SI2972360T1 - Sinergistično izboljšanje dostave nukleinskih kislin preko mešanih formulacij - Google Patents

Sinergistično izboljšanje dostave nukleinskih kislin preko mešanih formulacij Download PDF

Info

Publication number
SI2972360T1
SI2972360T1 SI201430708T SI201430708T SI2972360T1 SI 2972360 T1 SI2972360 T1 SI 2972360T1 SI 201430708 T SI201430708 T SI 201430708T SI 201430708 T SI201430708 T SI 201430708T SI 2972360 T1 SI2972360 T1 SI 2972360T1
Authority
SI
Slovenia
Prior art keywords
mrna
cells
pharmaceutical composition
lipid
lipid nanoparticle
Prior art date
Application number
SI201430708T
Other languages
English (en)
Inventor
Frank Derosa
Lianne Smith
Michael Heartlein
Braydon Charles Guild
Original Assignee
Translate Bio, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Translate Bio, Inc. filed Critical Translate Bio, Inc.
Publication of SI2972360T1 publication Critical patent/SI2972360T1/sl

Links

Classifications

    • 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
    • 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
    • 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—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/146—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic macromolecular compounds
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/543—Immunoassay; Biospecific binding assay; Materials therefor with an insoluble carrier for immobilising immunochemicals

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Claims (17)

