HRP20240586T1 - Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima - Google Patents

Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima Download PDF

Info

Publication number
HRP20240586T1
HRP20240586T1 HRP20240586TT HRP20240586T HRP20240586T1 HR P20240586 T1 HRP20240586 T1 HR P20240586T1 HR P20240586T T HRP20240586T T HR P20240586TT HR P20240586 T HRP20240586 T HR P20240586T HR P20240586 T1 HRP20240586 T1 HR P20240586T1
Authority
HR
Croatia
Prior art keywords
lipid
tcv
competent
vesicle
optionally
Prior art date
Application number
HRP20240586TT
Other languages
English (en)
Inventor
Jayesh Kulkarni
Austin Hill
Pieter Cullis
Blair Leavitt
Terri PETKAU
Pamela WAGNER
Original Assignee
The University Of British Columbia
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 The University Of British Columbia filed Critical The University Of British Columbia
Publication of HRP20240586T1 publication Critical patent/HRP20240586T1/hr

Links

Classifications

    • 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
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12—Carboxylic acids; Salts or anhydrides thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/186—Quaternary ammonium compounds, e.g. benzalkonium chloride or cetrimide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • 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—Liposomes
    • A61K9/1277—Processes for preparing; Proliposomes
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005—Wall or coating material
    • A61K9/5015—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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 N.A.
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dispersion Chemistry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Manufacturing & Machinery (AREA)
  • Dermatology (AREA)

Claims (25)

