HRP20230342T1 - Kationski lipid koji se može ionizirati za isporuku rna - Google Patents

Kationski lipid koji se može ionizirati za isporuku rna Download PDF

Info

Publication number
HRP20230342T1
HRP20230342T1 HRP20230342TT HRP20230342T HRP20230342T1 HR P20230342 T1 HRP20230342 T1 HR P20230342T1 HR P20230342T T HRP20230342T T HR P20230342TT HR P20230342 T HRP20230342 T HR P20230342T HR P20230342 T1 HRP20230342 T1 HR P20230342T1
Authority
HR
Croatia
Prior art keywords
compound according
consist
branched alkyl
cationic lipid
carbon atoms
Prior art date
Application number
HRP20230342TT
Other languages
English (en)
Croatian (hr)
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 HRP20230342T1 publication Critical patent/HRP20230342T1/hr

Links

Classifications

    • 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
    • A61K9/0012Galenical 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
    • 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
    • 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
    • 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
    • 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)
HRP20230342TT 2014-11-18 2015-05-11 Kationski lipid koji se može ionizirati za isporuku rna HRP20230342T1 (hr)

Applications Claiming Priority (5)

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
EP15724466.6A EP3221293B1 (en) 2014-11-18 2015-05-11 Ionizable cationic lipid for rna delivery

Publications (1)

Publication Number Publication Date
HRP20230342T1 true HRP20230342T1 (hr) 2023-06-09

Family

ID=56014367

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20230342TT HRP20230342T1 (hr) 2014-11-18 2015-05-11 Kationski lipid koji se može ionizirati za isporuku rna

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 (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9593077B2 (en) 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US12460231B2 (en) 2014-04-02 2025-11-04 Editas Medicine, Inc. Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
CA3045134A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Compositions and methods for nucleic acid and/or protein payload delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA 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
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
EP3773476A1 (en) * 2018-03-30 2021-02-17 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
EP3784284B1 (en) 2018-04-25 2023-08-02 Ethris GmbH Lipid-based formulations for the delivery of rna
KR20190127277A (ko) * 2018-05-04 2019-11-13 주식회사 삼양바이오팜 mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법
PL3805198T3 (pl) 2018-06-08 2024-02-12 Fujifilm Corporation Związek, jego sól i cząstki lipidowe
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
BR112021018526A2 (pt) * 2019-03-19 2021-11-23 Arcturus Therapeutics Inc Método de fabricação de nanopartículas de rna encapsuladas em lipídio
EP3981435B1 (en) 2019-06-07 2023-08-16 FUJIFILM Corporation Lipid composition
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
EP4328309B1 (en) 2019-11-15 2025-07-02 FUJIFILM Corporation Lipid composition
WO2021178510A1 (en) 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
IL296660A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
CA3172591A1 (en) 2020-03-24 2021-09-30 Douglas Anthony KERR Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
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
EP4189098A1 (en) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions of dna molecules, methods of making therefor, and methods of use thereof
JP7774615B2 (ja) 2020-09-13 2025-11-21 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 大型rnaの脂質ナノ粒子封入
MX2023009345A (es) 2021-02-10 2023-10-24 Oncorus Inc Compuestos, composiciones y metodos de uso de estos.
AU2022260111A1 (en) 2021-04-20 2023-11-30 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
AU2022264509A1 (en) 2021-04-27 2023-12-14 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
EP4395748A1 (en) 2021-09-03 2024-07-10 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
US20250027108A1 (en) 2021-10-29 2025-01-23 CureVac SE Improved circular rna for expressing therapeutic proteins
TW202334080A (zh) 2021-11-08 2023-09-01 美商歐納醫療公司 用於遞送環狀聚核苷酸之脂質奈米粒子組合物
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
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
CN119072330A (zh) 2022-03-14 2024-12-03 世代生物公司 异源初免加强疫苗组合物和使用方法
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
AU2023283348A1 (en) 2022-06-07 2025-01-09 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
IL319704A (en) 2022-09-23 2025-05-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of HBV gene expression
US12186389B2 (en) 2022-10-28 2025-01-07 Glaxosmithkline Biologicals Sa Nucleic acid base vaccine against emerging SARS-CoV-2 variants
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
TW202425959A (zh) 2022-11-08 2024-07-01 美商歐納醫療公司 遞送多核苷酸的脂質及奈米顆粒組合物
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
EP4626444A2 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
IL320873A (en) 2022-12-01 2025-07-01 Generation Bio Co Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions containing them
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
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
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
AU2024269222A1 (en) 2023-05-05 2025-10-09 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
EP4713453A1 (en) 2023-05-15 2026-03-25 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
CN121666232A (zh) 2023-05-31 2026-03-13 开普斯坦治疗公司 脂质纳米颗粒制剂和组合物
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
WO2025166325A1 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
US20250381150A1 (en) 2024-03-08 2025-12-18 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
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof

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
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. 修飾リボザイム
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
US9408914B2 (en) * 2010-04-28 2016-08-09 Kyowa Hakko Kirin Co., Ltd. Cationic lipid
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
CA2876148C (en) * 2012-06-08 2021-01-12 Nitto Denko Corporation Lipids for therapeutic agent delivery formulations
US9593077B2 (en) * 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery

Also Published As

Publication number Publication date
JP2017535610A (ja) 2017-11-30
KR102380363B1 (ko) 2022-03-29
PT3221293T (pt) 2023-03-16
IL252291B (en) 2021-08-31
DK3221293T3 (da) 2023-04-03
EP3221293B1 (en) 2023-03-01
WO2016081029A1 (en) 2016-05-26
HUE062130T2 (hu) 2023-09-28
NZ732777A (en) 2023-06-30
CA2968060C (en) 2023-06-20
IL252291A0 (en) 2017-07-31
CA2968060A1 (en) 2016-05-26
AU2015350561B2 (en) 2020-01-30
SI3221293T1 (sl) 2023-05-31
HK1244479A1 (zh) 2018-08-10
EP3221293A1 (en) 2017-09-27
JP6637988B2 (ja) 2020-01-29
RS64078B1 (sr) 2023-04-28
KR20170095241A (ko) 2017-08-22
AU2015350561A1 (en) 2017-07-06
CN107207428A (zh) 2017-09-26
LT3221293T (lt) 2023-03-27
FI3221293T3 (fi) 2023-04-05
ES2946110T3 (es) 2023-07-12
PL3221293T3 (pl) 2023-07-10

Similar Documents

Publication Publication Date Title
HRP20230342T1 (hr) Kationski lipid koji se može ionizirati za isporuku rna
HRP20221536T1 (hr) Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
HRP20231742T1 (hr) Kationski lipid koji se može ionizirati za isporuku rna
HRP20241239T1 (hr) Novi heterociklički derivati korisni kao shp2 inhibitori
HRP20250181T1 (hr) Spojevi i postupci za ciljanu razgradnju receptora androgena
ME03404B (me) Derivati policikličnih amida kao cdk9 inhibitori
MX377255B (es) Lipidos y composiciones de lipidos para el suministro de agentes activos.
HRP20210529T1 (hr) Inhibitori za mk2 i njihove uporabe
MX2016007522A (es) Estimuladores de guanilato ciclasa soluble (sgc).
MX354175B (es) Derivados de imidazotiadiazol e imidazopirazina como inhibidores del receptor activado de proteasa 4 (par4) para el tratamiento de agregacion plaquetaria.
MX2016005855A (es) Compuestos de conjugados antisentido de apolipoproteina b (apob).
NZ720174A (en) Ionizable cationic lipid for rna delivery
PH12016500806A1 (en) Urea derivative or pharmacologically acceptable salt thereof
ES2626555T3 (es) Pyridone amides como moduladores de canales de sodio
EP4643945A3 (en) Neuroactive steroids and methods of use thereof
MX373716B (es) Compuesto de pirazol-amida y sus usos farmaceuticos.
MX2013010046A (es) Administracion parenteral de tapentadol.
MX380546B (es) Derivados de pirazolopirimidona o pirrolotriazona, metodo y preparacion de los mismos y aplicaciones farmaceuticas de los mismos.
TN2016000061A1 (en) Amide derivatives as lysophosphatidic acid receptor antagonists
FR3010147B1 (fr) Suspension isostatique d'un turboreacteur par double support arriere
PL3509573T3 (pl) Kompozycje farmaceutyczne do dostarczania peptydu
MX373162B (es) Piridopirimidinonas y utilización de las mismas como moduladores de receptores de n-metil-d-aspartato (nmda).
PH12015502632A1 (en) Cxcr7 receptor modulators
EP2994476A4 (en) STRUCTURE-BASED PEPTIDINHIBITORS OF THE P53 AGGREGATION AS A NEW APPROACH TO CANCER THERAPEUTICS
EP3318259A4 (en) STABLE PHARMACEUTICAL COMPOSITION FOR ORAL ADMINISTRATION