  1. Sinergistično izboljšanje dostave nukleinskih kislin preko mešanih formulacij Patentni zahtevki
    1. Farmacevtski sestavek za dostavo polinukleotida sporočilne RNA (mRNA) eni ali več tarčnih celic, kjer omenjeni sestavek vsebuje mRNA in mešanico vsaj prvega lipidnega nanodelca in drugega lipidnega nanodelca, kjer prvi lipidni nanodelec vsebuje mRNA; in, kjer prvi lipidni nanodelec vsebuje vsaj en lipid, ki se razlikuje od drugega lipidnega nanodelca.
  2. 2. Farmacevtski sestavek po zahtevku 1, kjer drugi lipidni nanodelec vsebuje mRNA, kije (i) enaka kot mRNA v prvem lipidnem nanodelcu ali (ii) ni enaka kot mRNA v prvem lipidnem nanodelcu.
  3. 3. Farmacevtski sestavek po zahtevku 1, kjer je omenjeni vsaj en različen lipid kationski lipid, kjer je kationski lipid neobvezno izbran iz skupine, ki vsebuje 02-200, DOTAP (l,2-diolel-3-trimetilamonijev propan), DODAP (1,2-dioleil-3-dimetilamonijev propan), DOTMA (l,2-di-O-oktadecenil-3-trimetilamonijev propan), DLinDMA, DLin-KC2-DMA, HGT4003 (2-((2,3-Bis((9Z,12Z)-oktadeka-9,12-dien-1 -iloksi)propil)disulfanil)-N,N-dimetiletanamin) in ICE (imidazol holesterol ester).
  4. 4. Farmacevtski sestavek po zahtevku 1, kjer prvi lipidni nanodelec in drugi lipidni nanodelec vsebujeta: (i) enega ali več pomožnih lipidov, kjer je eden ali več pomožnih lipidov neobvezno izbran iz skupine, ki vsebuje DSPC (1,2-distearoil-sn-glicero-3-fosfoholin), DPPC (1,2-dipalmitoil-sn-glicero-3-fosfoholin), DOPE (l,2-dioleil-sn-glicero-3-fosfoetanolamin), DPPE (l,2-dipalmitoil-sn-glicero-3-fosfoetanolamin), DMPE (1,2-dimiristoil-sn-glicero-3-fosfoetanolamin), DOPG (1,2-dioleoil-sn-glicero-3-fosfo-(l’-rac-glicerol)) in holesterol, ali (ii) enega ali več PEG-modificiranih lipidov, kjer eden ali več PEG- modificiranih lipidov neobvezno vsebuje verigo poli(etilen)glikol z dolžino do 5kDa, kovalentno vezano na lipid, ki vsebuje eno ali več alkilnih verig z dolžino C6-C20.
  5. 5. Farmacevtski sestavek po zahtevku 1, kjer prvi lipidni nanodelec ali drugi lipidni nanodelec vsebuje (i) DLinDMA, CHOL, DOPE in DMG-PEG-2000, (ii) C12-200, CHOL, DOPE in DMG-PEG-2000, (iii) DLinKC2, CHOL, DOPE in DMG-PEG-2000, (iv) enega ali več lipidov izbranih iz skupine, ki vsebuje ICE, DSPC, CHOL, DODAP, DOTAP in C8-PEG-2000, (v) DSPC, CHOL, DODAP in C8-PEG-2000, (vi) ICE, DOPE in DMG-PEG-2000 ali (vii) HGT4003, DOPE, CHOL in DMG-PEG-2000.
  6. 6. Farmacevtski sestavek po kateremkoli predhodnem zahtevku, kjer mRNA kodira: (i) encim, kjer je encim neobvezno izločen iz ene ali več tarčnih celic; (ii) protein, ki je izločen iz tarčne celice; ali (iii) polipeptid, ki je ga izraža subjekt, kjer je polipeptid neobvezno izbran iz skupine, ki vsebuje agalzidazo alfa, alfa-L-iduronidazo, iduronat-2-sulfatazo, N-acetilglucozamin-1 -fosfat transferazo, beta-glukozidazo, galaktoza-6-sulfat sulfatazo, beta-galaktosidazo, glukocerebrozidazo, heparan sulfamidazo, hialuronidazo, galaktocerebrozidazo, ornitin transkarbamilazo (OTC), karbamoil-fosfat sintetazo 1 (CPS1), argininosukcinat sintetazo (ASS1), argininosukcinat liazo (ASL) in arginazo 1 (ARG1); kjer je mRNA neobvezno izbrana izmed SEQ ID NO: 2 ali SEQ ID NO: 3.
  7. 7. Farmacevtski sestavek po kateremkoli predhodnem zahtevku, kjer mRNA vsebuje kemično modifikacijo, kjer neobvezno (i) kemična modifikacija naredi mRNA bolj stabilno; (ii) kemična modifikacija vsebuje modifikacijo, ki blokira konec 5’ neprevedljive regije mRNA; (iii) kemična modifikacija vsebuje modifikacijo, ki blokira konec 3’ neprevedljive regije mRNA; (iv) kemična modifikacija vsebuje vključitev delnega zaporedja CMV gena 1 takoj-zgodaj (IE1) v 5’ neprevedljivo regijo mRNA; (v) kemična modifikacija vsebuje vključitev poli A repa do 3’ neprevedljive regije mRNA; (vi) kemična modifikacija vsebuje vključitev strukture Capi do 5’ neprevedljive regije mRNA; ali (vii) kemična modifikacija vsebuje vključitev zaporedja, ki kodira človeški rastni hormon (hGH) do 3’ neprevedljive regije mRNA.