1. Postupak kapsuliranja odabranog tereta u vezikulu sposobnu za transfekciju na bazi lipida (TCV) koja se sastoji od: (a) osiguravanje otopine na bazi vode koji sadrži TCV pri čemu je pri čemu je otopina na bazi vode slobodna bez organskih otapala i deterdženata; i, (b) miješanje odabranog tereta u otopina pod prikladnim uvjetima i tijekom vremena dovoljnog da se odabrani teret inkapsulira unutar kationske na lipidu osnovane transfekcijski kompetentne vezikule (TCV) da bi se dobio inkapsuliran odabrani teret u na lipidu osnovanoj transfekcijski kompetentnoj vezikuli (TCV), pri čemu se miješanje vrši bez prisustva organskog otapala i bez prisustva deterdženta, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži ionizirajući kationski lipid u oko 20 mol% ili sa oko 30 mol% i pri čemu taj odabrani teret sadrži bjelančevine.
2. Postupak iz patentnog zahtjeva 1, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži ionizirajući kationski lipid s oko 20 mol%.
3. Postupak iz patentnog zahtjeva 1 ili 2, pri čemu ionizirajući kationski lipid sadrži ili je 1,2-dioleiloksi-3-dimetilaminopropan (DODMA).
4. Postupak iz bilo kojeg od patentnih zahtjeva 1-3, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži DODMA u oko 20 mol%.
5. Postupak iz patentnog zahtjeva 1, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži DODMA u oko 30 mol%.
6. Postupak iz bilo kojeg od patentnih zahtjeva 1-5, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) još sadrži najmanje jedan polietilen glikol (PEG) ili kolesterol.
7. Postupak iz bilo kojeg od patentnih zahtjeva 1-6, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži mješavinu od: (i) Ionizirajućeg kationskog lipida, izborno DODMA; (ii) jednog ili više fosfolipida, izborno 1,2-dioleoil-sn-glicero-3-fosfoetanolamin (DOPE) i 1,2-distearoil-sn-glicero-3-fosfoholina (DSPC); (iii) kolesterola; i (iv) PEG-lipida.
8. Postupak iz bilo kojeg od patentnih zahtjeva 1-3, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži: (i) mješavinu DODMA/DOPE/DSPC/ChoI/PEG lipida u oko 20/30/10/39/1 mol%; ili (ii) mješavinu DODMA/DOPE/DSPC/Chol u oko 20/30/10/40 mol%.
9. Postupak iz bilo kojeg od patentnih zahtjeva 1-8, pri čemu odabrani teret nadalje sadrži nukleinsku kiselinu, izborno, pri čemu je nukleinska kiselina: (i) ribonukleinska kiselina (RNK), izborno sadrži informacijsku RNK (iRNK), malu interferirajuću RNK (siRNK), kratku ukosničku RNK, i/ili mikroRNK (miRNK); i/ili (ii) dezoksiribonukleinska kiselina (DNK), izborno sadrži dvolančanu DNK, jednolančanu DNK, plazmidnu DNK, i/ili komplementarnu DNK (kDNK).
10. Postupak iz bilo kojeg od patentnih zahtjeva 1-9, pri čemu bjelančevina sadrži dio ribonukleobjelančevinua (RNP), izborno pri čemu ribonukleobjelančevinu (RNP) sadrži Cas9 bjelančevinu i vodeću RNK, i još izborno pri čemu odabrani teret nadalje sadrži jednolančanu DNK (jlDNK, ssDNA).
11. Postupak iz bilo kojeg od patentnih zahtjeva 1-10, pri čemu taj na lipidu osnovana otopina sadrži 25 mM do 100 mM acetatnog puferskog sredstva.
12. Supstanca koja sadrži na lipidu osnovanu transfekcijski kompetentnu vezikulu (TCV) u otopini na bazi vode pri čemu je supstanca bez organskih otapala i deterdženata pri čemu osim toga na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži ionizirajući kationski lipid sa oko 20 mol% ili sa oko 30%, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži odabrani teret koji sadrži bjelančevinu.
13. Supstanca iz patentnog zahtjeva 12, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži ionizirajući kationski lipid u oko 20 mol%.
14. Supstanca iz patentnog zahtjeva 12 ili 13, pri čemu ionizirajući kationski lipid sadrži ili je DODMA.
15. Supstanca iz bilo kojeg od patentnih zahtjeva 12-14, pri čemu ta na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži DODMA u oko 20 mol%.
16. Supstanca iz patentnog zahtjeva 12, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži DODMA u oko 30 mol%.
17. Supstanca iz bilo kojeg od patentnih zahtjeva 12-16, pri čemu ta na lipidu osnovana transfekcijski kompetentna vezikula (TCV) još sadrži najmanje jedan polietilen glikol (PEG) ili kolesterol.
18. Supstanca iz bilo kojeg od patentnih zahtjeva 12-17, pri čemu ta na lipidu osnovana tansfekcijski kompetentna vezikula (TCV) sadrži mješavinu: ionizirajućeg kationskog lipida, izborno DODMA; jedan ili više fosfolipida, izborno DOPE i DSPC; kolesterol; i PEG lipid.
19. Supstanca iz bilo kojeg od patentnih zahtjeva 12-14, pri čemu na lipidu osnovana transfekcijski kompetentna vezikula (TCV) sadrži: (i) mješavinu DODMA/DOPE/DSPC/ChoI/PEG lipida u oko 20/30/10/39/1 mol%; ili (ii) mješavinu DODMA/DOPE/DSPC/Cho l u oko 20/30/10/40 mol%.
20. Supstanca iz bilo kojeg od patentnih zahtjeva 12-19, pri čemu taj na lipidu osnovana otopina sadrži 25 mM do 100 mM acetatnog puferskog sredstva.
21. Supstanca bilo kojeg od patentnih zahtjeva 12-20, pri čemu odabrani teret nadalje sadrži nukleinsku kiselinu, izborno pri čemu je nukleinska kiselina: (i) ribonukleinska kiselina (RNK), izborno sadrži informacijsku RNK (iRNK), malu interferirajuću RNK (siRNK), kratku ukosničku RNK, i/ili mikroRNK (miRNK); i/ili (ii) dezoksiribonukleinska kiselina (DNK), izborno sadrži dvolančanu DNK, jednolančanu DNK, plazmidnu DNK, i/ili komplementarnu DNK (kDNK).
22. Supstanca iz bilo kojeg od patentnih zahtjeva 12-21, pri čemu ta bjelančevina sadrži dio (RNP), izborno pri čemu ribonukleobjelančevina (RNP) sadrži Cas9 bjelančevinu i vodeću RNK, i još izbornije, pri čemu odabrani teret nadalje sadrži jednolančanu DNK (jlDNK, ssDNA).
23. Supstanca koja sadrži na lipidu osnovan teret odabran inkapsulacijom tranfekcijski kompetentne vezikule (TCV) u na vodi osnovanim organskim otapalima i deterdžentima bez otopine, koji je proizveden postupkom sukladno bilo kojem od patentnih zahtjeva 1-11.
24. Postupak transfekcije, pri čemu postupak sadrži transfektiranje ciljne stanice na lipidu osnovanom teretu odabranim inkapsulacijom transfekcijski kompetentne vezikule (TCV) na odabranom teretu na lipidu osnovane inkapsulirane transfekcijski kompetentne vezikule (TCV) sukladno bilo kojem od patentnih zahtjeva 12-23, pri čemu izborno ciljna stanica je stanica sisavca, osim toga izborno primarna stanica sisavca, i osim toga izborno primarna neuronska stanica sisavca ili kultivirana stanica sisavca.
25. Supstanca koja sadrži na lipidu osnovanu transfekcijsku kompetentnu vezikulu (TCV) koja sadrži odabrani teret sukladno bilo kojem od patentnih zahtjeva 12-23, za liječenje subjekta kojem je potrebno, izborno pri čemu je to liječenje putem terapije genom a osim toga izborno i liječenje neurološkog poremećaja, i još izbornije Huntingtonove bolesti, Alzheimerove bolesti, Parkinsonove bolesti, demencije prednjeg režnja, amiotrofne lateralne skleroze, ili atrofije mišića kralježnice.
HRP20240586TT 2018-10-09 2019-10-09 Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima HRP20240586T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862743116P 2018-10-09 2018-10-09
EP19871608.6A EP3864163B1 (en) 2018-10-09 2019-10-09 Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto
PCT/US2019/055472 WO2020077007A1 (en) 2018-10-09 2019-10-09 Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto

Publications (1)

Publication Number Publication Date
HRP20240586T1 true HRP20240586T1 (hr) 2024-07-19

Family

ID=70164385

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20240586TT HRP20240586T1 (hr) 2018-10-09 2019-10-09 Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima

Country Status (19)

