ES2946110T3 - Lípido catiónico ionizable para la administración de ARN - Google Patents

Lípido catiónico ionizable para la administración de ARN Download PDF

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Publication number
ES2946110T3
ES2946110T3 ES15724466T ES15724466T ES2946110T3 ES 2946110 T3 ES2946110 T3 ES 2946110T3 ES 15724466 T ES15724466 T ES 15724466T ES 15724466 T ES15724466 T ES 15724466T ES 2946110 T3 ES2946110 T3 ES 2946110T3
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Prior art keywords
compound
carbons
lipid
mol
consist
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ES15724466T
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English (en)
Spanish (es)
Inventor
Joseph Payne
Padmanabh Chivukula
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Arcturus Therapeutics Inc
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Arcturus Therapeutics Inc
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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
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Publication of ES2946110T3 publication Critical patent/ES2946110T3/es
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02—Monothiocarbamic acids; Derivatives thereof
    • C07C333/04—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C323/00—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
    • C07C323/50—Thiols, 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
    • C07C323/51—Thiols, 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
    • C07C323/60—Thiols, 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02—Monothiocarbamic acids; Derivatives thereof
    • C07C333/10—Monothiocarbamic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J1/00—Normal steroids containing carbon, hydrogen, halogen or oxygen, not substituted in position 17 beta by a carbon atom, e.g. estrane, androstane
    • C07J1/0003—Androstane derivatives
    • C07J1/0007—Androstane derivatives not substituted in position 17
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J31/00—Normal steroids containing one or more sulfur atoms not belonging to a hetero ring
    • C07J31/006—Normal steroids containing one or more sulfur atoms not belonging to a hetero ring not covered by C07J31/003
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0055—Normal 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0043—Nose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • 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
    • A61K9/12—Aerosols; Foams
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • 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

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  • 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)
ES15724466T 2014-11-18 2015-05-11 Lípido catiónico ionizable para la administración de ARN Active ES2946110T3 (es)

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
ES2946110T3 true ES2946110T3 (es) 2023-07-12

Family

ID=56014367

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15724466T Active ES2946110T3 (es) 2014-11-18 2015-05-11 Lípido catiónico ionizable para la administración de ARN

Country Status (20)

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

Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3071547B1 (en) 2013-11-18 2024-07-10 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
CN110582301A (zh) 2016-12-14 2019-12-17 利甘达尔股份有限公司 用于核酸和蛋白质有效负载递送的方法和组合物
US10383952B2 (en) * 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10526284B2 (en) 2016-12-21 2020-01-07 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
EP3600396A4 (en) * 2017-03-30 2021-01-13 The Government of the United States of America as represented by the Secretary of the Army NUCLEIC ACID VACCINE COMPOSITION WITH A LIPID FORMULATION AND METHOD FOR INCREASING THE POTENTIAL OF NUCLEIC ACID VACCINE
WO2018222890A1 (en) * 2017-05-31 2018-12-06 Arcturus Therapeutics, Inc. Synthesis and structure of high potency rna therapeutics
US12083224B2 (en) 2018-03-30 2024-09-10 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
PL3784285T3 (pl) 2018-04-25 2023-12-18 Ethris Gmbh Środki krioprotekcyjne do formulacji cząstek stałych
KR20190127277A (ko) * 2018-05-04 2019-11-13 주식회사 삼양바이오팜 mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법
KR102591743B1 (ko) 2018-06-08 2023-10-19 후지필름 가부시키가이샤 화합물 또는 그 염 및 지질 입자
EP4299750A3 (en) 2018-12-06 2024-07-10 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
SG11202110036RA (en) 2019-03-19 2021-10-28 Arcturus Therapeutics Inc Method of making lipid-encapsulated rna nanoparticles
KR102660546B1 (ko) 2019-06-07 2024-04-24 후지필름 가부시키가이샤 지질 조성물
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
EP4328309B1 (en) 2019-11-15 2025-07-02 FUJIFILM Corporation Lipid composition
JP2023516676A (ja) 2020-03-03 2023-04-20 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 オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法
AU2021244555A1 (en) 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2021195218A1 (en) 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
US20230227826A1 (en) * 2020-06-18 2023-07-20 Arcturus Therapeutics, Inc. Una oligomers for the treatment of polyglutamine diseases
AU2021314809A1 (en) 2020-07-27 2023-02-23 Anjarium Biosciences Ag Compositions of DNA molecules, methods of making therefor, and methods of use thereof
WO2022056413A1 (en) 2020-09-13 2022-03-17 Arcturus Therapeutics, Inc. Lipid nanoparticles encapsulation of large rna
KR20230155448A (ko) * 2021-02-10 2023-11-10 온코루스, 인크. 화합물, 조성물 및 이의 사용 방법
CA3214538A1 (en) 2021-04-20 2022-10-27 Joel DE BEER Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
US20240226324A1 (en) 2021-04-27 2024-07-11 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
JP2024538489A (ja) 2021-09-03 2024-10-23 キュアバック エスイー 核酸を送達するための新規な脂質ナノ粒子
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
CA3236235A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法
EP4469091A1 (en) 2022-01-28 2024-12-04 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
CA3245737A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. PRIME-BOOST HETEROLOGICAL VACCINE COMPOSITIONS AND METHODS OF USE
KR20250028547A (ko) 2022-05-25 2025-02-28 큐어백 에스이 대장균 FimH 항원성 폴리펩타이드를 인코딩하는 핵산 기반 백신
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
JP2025532177A (ja) 2022-09-23 2025-09-29 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
JP2025537178A (ja) 2022-11-08 2025-11-14 オーナ セラピューティクス， インコーポレイテッド 環状ｒｎａ組成物
CN120615013A (zh) 2022-12-01 2025-09-09 世代生物公司 用于细胞靶向的隐形脂质纳米颗粒组合物
AU2023406303A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
AU2023406321A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
DE112024001143T5 (de) 2023-03-08 2025-12-18 CureVac SE Neue lipid-nanopartikel-formeln für die abgabe von nukleinsäuren
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
US20250332281A2 (en) 2023-12-15 2025-10-30 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
ATE21633T1 (de) 1981-06-04 1986-09-15 Pharmasol Corp Druckbehaelter mit abgabepumpe.
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
EP0552178B1 (en) 1990-10-12 1997-01-02 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
CA2800818C (en) * 2010-04-28 2017-10-31 Kyowa Hakko Kirin Co., Ltd. Cationic lipid
KR102029654B1 (ko) * 2011-06-08 2019-10-08 닛토덴코 가부시키가이샤 표적 약물 전달체 및 ｓｉＲＮＡ 활성을 증가시키는 화합물
WO2013086373A1 (en) * 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
WO2013185116A1 (en) * 2012-06-08 2013-12-12 Payne Joseph E Lipids for therapeutic agent delivery formulations
EP3071547B1 (en) * 2013-11-18 2024-07-10 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery

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