  8. 8. Farmacevtski sestavek po kateremkoli predhodnem zahtevku, kjer je razmerje prvega lipidnega nanodelca proti drugemu lipidnemu nanodelcu v farmacevtskem sestavku približno 1:1, približno 2:1, približno 3:1 ali približno 4:1.
  9. 9. Farmacevtski sestavek po kateremkoli zahtevku od 1 do 8, za uporabo kot zdravilo.
  10. 10. Farmacevtski sestavek, za uporabo po zahtevku 9, kjer je mRNA dostavljena tarčnim celicam, ki so izbrane iz skupine, ki vsebuje hepatocite, epitelne celice, hematopoetske celice, epitelne celice, endotelne celice, pljučne celice, kostne celice, zarodne celice, mezenhimske celice, nevronske celice, srčne celice, adipocite, vaskulame gladke mišične celice, kardiomiocite, celice skeletnih mišic, beta celice, celice hipofize, celice sinovialne ovojnice, celice jajčnika, celice testisa, fibroblaste, B celice, T celice, retikulocite, levkocite, granulocite in tumorske celice, kjer ima adminicija sestavka neobvezno za posledico izražanje mRNA v tarčnih celicah.
  11. 11. Farmacevtski sestavek za uporabo po zahtevku 9 ali 10, kjer izražanje mRNA, ki sledi administraciji farmacevtskega sestavka subjektu presega izražanje mRNA administrirane s prvim lipidnim nanodelcem vendar brez drugega lipidnega nanodelca, kjer izražanje mRNA, ki sledi administraciji farmacevtskega sestavka subjektu neobvezno presega izražanje mRNA administrirane s prvim lipidnim nanodelcem vendar brez drugega lipidnega nanodelca za vsaj dvakrat, vsaj pet krat ali vsaj deset krat.
  12. 12. Farmacevtski sestavek za uporabo po kateremkoli zahtevku od 9 do 11, kjer je farmacevtski sestavek subjektu administriran po eni ali več izmed naslednjih poti administracije: intravensko, oralno, rektalno, vaginalno, transmukozalno, sublingvalno, subduralno, nazalno, intramuskulamo, subkutano, z intramedulamo injekcijo, intratekalno, intraventrikularno, intraperitonealno, intranazalno, intracerebroventrikulamo (ICV), oftalmično in intraokularno.
  13. 13. Sestavek, ki vsebuje sporočilno RNA (mRNA), formulirano v vsaj dveh različnih lipidnih formulacijah, kjer se vsaj dve različni lipidni formulaciji razlikujeta v vsaj enem kationskem lipidu.
  14. 14. Sestavek po zahtevku 13, kjer se vsaj dve različni lipidni formulaciji nadalje razlikujeta v odstotku PEGiliranega lipida.
  15. 15. Sestavek po zahtevku 13 ali 14, kjer sta vsaj dve različni lipidni formulaciji prisotni v razmerju v območju med približno 10:1 in 1:10, neobvezno v razmerju v območju med približno 3:1 in 1:3, neobvezno v razmerju približno 1:1.
  16. 16. Sestavek po kateremkoli zahtevku od 13 do 15, kjer mRNA kodira encim.
  17. 17. Sestavek po kateremkoli zahtevku od 13 do 16 za uporabo kot zdravilo.
SI201430708T 2013-03-15 2014-03-14 Sinergistično izboljšanje dostave nukleinskih kislin preko mešanih formulacij SI2972360T1 (sl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361789375P 2013-03-15 2013-03-15
EP14721647.7A EP2972360B1 (en) 2013-03-15 2014-03-14 Synergistic enhancement of the delivery of nucleic acids via blended formulations
PCT/US2014/028498 WO2014144196A1 (en) 2013-03-15 2014-03-14 Synergistic enhancement of the delivery of nucleic acids via blended formulations