Country Link
US (2) US11980673B2 (hr)
EP (2) EP3864163B1 (hr)
JP (1) JP2024028838A (hr)
KR (1) KR20210093232A (hr)
CN (1) CN113166783A (hr)
AU (2) AU2019359299B2 (hr)
BR (1) BR112021006539A2 (hr)
CA (1) CA3114699A1 (hr)
DK (1) DK3864163T3 (hr)
EA (1) EA202190688A1 (hr)
FI (1) FI3864163T3 (hr)
HR (1) HRP20240586T1 (hr)
HU (1) HUE066181T2 (hr)
IL (1) IL282063A (hr)
PH (1) PH12021550626A1 (hr)
PL (1) PL3864163T3 (hr)
PT (1) PT3864163T (hr)
RS (1) RS65449B1 (hr)
WO (1) WO2020077007A1 (hr)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
PT3864163T (pt) 2018-10-09 2024-04-30 Univ British Columbia Composições e sistemas que compreendem vesículas competentes para transfeção isentas de solventes orgânicos e detergentes e métodos relacionados com as mesmas
WO2021231901A1 (en) * 2020-05-15 2021-11-18 Translate Bio, Inc. Lipid nanoparticle formulations for mrna delivery
EP4251170A1 (en) 2020-11-25 2023-10-04 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
WO2023161873A1 (en) * 2022-02-25 2023-08-31 Incisive Genetics, Inc. Gene editing reporter system and guide rna and composition related thereto; composition and method for knocking out dna with more than two grnas; gene editing in the eye; and gene editing using base editors
US12064479B2 (en) 2022-05-25 2024-08-20 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof

Family Cites Families (244)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5552155A (en) * 1992-12-04 1996-09-03 The Liposome Company, Inc. Fusogenic lipsomes and methods for making and using same
US5776486A (en) 1993-05-28 1998-07-07 Aphios Corporation Methods and apparatus for making liposomes containing hydrophobic drugs
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
HUP9802445A3 (en) 1995-08-01 1999-03-29 Novartis Ag Liposomal oligonucleotide compositions and process for inhibiting the human raf
US6093816A (en) 1996-06-27 2000-07-25 Isis Pharmaceuticals, Inc. Cationic lipids
US7262173B2 (en) 1997-03-21 2007-08-28 Georgetown University Chemosensitizing with liposomes containing oligonucleotides
WO1998051278A2 (en) 1997-05-14 1998-11-19 Inex Pharmaceuticals Corporation High efficiency encapsulation of charged therapeutic agents in lipid vesicles
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
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
US6693086B1 (en) 1998-06-25 2004-02-17 National Jewish Medical And Research Center Systemic immune activation method using nucleic acid-lipid complexes
US20030022854A1 (en) 1998-06-25 2003-01-30 Dow Steven W. Vaccines using nucleic acid-lipid complexes
WO2000030444A1 (en) 1998-11-25 2000-06-02 Vanderbilt University Cationic liposomes for gene transfer
AU5905400A (en) 1999-07-14 2001-02-05 Board Of Regents, The University Of Texas System Methods and compositions for delivery and retention of active agents to lymph nodes
US7094423B1 (en) 1999-07-15 2006-08-22 Inex Pharmaceuticals Corp. Methods for preparation of lipid-encapsulated therapeutic agents
EP1323415B1 (en) 2000-10-04 2010-01-13 Kyowa Hakko Kirin Co., Ltd. Method of coating fine particle with lipid film
US7713942B2 (en) 2001-04-04 2010-05-11 Nordic Vaccine Technology A/S Cage-like microparticle complexes comprising sterols and saponins for delivery of polynucleotides
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
US7364750B2 (en) 2001-04-30 2008-04-29 The University Of British Columbia Autogene nucleic acids encoding a secretable RNA polymerase
EP1441763A2 (en) 2001-11-07 2004-08-04 Inex Pharmaceuticals Corp. Mucosal adjuvants comprising an oligonucleotide and a cationic lipid
CA2491164C (en) 2002-06-28 2012-05-08 Cory Giesbrecht Method and apparatus for producing liposomes
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
WO2005026372A1 (en) 2003-09-15 2005-03-24 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
CN101031287A (zh) 2004-03-02 2007-09-05 麻省理工学院 纳米细胞药物递送系统
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
JP4764426B2 (ja) 2004-06-07 2011-09-07 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 カチオン性脂質および使用方法
CA2572439A1 (en) 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
EP2199298A1 (en) 2004-11-17 2010-06-23 Protiva Biotherapeutics Inc. Sirna silencing of Apolipoprotein B
JP2006248978A (ja) 2005-03-10 2006-09-21 Mebiopharm Co Ltd 新規なリポソーム製剤
EP1874793A4 (en) 2005-04-15 2008-12-24 Univ Texas DISTRIBUTION OF SIRNA BY NEUTRAL LIPID COMPOSITIONS
US20080088046A1 (en) 2006-10-13 2008-04-17 Steffen Panzner Amphoteric liposomes, a method of formulating an amphoteric liposome and a method of loading an amphoteric liposome
US20070087045A1 (en) 2005-10-14 2007-04-19 Industrial Technology Research Institute Lipid carrier and method of preparing the same
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
US20070218122A1 (en) 2005-11-18 2007-09-20 Protiva Biotherapeutics, Inc. siRNA silencing of influenza virus gene expression
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
AU2007284036A1 (en) 2006-08-16 2008-02-21 Protiva Biotherapeutics, Inc. Nucleic acid modulation of Toll-like receptor-mediated immune stimulation
EP2086507B1 (en) * 2006-10-06 2018-11-07 BioNet Pharma GmbH A spinal nucleus pulposus implant
US20100297242A1 (en) 2007-10-17 2010-11-25 Tae-Gwan Park Ldl-like cationic nanoparticles for deliverying nucleic acid gene, method for preparing thereof and method for deliverying nucleic acid gene using the same
CA2710713C (en) 2007-12-27 2017-09-19 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US8268796B2 (en) 2008-06-27 2012-09-18 Children's Hospital & Research Center At Oakland Lipophilic nucleic acid delivery vehicle and methods of use thereof
TW201004658A (en) 2008-07-31 2010-02-01 Enzon Pharmaceuticals Inc Nanoparticle compositions for nucleic acids delivery system
PL2350043T3 (pl) 2008-10-09 2014-09-30 Tekmira Pharmaceuticals Corp Ulepszone aminolipidy i sposoby dostarczania kwasów nukleinowych
CN103937793B (zh) 2008-10-20 2017-08-11 阿尔尼拉姆医药品有限公司 抑制运甲状腺素蛋白表达的组合物和方法
EP3808177A1 (en) 2008-11-10 2021-04-21 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
WO2010057155A1 (en) 2008-11-17 2010-05-20 Enzon Pharmaceuticals, Inc. Releasable cationic lipids for nucleic acids delivery systems
JP2012509258A (ja) 2008-11-17 2012-04-19 エンゾン ファーマシューティカルズ，インコーポレーテッド 核酸送達系のための分岐カチオン性脂質
US10292932B2 (en) 2008-12-26 2019-05-21 Samyang Biopharmaceuticals Corporation Polymeric micelle particle comprising anionic drugs and method of preparing the same
BRPI1012246A2 (pt) 2009-03-25 2016-03-29 Novartis Ag composição farmacêutica contendo um fármaco e sirna
EP2416760A4 (en) 2009-05-05 2014-01-22 Tekmira Pharmaceuticals Corp LIPID COMPOSITIONS
PL3431076T3 (pl) 2009-06-10 2022-01-31 Arbutus Biopharma Corporation Ulepszona formulacja lipidowa
JP5766188B2 (ja) 2009-07-01 2015-08-19 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 固形腫瘍に治療剤を送達するための脂質製剤
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
WO2011020023A2 (en) * 2009-08-14 2011-02-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
CA2773886C (en) 2009-09-11 2018-01-09 Isis Pharmaceuticals, Inc. Modulation of huntingtin expression
CA2816925C (en) 2009-11-04 2023-01-10 The University Of British Columbia Nucleic acid-containing lipid particles and related methods
NZ716192A (en) 2009-12-01 2017-07-28 Shire Human Genetic Therapies Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
