show.php)
CA (1) CA2968060C (show.php)
DK (1) DK3221293T3 (show.php)
ES (1) ES2946110T3 (show.php)
FI (1) FI3221293T3 (show.php)
HK (1) HK1244479A1 (show.php)
HR (1) HRP20230342T1 (show.php)
HU (1) HUE062130T2 (show.php)
IL (1) IL252291B (show.php)
LT (1) LT3221293T (show.php)
NZ (1) NZ732777A (show.php)
PL (1) PL3221293T3 (show.php)
PT (1) PT3221293T (show.php)
RS (1) RS64078B1 (show.php)
SI (1) SI3221293T1 (show.php)
WO (1) WO2016081029A1 (show.php)

Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2015153780A1 (en) 2014-04-02 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2018112278A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Methods and compositions for nucleic acid and protein payload delivery
US10383952B2 (en) * 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119163A1 (en) * 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3600396A4 (en) * 2017-03-30 2021-01-13 The Government of the United States of America as represented by the Secretary of the Army VACCINE COMPOSITION OF NUCLEIC ACID INCLUDING A LIPIDIC FORMULATION, AND PROCESS FOR INCREASING THE EFFECTIVENESS OF NUCLEIC ACID VACCINES
US11015204B2 (en) * 2017-05-31 2021-05-25 Arcturus Therapeutics, Inc. Synthesis and structure of high potency RNA therapeutics
WO2019191780A1 (en) * 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
MX2020011166A (es) 2018-04-25 2022-10-19 Ethris Gmbh Agentes crioprotectores para formulaciones en forma de particulas.
KR20190127277A (ko) * 2018-05-04 2019-11-13 주식회사 삼양바이오팜 mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법
CN112262122B (zh) * 2018-06-08 2023-10-13 富士胶片株式会社 化合物或其盐及脂质颗粒
US11685906B2 (en) 2018-12-06 2023-06-27 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
KR20220018960A (ko) 2019-03-19 2022-02-15 아크투루스 쎄라퓨틱스, 인크. 지질-캡슐화된 rna 나노입자 제조 방법
JP7241869B2 (ja) 2019-06-07 2023-03-17 富士フイルム株式会社 脂質組成物
US20220347298A1 (en) 2019-10-04 2022-11-03 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
PL4328309T3 (pl) 2019-11-15 2025-11-17 Fujifilm Corporation Kompozycja lipidowa
AU2021231795A1 (en) 2020-03-03 2022-10-06 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
CN115667531A (zh) 2020-03-24 2023-01-31 世代生物公司 非病毒DNA载体和其用于表达戈谢(Gaucher)治疗剂的用途
KR20230003477A (ko) 2020-03-24 2023-01-06 제너레이션 바이오 컴퍼니 비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도
JP2023530487A (ja) * 2020-06-18 2023-07-18 アークトゥラス・セラピューティクス・インコーポレイテッド ポリグルタミン病の治療のためのunaオリゴマー
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
AU2021340876A1 (en) 2020-09-13 2023-04-13 Arcturus Therapeutics, Inc. Lipid nanoparticles encapsulation of large RNA
BR112023015937A2 (pt) 2021-02-10 2023-10-24 Oncorus Inc Compostos, composições e métodos de uso dos mesmos
EP4326860A1 (en) 2021-04-20 2024-02-28 Anjarium Biosciences AG Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
WO2022232286A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4329885A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4395748A1 (en) 2021-09-03 2024-07-10 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
CN118541346A (zh) 2021-11-08 2024-08-23 奥纳治疗公司 用于递送环状多核苷酸的脂质纳米颗粒组合物
EP4463475A2 (en) 2022-01-14 2024-11-20 Anjarium Biosciences AG Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
CN114213295B (zh) * 2022-02-22 2022-05-20 中国科学院基础医学与肿瘤研究所(筹) 一种阳离子化合物、制备方法及其复合物和用途
AU2023235112A1 (en) 2022-03-14 2024-10-17 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
JP2025517508A (ja) 2022-05-25 2025-06-05 キュアバック エスイー 核酸ベースのワクチン
CA3258303A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Compositions of lipid nanoparticles and their uses
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
AU2023347320A1 (en) 2022-09-23 2025-04-10 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
EP4615517A1 (en) 2022-11-08 2025-09-17 Orna Therapeutics, Inc. Circular rna compositions
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
WO2024119051A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
AU2023406483A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
KR20250129819A (ko) 2022-12-01 2025-08-29 제너레이션 바이오 컴퍼니 핵산, 이온화 가능한 지질, 스테롤, 지질에 고정된 중합체, 및 헬퍼 지질을 포함하는 지질 나노입자 및 이의 용도
AU2024233180A1 (en) 2023-03-08 2025-09-25 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
CN116270543B (zh) * 2023-05-19 2023-09-26 清华大学 脂质纳米颗粒在制备通过雾吸或鼻滴给药递送核酸的药物中的用途
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025080939A1 (en) 2023-10-13 2025-04-17 Ultragenyx Pharmaceutical, Inc. Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025128853A2 (en) 2023-12-13 2025-06-19 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with ube3a overexpression
WO2025129201A1 (en) 2023-12-15 2025-06-19 Capstan Therapeutics, Inc. Humanized anti-cd8 antibodies and uses thereof
WO2025166323A2 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. Crispr-related methods and compositions targeting lipoprotein (a) expression
WO2025189064A1 (en) 2024-03-08 2025-09-12 Genzyme Corporation Lipid nanoparticles
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS58500845A (ja) 1981-06-04 1983-05-26 デラウェア・ケミカルス・コーポレーション ポンプ
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
WO1992007065A1 (en) 1990-10-12 1992-04-30 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modified ribozymes
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
WO2011136368A1 (ja) * 2010-04-28 2011-11-03 協和発酵キリン株式会社 カチオン性脂質
AU2012267467B8 (en) * 2011-06-08 2017-06-15 Nitto Denko Corporation Compounds for targeting drug delivery and enhancing siRNA activity
US20140308304A1 (en) * 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
SI2858974T1 (sl) * 2012-06-08 2019-01-31 Nitto Denko Corporation Lipidi za formulacije za dostavo terapevtskega sredstva
AU2014348212C1 (en) * 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery

Also Published As

Publication number Publication date
AU2015350561A1 (en) 2017-07-06
EP3221293B1 (en) 2023-03-01
HUE062130T2 (hu) 2023-09-28
WO2016081029A1 (en) 2016-05-26
KR20170095241A (ko) 2017-08-22
EP3221293A1 (en) 2017-09-27
SI3221293T1 (sl) 2023-05-31
IL252291A0 (en) 2017-07-31
AU2015350561B2 (en) 2020-01-30
NZ732777A (en) 2023-06-30
DK3221293T3 (da) 2023-04-03
RS64078B1 (sr) 2023-04-28
CA2968060C (en) 2023-06-20
JP6637988B2 (ja) 2020-01-29
HRP20230342T1 (hr) 2023-06-09
HK1244479A1 (zh) 2018-08-10
FI3221293T3 (fi) 2023-04-05
ES2946110T3 (es) 2023-07-12
IL252291B (en) 2021-08-31
KR102380363B1 (ko) 2022-03-29
PT3221293T (pt) 2023-03-16
CA2968060A1 (en) 2016-05-26
CN107207428A (zh) 2017-09-26
JP2017535610A (ja) 2017-11-30
LT3221293T (lt) 2023-03-27

Similar Documents

Publication Publication Date Title
IL290592A (en) A cationic lipid that can be ionized for the administration of RNA
IL252291B (en) A cationic lipid that undergoes ionization to provide RNA
IL245638A0 (en) A cationic lipid that undergoes ionization for DNA delivery
IL250236B (en) cationic fat
EP3252043A4 (en) Cationic lipid
HUE061809T2 (hu) Készítmények csípõbél-éhbél szakaszba történõ hatóanyag beadáshoz
EP3239132A4 (en) Cationic lipid
EP3093283A4 (en) Cationic lipid
HUE047207T2 (hu) Bortezomib alapú adagolórendszer
IL262310A (en) Advanced methods of gene insertion
IL247106A0 (en) Material delivery system
EP3184506A4 (en) Cationic lipid for nucleic acid delivery
EP3162794A4 (en) Cationic lipid
ZA201605787B (en) Formulations for small intestinal delivery
GB201514902D0 (en) Formulations for transmucosal delivery
GB201514906D0 (en) Formulations for transmucosal delivery
GB201507125D0 (en) Gene delivery
GB201522636D0 (en) Delivery system