Publications (1)

Publication Number Publication Date
SI2972360T1 true SI2972360T1 (sl) 2018-08-31

Family

ID=50639976

Family Applications (1)

Application Number Title Priority Date Filing Date
SI201430708T SI2972360T1 (sl) 2013-03-15 2014-03-14 Sinergistično izboljšanje dostave nukleinskih kislin preko mešanih formulacij

Country Status (13)

Country Link
US (3) US10130649B2 (sl)
EP (4) EP3388834B1 (sl)
CY (1) CY1120507T1 (sl)
DK (2) DK3388834T3 (sl)
ES (3) ES2795249T3 (sl)
HR (1) HRP20180749T1 (sl)
HU (1) HUE039189T2 (sl)
LT (1) LT2972360T (sl)
NO (1) NO3014045T3 (sl)
PT (1) PT2972360T (sl)
RS (1) RS57316B1 (sl)
SI (1) SI2972360T1 (sl)
WO (1) WO2014144196A1 (sl)

Families Citing this family (109)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110244026A1 (en) 2009-12-01 2011-10-06 Braydon Charles Guild Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
AU2012267531B2 (en) 2011-06-08 2017-06-22 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mRNA delivery
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
EP3467108B1 (en) 2013-03-14 2024-05-22 Translate Bio, Inc. Methods for purification of messenger rna
IL290953B2 (en) 2013-03-14 2024-01-01 Ethris Gmbh CFTR mRNA Assemblies and Related Methods and Uses
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
EA201690675A1 (ru) 2013-10-03 2016-08-31 Модерна Терапьютикс, Инк. Полинуклеотиды, кодирующие рецептор липопротеинов низкой плотности
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
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
US9522176B2 (en) 2013-10-22 2016-12-20 Shire Human Genetic Therapies, Inc. MRNA therapy for phenylketonuria
SG11201608798YA (en) 2014-04-23 2016-11-29 Modernatx Inc Nucleic acid vaccines
EA201691696A1 (ru) 2014-04-25 2017-03-31 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
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
TW201718638A (zh) 2015-07-21 2017-06-01 現代治療公司 傳染病疫苗
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
EP3350157B1 (en) 2015-09-17 2022-01-05 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
EP3364950A4 (en) 2015-10-22 2019-10-23 ModernaTX, Inc. VACCINES AGAINST TROPICAL DISEASES
KR20180096591A (ko) * 2015-10-22 2018-08-29 모더나티엑스, 인크. 광범위 인플루엔자 바이러스 백신
AU2016342371B2 (en) * 2015-10-22 2023-05-11 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (VZV)
DE20164728T1 (de) 2015-10-22 2021-09-30 Modernatx, Inc. Impfstoffe gegen atemwegsvirus
AU2016342045A1 (en) * 2015-10-22 2018-06-07 Modernatx, Inc. Human cytomegalovirus vaccine
KR20180096592A (ko) * 2015-10-22 2018-08-29 모더나티엑스, 인크. 호흡기 세포융합 바이러스 백신
CN108430456B (zh) * 2015-10-22 2022-01-18 摩登纳特斯有限公司 癌症疫苗
SG11201803365RA (en) * 2015-10-22 2018-05-30 Modernatx Inc Herpes simplex virus vaccine
EP3364982A4 (en) * 2015-10-22 2019-04-17 ModernaTX, Inc. VACCINES AGAINST SEXUALLY TRANSMITTED DISEASES
EP3386484B1 (en) 2015-12-10 2022-03-30 ModernaTX, Inc. Compositions and methods for delivery of therapeutic agents
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
EP3405579A1 (en) 2016-01-22 2018-11-28 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
WO2017180917A2 (en) 2016-04-13 2017-10-19 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
DK3458083T3 (da) 2016-05-18 2023-01-30 Modernatx Inc Polynukleotider, der koder for interleukin-12 (il12), og anvendelser heraf
EP3458105B1 (en) * 2016-05-18 2024-01-17 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
CA3024625A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding citrin for the treatment of citrullinemia type 2
WO2017201328A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