CA2783001C (en) 2010-01-19 2017-12-12 Polypid Ltd. Sustained-release nucleic acid matrix compositions
US10077232B2 (en) 2010-05-12 2018-09-18 Arbutus Biopharma Corporation Cyclic cationic lipids and methods of use
KR101198715B1 (ko) 2010-05-14 2012-11-13 한국생명공학연구원 핵산 및 친수성 음이온 화합물의 고효율 포획을 위한 비대칭 리포솜 및 이의 제조방법
US20100239521A1 (en) 2010-06-01 2010-09-23 Mohammad Reza Mozafari Method for the preparation of micro- and nano-sized carrier systems for the encapsulation of bioactive substances
KR20190039347A (ko) 2010-06-03 2019-04-10 알닐람 파마슈티칼스 인코포레이티드 활성제의 전달을 위한 생분해성 지질
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
PL4008357T3 (pl) 2010-08-31 2023-03-06 Glaxosmithkline Biologicals Sa Małe liposomy do dostarczania rna kodującego immunogen
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
EP2718269B1 (en) 2011-06-08 2018-01-31 Translate Bio, Inc. Cleavable lipids
DK2717893T3 (da) 2011-06-08 2019-07-22 Translate Bio Inc Lipid nanopartikelsammensætninger og fremgangsmåder til mrna-levering
US9011903B2 (en) 2011-06-08 2015-04-21 Nitto Denko Corporation Cationic lipids for therapeutic agent delivery formulations
WO2013004234A2 (en) 2011-07-04 2013-01-10 Statens Serum Institut Methods for producing liposomes
ES2705498T3 (es) 2011-08-31 2019-03-25 Glaxosmithkline Biologicals Sa Liposomas pegilados para administración de ARN que codifica inmunógeno
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
AU2012318249B2 (en) 2011-10-05 2016-04-21 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
US9579338B2 (en) 2011-11-04 2017-02-28 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
RU2647476C2 (ru) 2011-11-04 2018-03-15 Нитто Денко Корпорейшн Способ получения липидных наночастиц для доставки лекарственного средства
DK2791160T3 (da) 2011-12-16 2022-05-30 Modernatx Inc Modificerede mrna-sammensætninger
KR102075810B1 (ko) 2012-02-24 2020-02-10 아뷰터스 바이오파마 코포레이션 트리알킬 양이온성 지질 및 그의 사용 방법
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
DK2830595T3 (da) 2012-03-29 2019-12-02 Translate Bio Inc Ioniserbare kationiske lipider
US20160289674A1 (en) 2012-04-02 2016-10-06 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
DE18200782T1 (de) 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
EP3489220B9 (en) 2012-06-08 2021-11-10 Nitto Denko Corporation Lipids for therapeutic agent delivery formulations
CA2876155C (en) 2012-06-08 2022-12-13 Ethris Gmbh Pulmonary delivery of mrna to non-lung target cells
BR112015022855A2 (pt) 2013-03-14 2017-11-07 Shire Human Genetic Therapies composições e método para produção de um anticorpo in vitro
ES2797974T3 (es) 2013-03-14 2020-12-04 Translate Bio Inc Acidos ribonucleicos con nucleótidos modificados con 4-tio y procedimientos relacionados
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
EP2971013B1 (en) 2013-03-15 2020-08-19 The University Of British Columbia Lipid nanoparticles for transfection and related methods
KR102109188B1 (ko) 2013-04-01 2020-05-11 삼성전자주식회사 양이온성 지질을 포함하는 온도민감성 리포좀 및 그의 용도
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
EP3677567A1 (en) 2013-07-23 2020-07-08 Arbutus Biopharma Corporation Compositions and methods for delivering messenger rna
US9453060B2 (en) 2013-08-09 2016-09-27 New York University Protein engineered systems for delivery of molecules
CN105658800A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 Mrna的cns递送及其用途
EP3060303B1 (en) 2013-10-22 2018-11-14 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
EP3060257B1 (en) 2013-10-22 2021-02-24 Translate Bio, Inc. Lipid formulations for delivery of messenger rna
EA201992208A1 (ru) 2013-10-22 2020-07-31 Транслейт Био, Инк. ЛЕЧЕНИЕ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
US10561610B2 (en) 2014-01-21 2020-02-18 Anjarium Biosciences Ag Hybridosomes, compositions comprising the same, processes for their production and uses thereof
DK3116997T3 (da) 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
ES2774552T3 (es) 2014-03-24 2020-07-21 Translate Bio Inc Terapia de ARNm para el tratamiento de enfermedades oculares
WO2015154002A1 (en) 2014-04-04 2015-10-08 Ohio State Innovation Foundation Oligonucleotide lipid nanoparticle compositions, methods of making and methods of using the same
RU2746406C2 (ru) * 2014-04-23 2021-04-13 МОДЕРНАТиЭкс, ИНК. Вакцины на основе нуклеиновых кислот
JP6557722B2 (ja) 2014-05-30 2019-08-07 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生分解性脂質
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
ES2964588T3 (es) 2014-06-24 2024-04-08 Translate Bio Inc Composiciones enriquecidas estereoquímicamente para la administración de ácidos nucleicos
JP2017520549A (ja) 2014-06-26 2017-07-27 ラモット アット テル アビブ ユニバーシティ， リミテッド 核酸の送達のためのリポソーム製剤
CN106456547B (zh) 2014-07-02 2021-11-12 川斯勒佰尔公司 信使rna的包封
ES2861597T3 (es) 2014-12-05 2021-10-06 Translate Bio Inc Terapia de ARN mensajero para el tratamiento de enfermedad articular
CN107530436B (zh) 2015-01-21 2022-03-29 菲泽尔克斯公司 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统
US20180085474A1 (en) 2015-01-23 2018-03-29 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
CA2979695A1 (en) 2015-03-19 2016-09-22 Translate Bio, Inc. Mrna therapy for pompe disease
US10920246B2 (en) 2015-05-26 2021-02-16 Ramot At Tel-Aviv University Ltd. Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes
WO2017008076A1 (en) 2015-07-09 2017-01-12 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
SI3350157T1 (sl) 2015-09-17 2022-04-29 Modernatx, Inc. Sestave za doziranje terapevtskih sredstev v celice
WO2017070618A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Cancer vaccines
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
EP3364981A4 (en) 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
WO2017075038A1 (en) 2015-10-26 2017-05-04 Rana Therapeutics, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
DK3386484T3 (da) 2015-12-10 2022-07-04 Modernatx Inc Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler
CN108366964B (zh) 2015-12-18 2022-04-08 三养控股公司 制备含阴离子药物的聚合物胶束的方法
JP7114465B2 (ja) 2015-12-22 2022-08-08 モデルナティエックス インコーポレイテッド 薬剤の細胞内送達のための化合物および組成物