US20190298657A1 (en) * 2016-05-18 2019-10-03 Modernatx, Inc. Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
EP3315125A1 (en) * 2016-10-31 2018-05-02 Silence Therapeutics (London) Ltd Lipid nanoparticle formulation
WO2018089540A1 (en) 2016-11-08 2018-05-17 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
IL266501B1 (en) 2016-11-10 2024-02-01 Translate Bio Inc An improved ICE-based lipid nanoparticle formulation for delivery of mRNA
MA47515A (fr) 2017-02-16 2019-12-25 Modernatx Inc Compositions immunogènes très puissantes
MA47603A (fr) 2017-02-27 2020-01-01 Translate Bio Inc Nouvel arnm cftr à codons optimisés
EP3592728A1 (en) 2017-03-07 2020-01-15 Translate Bio, Inc. Polyanionic delivery of nucleic acids
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
SG11201907916TA (en) 2017-03-15 2019-09-27 Modernatx Inc Compounds and compositions for intracellular delivery of therapeutic agents
US11203569B2 (en) 2017-03-15 2021-12-21 Modernatx, Inc. Crystal forms of amino lipids
US11464848B2 (en) 2017-03-15 2022-10-11 Modernatx, Inc. Respiratory syncytial virus vaccine
AU2018234828A1 (en) 2017-03-15 2019-09-19 Modernatx, Inc. Lipid nanoparticle formulation
AU2018268859A1 (en) 2017-05-16 2019-12-12 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding CFTR
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
MA50253A (fr) 2017-09-14 2020-07-22 Modernatx Inc Vaccins à arn contre le virus zika
CA3079428A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
MA54676A (fr) 2018-01-29 2021-11-17 Modernatx Inc Vaccins à base d'arn contre le vrs
BR112020021037A2 (pt) * 2018-04-25 2021-01-19 Ethris Gmbh Composição, composição sólida, processo para a preparação de uma composição, método para preservar uma formulação de nano ou micropartícula de um agente terapeuticamente ativo, uso de um composto selecionado de alcanos c3-c5 substituídos com um ou dois grupos hidróxi, e, dispositivo
AU2019271132A1 (en) * 2018-05-15 2020-11-12 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
JP2021528426A (ja) * 2018-06-19 2021-10-21 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 ｍＲＮＡおよび長い核酸の送達のための脂質ナノ粒子組成物
WO2020012036A1 (en) 2018-07-13 2020-01-16 Genmab A/S Variants of cd38 antibody and uses thereof
EP3820890A1 (en) 2018-07-13 2021-05-19 Genmab A/S Trogocytosis-mediated therapy using cd38 antibodies
CN112930396B (zh) 2018-08-24 2024-05-24 川斯勒佰尔公司 用于纯化信使rna的方法
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
CA3111484A1 (en) 2018-09-04 2020-03-12 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
MA53734A (fr) * 2018-09-27 2021-08-04 Modernatx Inc Polynucléotides codant pour l'arginase 1 pour le traitement d'une déficience en arginase
KR20210093232A (ko) 2018-10-09 2021-07-27 더 유니버시티 오브 브리티시 콜롬비아 유기용매와 세제가 없는 형질감염 적격 소포를 포함하는 조성물과 시스템 및 관련 방법
EP4031524A1 (en) 2019-09-19 2022-07-27 ModernaTX, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
CA3165388A1 (en) * 2019-12-20 2021-06-24 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
JP2023511633A (ja) 2020-01-28 2023-03-20 モデルナティエックス インコーポレイテッド コロナウイルスｒｎａワクチン
CA3170150A1 (en) 2020-02-07 2021-08-12 Modernatx, Inc. Sars-cov-2 mrna domain vaccines
WO2021222304A1 (en) 2020-04-27 2021-11-04 Modernatx, Inc. Sars-cov-2 rna vaccines
WO2021159130A2 (en) 2020-05-15 2021-08-12 Modernatx, Inc. Coronavirus rna vaccines and methods of use
EP4217371A1 (en) 2020-09-25 2023-08-02 ModernaTX, Inc. Multi-proline-substituted coronavirus spike protein vaccines
CA3094859A1 (en) * 2020-10-01 2022-04-01 Entos Pharmaceuticals Inc. Proteolipid vesicles formulated with fusion associated small transmembrane proteins