CN107281103A (zh) 2016-04-11 2017-10-24 百奥迈科生物技术有限公司 具有高含量阳性脂质化合物的脂质体制剂及其应用
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2017201332A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding acyl-coa dehydrogenase, very long-chain for the treatment of very long-chain acyl-coa dehydrogenase deficiency
US10835583B2 (en) 2016-06-13 2020-11-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
WO2017218704A1 (en) 2016-06-14 2017-12-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
US20210284998A1 (en) 2016-10-03 2021-09-16 Precision Nanosystems Inc. Compositions for Transfecting Resistant Cell Types
WO2018075980A1 (en) 2016-10-21 2018-04-26 Modernatx, Inc. Human cytomegalovirus vaccine
JP7289265B2 (ja) 2016-10-26 2023-06-09 キュアバック エスイー 脂質ナノ粒子ｍＲＮＡワクチン
DK3538515T3 (da) 2016-11-08 2022-10-17 Univ Ramot Kationiske lipider til nukleinsyreafgivelse og fremstilling deraf
WO2018089540A1 (en) 2016-11-08 2018-05-17 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
EP3538068A1 (en) 2016-11-10 2019-09-18 Translate Bio, Inc. Improved ice-based lipid nanoparticle formulation for delivery of mrna
US20180153822A1 (en) 2016-11-10 2018-06-07 Translate Bio, Inc. Process of Preparing mRNA-Loaded Lipid Nanoparticles
EP3551169A4 (en) 2016-12-09 2020-09-09 Sangamo Therapeutics, Inc. ADMINISTRATION OF TARGET-SPECIFIC NUCLEASES
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP4186888A1 (en) 2017-03-15 2023-05-31 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
JP7332478B2 (ja) 2017-03-15 2023-08-23 モデルナティエックス インコーポレイテッド 脂質ナノ粒子製剤
WO2018213726A1 (en) * 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
JP2020521781A (ja) 2017-05-30 2020-07-27 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム リポソームに内包されたｒｎａを製造する方法
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
WO2018236849A1 (en) 2017-06-19 2018-12-27 Translate Bio, Inc. MESSENGER RNA THERAPY FOR THE TREATMENT OF FRIEDREICH ATAXIA
AU2018301701A1 (en) 2017-07-14 2020-02-27 Elevatebio Technologies, Inc. Encapsulated polynucleotides and methods of use
CA3071968A1 (en) 2017-08-04 2019-02-07 Kyowa Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticle
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036000A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
CN111565706A (zh) 2017-11-09 2020-08-21 免疫疫苗技术公司 药物组合物、包括脂质囊泡颗粒尺寸设定的制备方法及其用途
US12036282B2 (en) 2017-12-06 2024-07-16 Newsouth Innovations Pty Limited Liposomal system for drug delivery
WO2019118621A1 (en) 2017-12-12 2019-06-20 The Johns Hopkins University Methods for making giant vesicles and their use
AU2018392716A1 (en) 2017-12-20 2020-06-18 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
EP3752165A1 (en) 2018-02-16 2020-12-23 Mina Therapeutics Limited C/ebp alpha sarna compositions and methods of use
US20210002640A1 (en) 2018-02-28 2021-01-07 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors
EP3775202A4 (en) 2018-03-27 2022-04-06 Factor Bioscience Inc. NUCLEIC ACID THERAPEUTIC AGENTS
EP3773476A1 (en) 2018-03-30 2021-02-17 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
EP3781591A1 (en) 2018-04-17 2021-02-24 CureVac AG Novel rsv rna molecules and compositions for vaccination
JP7379776B2 (ja) 2018-04-29 2023-11-15 プレシジョン ナノシステムズ ユーエルシー 耐性細胞型をトランスフェクトするための組成物
CN112055695A (zh) 2018-05-01 2020-12-08 弗莱德哈钦森癌症研究中心 用于基因表达的纳米粒子和其用途
WO2019217870A1 (en) 2018-05-10 2019-11-14 Northwestern University Self-manageable abnormal scar treatment with spherical nucleic acid (sna) technology
US20210346306A1 (en) 2018-05-23 2021-11-11 Modernatx, Inc. Delivery of dna
CN112437767B (zh) 2018-05-24 2023-10-27 川斯勒佰尔公司 硫酯阳离子脂质
AU2019310011A1 (en) 2018-07-21 2021-02-11 University Of Tennessee Research Foundation Neutral liposomes containing biologically active agents
CA3107055A1 (en) 2018-07-23 2020-01-30 Translate Bio, Inc. Dry powder formulations for messenger rna
WO2020028133A1 (en) 2018-07-30 2020-02-06 Trucode Gene Repair, Inc. Lipid nanoparticle formulations comprising nucleic acid mimics
JP2021535226A (ja) * 2018-09-04 2021-12-16 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 核酸を臓器特異的送達するための組成物および方法
WO2020051243A1 (en) 2018-09-04 2020-03-12 Ohio State Innovation Foundation Lipid nanoparticles and methods of using thereof
WO2020056294A1 (en) 2018-09-14 2020-03-19 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
PT3864163T (pt) 2018-10-09 2024-04-30 Univ British Columbia Composições e sistemas que compreendem vesículas competentes para transfeção isentas de solventes orgânicos e detergentes e métodos relacionados com as mesmas
CA3117877A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Peg lipidoid compounds
EP3876914A2 (en) 2018-11-09 2021-09-15 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
CA3118557C (en) 2018-11-09 2024-05-28 Arbutus Biopharma Corporation Negatively charged peg-lipid conjugates
US20220001029A1 (en) 2018-11-09 2022-01-06 Arbutus Biopharma Corporation Lipid nanoparticle formulations
US20220133636A1 (en) 2018-11-09 2022-05-05 Arbutus Biopharma Corporation Cationic lipids containing silicon
CA3119449A1 (en) 2018-11-12 2020-05-22 Translate Bio, Inc. Methods for inducing immune tolerance
US11708575B2 (en) 2018-11-16 2023-07-25 Nitto Denko Corporation RNA interference delivery formulation and methods for malignant tumors
US20200157157A1 (en) 2018-11-21 2020-05-21 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
JP2022516318A (ja) 2019-01-04 2022-02-25 オンコラス， インコーポレイテッド カプセル化ポリヌクレオチド及び使用方法
US20220151928A1 (en) 2019-01-24 2022-05-19 Northwestern University High density lipoprotein mimetic nanoparticles using lipid conjugated core scaffolds
US20220023214A1 (en) 2019-01-24 2022-01-27 CannaClear Inc. Lecithin Vesicles for Oral Delivery
CN113767172A (zh) 2019-01-31 2021-12-07 新南创新私人有限公司 脂质体纳米颗粒
CA3128215A1 (en) 2019-01-31 2020-08-06 Modernatx, Inc. Methods of preparing lipid nanoparticles
JP2022525540A (ja) 2019-03-19 2022-05-17 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 脂質封入ｒｎａナノ粒子の製造方法
WO2020189773A1 (ja) 2019-03-20 2020-09-24 洋文 竹内 後眼部送達用組成物