WO2022099003A1 (en) 2020-11-06 2022-05-12 Sanofi Lipid nanoparticles for delivering mrna vaccines
WO2022150717A1 (en) 2021-01-11 2022-07-14 Modernatx, Inc. Seasonal rna influenza virus vaccines
JP2024503698A (ja) 2021-01-15 2024-01-26 モデルナティエックス インコーポレイテッド 変異型株ベースのコロナウイルスワクチン
EP4277653A1 (en) 2021-01-15 2023-11-22 ModernaTX, Inc. Variant strain-based coronavirus vaccines
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2022197624A1 (en) 2021-03-15 2022-09-22 Modernatx, Inc. Therapeutic use of sars-cov-2 mrna domain vaccines
WO2022216787A2 (en) * 2021-04-08 2022-10-13 City Of Hope Lipid nanoparticles and methods of use
AU2022258335A1 (en) 2021-04-13 2023-11-23 Modernatx, Inc. Respiratory virus combination vaccines
WO2022221440A1 (en) 2021-04-14 2022-10-20 Modernatx, Inc. Influenza-coronavirus combination vaccines
WO2022245888A1 (en) 2021-05-19 2022-11-24 Modernatx, Inc. Seasonal flu rna vaccines and methods of use
EP4355308A1 (en) 2021-06-18 2024-04-24 Sanofi Multivalent influenza vaccines
AU2021461416A1 (en) 2021-08-24 2024-02-22 BioNTech SE In vitro transcription technologies
CA3230031A1 (en) 2021-09-03 2023-03-09 Patrick Baumhof Novel lipid nanoparticles for delivery of nucleic acids
PE20240883A1 (es) 2021-09-06 2024-04-24 Genmab As Anticuerpos capaces de unirse a cd27, variantes y usos de los mismos
WO2023039396A1 (en) * 2021-09-07 2023-03-16 The Trustees Of The University Of Pennsylvania Universal influenza vaccine and methods of use
AR127298A1 (es) 2021-10-08 2024-01-10 Genmab As Anticuerpos que se unen a cd30 y cd3
CA3235867A1 (en) 2021-10-22 2023-04-27 Munir MOSAHEB Mrna vaccine composition
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
TW202346593A (zh) 2021-11-05 2023-12-01 法商賽諾菲公司 呼吸道融合病毒rna疫苗
WO2023092069A1 (en) 2021-11-18 2023-05-25 Modernatx, Inc. Sars-cov-2 mrna domain vaccines and methods of use
WO2023096858A1 (en) 2021-11-23 2023-06-01 Senda Biosciences, Inc. A bacteria-derived lipid composition and use thereof
US20230310571A1 (en) 2021-11-30 2023-10-05 Sanofi Pasteur Inc. Human metapneumovirus vaccines
WO2023107999A2 (en) 2021-12-08 2023-06-15 Modernatx, Inc. Herpes simplex virus mrna vaccines
WO2023111262A1 (en) 2021-12-17 2023-06-22 Sanofi Lyme disease rna vaccine
WO2023122080A1 (en) 2021-12-20 2023-06-29 Senda Biosciences, Inc. Compositions comprising mrna and lipid reconstructed plant messenger packs
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023196914A1 (en) 2022-04-08 2023-10-12 Modernatx, Inc. Influenza nucleic acid compositions and uses thereof
WO2023214082A2 (en) 2022-05-06 2023-11-09 Sanofi Signal sequences for nucleic acid vaccines
WO2023230481A1 (en) 2022-05-24 2023-11-30 Modernatx, Inc. Orthopoxvirus vaccines
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2024015890A1 (en) 2022-07-13 2024-01-18 Modernatx, Inc. Norovirus mrna vaccines
WO2024044108A1 (en) 2022-08-22 2024-02-29 The Henry M. Jackson Foundation For The Advancement Of Military Medicine, Inc. Vaccines against coronaviruses
WO2024050483A1 (en) 2022-08-31 2024-03-07 Modernatx, Inc. Variant strain-based coronavirus vaccines and uses thereof
WO2024083345A1 (en) 2022-10-21 2024-04-25 BioNTech SE Methods and uses associated with liquid compositions
US20240156949A1 (en) 2022-10-28 2024-05-16 Glaxosmithkline Biologicals Sa Nucleic Acid Based Vaccine
WO2024094876A1 (en) 2022-11-04 2024-05-10 Sanofi Methods for messenger rna tailing
WO2024094881A1 (en) 2022-11-04 2024-05-10 Sanofi Respiratory syncytial virus rna vaccination
WO2024102434A1 (en) 2022-11-10 2024-05-16 Senda Biosciences, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs

Family Cites Families (144)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5223263A (en) 1988-07-07 1993-06-29 Vical, Inc. Liponucleotide-containing liposomes
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
US4946683A (en) 1987-11-18 1990-08-07 Vestar, Inc. Multiple step entrapment/loading procedure for preparing lipophilic drug-containing liposomes
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5194654A (en) 1989-11-22 1993-03-16 Vical, Inc. Lipid derivatives of phosphonoacids for liposomal incorporation and method of use
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
IL122290A0 (en) 1995-06-07 1998-04-05 Inex Pharmaceuticals Corp Lipid-nucleic acid complex its preparation and use
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US5677124A (en) 1996-07-03 1997-10-14 Ambion, Inc. Ribonuclease resistant viral RNA standards
FR2786104B1 (fr) * 1998-11-25 2002-12-27 Centre Nat Rech Scient Inhibiteurs de l'activation de nf-kb, et leurs utilisations pharmaceutiques
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
WO2004002453A1 (en) 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Method and apparatus for producing liposomes
JP2006516192A (ja) * 2002-10-18 2006-06-29 ジェネンテック・インコーポレーテッド 腫瘍の診断と治療のための組成物と方法
JP4842821B2 (ja) 2003-09-15 2011-12-21 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 ポリエチレングリコール修飾脂質化合物およびその使用
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
ATE537263T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Kationische lipide und verwendungsverfahren
DE102004042546A1 (de) 2004-09-02 2006-03-09 Curevac Gmbh Kombinationstherapie zur Immunstimulation
JP2008518998A (ja) * 2004-11-05 2008-06-05 ノヴォソム アクチェンゲゼルシャフト 活性物質としてオリゴヌクレオチドを含む医薬組成物におけるまたはそれに関する改善
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
FR2904144A1 (fr) 2006-07-19 2008-01-25 St Microelectronics Rousset Procede de fabrication d'un wafer de semi-conducteur comprenant un filtre optique integre
JP2010519203A (ja) 2007-02-16 2010-06-03 メルク・シャープ・エンド・ドーム・コーポレイション 生物活性分子の活性を強化するための組成物及び方法
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
WO2009046739A1 (en) 2007-10-09 2009-04-16 Curevac Gmbh Composition for treating prostate cancer (pca)
CA2930393C (en) 2007-12-04 2022-11-29 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
EP2238251B1 (en) 2007-12-27 2015-02-11 Protiva Biotherapeutics Inc. Silencing of polo-like kinase expression using interfering rna
WO2009088891A1 (en) 2008-01-02 2009-07-16 Alnylam Pharmaceuticals, Inc. Screening method for selected amino lipid-containing compositions
JP5788312B2 (ja) 2008-04-11 2015-09-30 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
WO2009127060A1 (en) 2008-04-15 2009-10-22 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
WO2010037408A1 (en) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
EP2350043B9 (en) 2008-10-09 2014-08-20 TEKMIRA Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
CN102245559B (zh) 2008-11-07 2015-05-27 麻省理工学院 氨基醇类脂质和其用途
CN105709229B (zh) 2008-11-10 2020-07-28 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
WO2010083615A1 (en) 2009-01-26 2010-07-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
NZ711583A (en) 2009-05-05 2017-03-31 Arbutus Biopharma Corp Lipid compositions
EP3431076B1 (en) 2009-06-10 2021-10-06 Arbutus Biopharma Corporation Improved lipid formulation
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
WO2011000108A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8716464B2 (en) 2009-07-20 2014-05-06 Thomas W. Geisbert Compositions and methods for silencing Ebola virus gene expression
US20110244026A1 (en) * 2009-12-01 2011-10-06 Braydon Charles Guild Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
US9687550B2 (en) 2009-12-07 2017-06-27 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
US20130017223A1 (en) 2009-12-18 2013-01-17 The University Of British Columbia Methods and compositions for delivery of nucleic acids
JP2013527856A (ja) 2010-05-12 2013-07-04 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 陽イオン性脂質およびその使用方法
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
LT3590949T (lt) 2010-10-01 2022-07-25 Modernatx, Inc. Ribonukleorūgštys, kurių sudėtyje yra n1-metil-pseudouracilų, ir jų naudojimas
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
WO2012116715A1 (en) 2011-03-02 2012-09-07 Curevac Gmbh Vaccination in newborns and infants
WO2012113413A1 (en) 2011-02-21 2012-08-30 Curevac Gmbh Vaccine composition comprising complexed immunostimulatory nucleic acids and antigens packaged with disulfide-linked polyethyleneglycol/peptide conjugates
WO2012116714A1 (en) 2011-03-02 2012-09-07 Curevac Gmbh Vaccination in elderly patients
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
EP2710136A4 (en) 2011-05-17 2015-01-21 Moderna Therapeutics Inc MANIPULATED NUCLEIC ACIDS AND USE METHOD FOR NON-HUMAN SPINE
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
AU2012267531B2 (en) 2011-06-08 2017-06-22 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mRNA delivery
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2013039861A2 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
EP2755986A4 (en) 2011-09-12 2015-05-20 Moderna Therapeutics Inc MODIFIED NUCLEIC ACIDS AND METHODS OF USE
MX354267B (es) 2011-10-03 2018-02-21 Moderna Therapeutics Inc Star Nucléosidos, nucleótidos, y ácidos nucleicos modificados, y usos de los mismos.