JP7447389B2 (ja) 2019-04-15 2024-03-12 プレシジョン ナノシステムズ ユーエルシー Ｔ細胞遺伝子発現の非ウイルス性改変
MX2021013418A (es) 2019-05-03 2021-12-10 Dicerna Pharmaceuticals Inc Moleculas inhibidoras de acido nucleico bicatenario con cadenas de sentido acortadas.
AU2020268388A1 (en) 2019-05-07 2021-12-02 Modernatx, Inc. Polynucleotides for disrupting immune cell activity and methods of use thereof
EP3968952A1 (en) 2019-05-14 2022-03-23 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
WO2020252375A1 (en) 2019-06-14 2020-12-17 Dnalite Therapeutics, Inc. Compositions and methods for biological delivery vehicles
EP3986452A1 (en) 2019-06-18 2022-04-27 CureVac AG Rotavirus mrna vaccine
US20220396548A1 (en) 2019-06-20 2022-12-15 Precision NanoSystems ULC Ionizable lipids for nucleic acid delivery
EP3990441A4 (en) 2019-06-29 2022-08-24 Precision Nanosystems Inc IONIZABLE LIPIDS FOR NUCLEIC ACID DELIVERY
CA3146675A1 (en) 2019-07-23 2021-01-28 Translate Bio, Inc. Stable compositions of mrna-loaded lipid nanoparticles and processes of making
KR20220040484A (ko) 2019-07-29 2022-03-30 조지아 테크 리서치 코포레이션 구속된 지질을 함유하는 나노물질 및 그의 용도
AU2020321021A1 (en) 2019-07-30 2022-03-10 Verndari, Inc. Virus-like particle vaccines
AU2020319876A1 (en) 2019-07-31 2022-02-24 Modernatx, Inc. Compositions and methods for delivery of RNA interference agents to immune cells
WO2021026358A1 (en) 2019-08-07 2021-02-11 Moderna TX, Inc. Compositions and methods for enhanced delivery of agents
CA3150458A1 (en) 2019-08-14 2021-02-18 Acuitas Therapeutics, Inc. Improved lipid nanoparticles for delivery of nucleic acids
AU2020328855A1 (en) 2019-08-14 2022-03-03 CureVac SE RNA combinations and compositions with decreased immunostimulatory properties
EP4021407A1 (en) 2019-08-30 2022-07-06 GlaxoSmithKline Biologicals S.A. Jet mixing lipid nanoparticle manufacturing process
WO2021055660A1 (en) 2019-09-18 2021-03-25 The Regents Of The University Of California Protein producing nanoliposomes and uses thereof
US11066355B2 (en) 2019-09-19 2021-07-20 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
CN116096428A (zh) 2019-10-22 2023-05-09 盖纳万科学有限公司 鸟氨酸氨甲酰基转移酶(otc)构建体及其使用方法
BR112022007481A2 (pt) 2019-11-22 2022-07-12 Generation Bio Co Lipídios ionizáveis e composições de nanopartículas dos mesmos
WO2021108731A1 (en) 2019-11-25 2021-06-03 The Methodist Hospital Aposome compositions and methods of use
EP3838266A1 (en) 2019-12-20 2021-06-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microfluidic production of biofunctionalized giant unilamellar vesicles for targeted intracellular cargo delivery
KR20220130712A (ko) 2019-12-31 2022-09-27 프레드 허친슨 캔서 센터 치료 부위에서 면역계 반응을 자극하고 유지하기 위한 나노입자 시스템
IL294866A (en) 2020-01-31 2022-09-01 Modernatx Inc Methods for preparing fat nanoparticles
EP3858332A1 (en) 2020-01-31 2021-08-04 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Bottom-up assembly of synthetic extracellular vesicles
CA3160511A1 (en) 2020-02-04 2021-08-12 Susanne RAUCH Coronavirus vaccine
CN113186203B (zh) 2020-02-13 2022-12-30 斯微(上海)生物科技股份有限公司 治疗或者预防冠状病毒病的疫苗试剂
AU2021226578A1 (en) 2020-02-25 2022-10-20 Translate Bio, Inc. Improved processes of preparing mRNA-loaded lipid nanoparticles
CN113999184A (zh) 2020-02-28 2022-02-01 深圳深信生物科技有限公司 一种胺基脂质化合物、其制备方法和应用
CA3173126A1 (en) 2020-03-27 2021-09-30 Matthew G. Stanton Nucleic acid sequences encapsulated in lipid nanoparticles
US11559577B2 (en) 2020-03-31 2023-01-24 Engimata, Inc. Immunogenic composition forming a vaccine, and a method for its manufacture
CN113456591B (zh) 2020-03-31 2022-06-14 武汉合生科技有限公司 糖基聚醚类化合物脂质体及其制备方法和药物
EP4132584A4 (en) 2020-04-07 2024-05-01 The Regents Of The University Of California MANUFACTURE OF SYNTHETIC EXOSOMES FOR CNS AND NON-CNS DELIVERY OF THERAPEUTIC AGENTS
EP4132461A1 (en) 2020-04-10 2023-02-15 Northwestern University Oxidized tumor cell lysates encapsulated in liposomal spherical nucleic acids as potent cancer immunotherapeutics
MX2022013254A (es) 2020-04-22 2023-01-24 BioNTech SE Vacuna contra el coronavirus.
WO2021222801A2 (en) 2020-05-01 2021-11-04 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
WO2021231901A1 (en) 2020-05-15 2021-11-18 Translate Bio, Inc. Lipid nanoparticle formulations for mrna delivery
EP4153144A4 (en) 2020-05-18 2024-07-03 Generation Bio Co NOVEL LIPIDS AND NANOPARTICLE COMPOSITIONS THEREOF
US20210353554A1 (en) 2020-05-18 2021-11-18 Max Biology Co. Ltd. Lipid-polymer compositions and methods of use
CA3179423A1 (en) 2020-05-19 2021-11-25 Robert Alexander WESSELHOEFT Circular rna compositions and methods
US20220049251A1 (en) 2020-05-20 2022-02-17 Nitto Denko Corporation dsRNA Directed to Coronavirus Proteins
EP4157456A4 (en) 2020-05-29 2024-06-26 Oncorus, Inc. ENCAPSULATED RNA REPLICONS AND METHODS OF USE
CA3170740A1 (en) 2020-05-29 2021-12-02 Curevac Ag Nucleic acid based combination vaccines
US20230029453A1 (en) 2020-07-06 2023-02-02 Janssen Biotech, Inc. Prostate Neoantigens And Their Uses
US20230338288A1 (en) 2020-07-14 2023-10-26 The Research Foundation For The State University Of New York Asymmetric charged vesicles and methods of preparing and use thereof
IL299896A (en) 2020-07-17 2023-03-01 Generation Bio Co Methods for thermoforming polynucleotides into reduced sizes of lipid nanoparticles and their new formulation
GB202011367D0 (en) 2020-07-22 2020-09-02 Micropore Tech Limited Method of preparing liposomes
US20230293434A1 (en) 2020-07-23 2023-09-21 The Regents Of The University Of California COMPOSITIONS COMPRISING miR-690 AND METHODS THEREFOR
KR20230087443A (ko) 2020-08-06 2023-06-16 모더나티엑스, 인크. 페이로드 분자를 기도 상피로 전달하기 위한 조성물
EP4192432A1 (en) 2020-08-06 2023-06-14 ModernaTX, Inc. Methods of preparing lipid nanoparticles
EP4200330A2 (en) 2020-08-19 2023-06-28 Vitruviae LLC Vaccine compositions and antibodies for lyme disease
CA3190597A1 (en) 2020-08-25 2022-03-03 Yury Miller Compositions and methods for the treatment of ocular neuroinflammation
CA3170743A1 (en) 2020-08-31 2022-03-03 Susanne RAUCH Multivalent nucleic acid based coronavirus vaccines
CA3189903A1 (en) 2020-09-11 2022-03-17 Wilfred Jefferies Vaccine for viral pathogens

Also Published As

Publication number Publication date
RS65449B1 (sr) 2024-05-31
US20230073819A1 (en) 2023-03-09
EP4379053A3 (en) 2024-09-18
PH12021550626A1 (en) 2022-02-14
AU2019359299B2 (en) 2022-04-21
PT3864163T (pt) 2024-04-30
BR112021006539A2 (pt) 2021-07-06
JP2022512578A (ja) 2022-02-07
JP2024028838A (ja) 2024-03-05
CA3114699A1 (en) 2020-04-16
PL3864163T3 (pl) 2024-05-20
US11865190B2 (en) 2024-01-09
IL282063A (en) 2021-05-31
CN113166783A (zh) 2021-07-23
EA202190688A1 (ru) 2021-06-07
HUE066181T2 (hu) 2024-07-28
EP3864163B1 (en) 2024-03-20
EP3864163A1 (en) 2021-08-18
AU2019359299A1 (en) 2021-05-13
WO2020077007A1 (en) 2020-04-16
KR20210093232A (ko) 2021-07-27
FI3864163T3 (fi) 2024-05-03
EP4379053A2 (en) 2024-06-05
DK3864163T3 (da) 2024-05-06
AU2022203149A1 (en) 2022-06-02
EP3864163A4 (en) 2022-08-10
US11980673B2 (en) 2024-05-14
US20220118112A1 (en) 2022-04-21

Similar Documents

Publication Publication Date Title
HRP20240586T1 (hr) Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
US20240018514A1 (en) Large-scale synthesis of messenger rna
US20230372245A1 (en) Lipid formulations for nucleic acid delivery
US12037600B2 (en) Transfection complexes and methods of using the same
US11866723B2 (en) Plant virus movement proteins and methods of using same
US11572575B2 (en) Compositions for transfecting resistant cell types
Kariko et al. Phosphate-enhanced transfection of cationic lipid-complexed mRNA and plasmid DNA
US11814333B2 (en) Cationic lipids and transfection methods
US20110313017A1 (en) Snalp formulations containing polyoxazoline-dialkyloxypropyl conjugates
US20240148663A1 (en) Peg lipids and lipid nanoparticles
WO2018119514A1 (en) Compositions for transfecting resistant cell types
US11679159B2 (en) Compositions for transfecting resistant cell types
Zhang et al. Nucleic acid degradation as barrier to gene delivery: a guide to understand and overcome nuclease activity
WO2024028492A1 (en) Quantitative assessment of rna encapsulation
Patra Lipofectamine mediated gene delivery: Cationic lipid helps to overcome the cell-surface barriers in hepatocytes
JPWO2020077007A5 (hr)