MX360391B (es) * 2011-10-31 2018-10-31 Mallinckrodt Llc Composiciones liposomales combinadas para terapia contra el cancer.
WO2013130161A1 (en) 2011-12-14 2013-09-06 modeRNA Therapeutics Methods of responding to a biothreat
EP2791159A4 (en) 2011-12-14 2015-10-14 Moderna Therapeutics Inc MODIFIED NUCLEIC ACIDS, AND SHORT-TERM CARE USES THEREOF
US20130156849A1 (en) * 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
RU2014129863A (ru) 2011-12-21 2016-02-10 Модерна Терапьютикс, Инк. Способы повышения жизнеспособности или увеличения продолжительности жизни органа или экспланта органа
WO2013101690A1 (en) 2011-12-29 2013-07-04 modeRNA Therapeutics Modified mrnas encoding cell-penetrating polypeptides
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
WO2013120499A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly (a) sequence or a polyadenylation signal for increasing the expression of an encoded pathogenic antigen
RU2718053C2 (ru) 2012-02-24 2020-03-30 Протива Байотерапьютикс Инк. Триалкиловые катионные липиды и способы их использования
ES2654205T3 (es) 2012-03-27 2018-02-12 Curevac Ag Moléculas artificiales de ácido nucleico para la expresión mejorada de proteínas o péptidos
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US20150050354A1 (en) 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
JP2015518705A (ja) 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
CA2868996A1 (en) 2012-04-02 2013-10-10 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
EA201492055A1 (ru) * 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
HRP20220607T1 (hr) 2012-11-26 2022-06-24 Modernatx, Inc. Terminalno modificirana rna
WO2014089216A1 (en) 2012-12-04 2014-06-12 Tekmira Pharmaceuticals Corporation In vitro release assay for liposome encapsulated vincristine
US20150366997A1 (en) 2012-12-07 2015-12-24 Shire Human Genetics Therapies, Inc. COMPOSITIONS AND METHODS FOR mRNA DELIVERY
US20150315541A1 (en) 2012-12-13 2015-11-05 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
EP2946014A2 (en) 2013-01-17 2015-11-25 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP2968397A4 (en) 2013-03-12 2016-12-28 Moderna Therapeutics Inc DIAGNOSIS AND TREATMENT OF FIBROSIS
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
WO2014144711A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Analysis of mrna heterogeneity and stability
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
EP2971033B8 (en) 2013-03-15 2019-07-10 ModernaTX, Inc. Manufacturing methods for production of rna transcripts
US20160032273A1 (en) 2013-03-15 2016-02-04 Moderna Therapeutics, Inc. Characterization of mrna molecules
EP4279610A3 (en) 2013-03-15 2024-01-03 ModernaTX, Inc. Ribonucleic acid purification
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014152030A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Removal of dna fragments in mrna production process
WO2015006747A2 (en) 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use.
JP6620093B2 (ja) 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
KR20160044566A (ko) 2013-08-21 2016-04-25 큐어백 아게 호흡기 세포융합 바이러스
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
WO2015051173A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc Polynucleotide molecules and uses thereof
EP3058082A4 (en) 2013-10-18 2017-04-26 ModernaTX, Inc. Compositions and methods for tolerizing cellular systems
CN105873902B (zh) 2013-11-18 2019-03-08 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
EP3076994A4 (en) 2013-12-06 2017-06-07 Modernatx, Inc. Targeted adaptive vaccines
EP3053585A1 (en) 2013-12-13 2016-08-10 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
RU2717986C2 (ru) 2013-12-30 2020-03-27 Куревак Аг Искусственные молекулы нуклеиновой кислоты
US20170002060A1 (en) 2014-01-08 2017-01-05 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
EP3116535B1 (en) 2014-03-12 2019-08-07 CureVac AG Combination of vaccination and ox40 agonists
SG11201608798YA (en) 2014-04-23 2016-11-29 Modernatx Inc Nucleic acid vaccines
MX2016016170A (es) 2014-06-10 2017-03-28 Curevac Ag Metodos y medios para aumentar la produccion de arn.
US20170136132A1 (en) 2014-06-19 2017-05-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
US10286086B2 (en) 2014-06-19 2019-05-14 Modernatx, Inc. Alternative nucleic acid molecules and uses thereof
US20170175129A1 (en) 2014-06-19 2017-06-22 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
SG11201702662UA (en) 2014-10-02 2017-04-27 Protiva Biotherapeutics Inc Compositions and methods for silencing hepatitis b virus gene expression
WO2016071857A1 (en) 2014-11-07 2016-05-12 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus expression
EP3041948B1 (en) 2014-11-10 2019-01-09 Modernatx, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
EP4324473A3 (en) 2014-11-10 2024-05-29 ModernaTX, Inc. Multiparametric nucleic acid optimization
EP3247363A4 (en) 2015-01-21 2018-10-03 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
WO2016118725A1 (en) 2015-01-23 2016-07-28 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
WO2016154127A2 (en) 2015-03-20 2016-09-29 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
WO2016164762A1 (en) 2015-04-08 2016-10-13 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor egf-a and intracellular domain mutants and methods of using the same
WO2016183366A2 (en) 2015-05-12 2016-11-17 Protiva Biotherapeutics, Inc. Compositions and methods for silencing expression of hepatitis d virus rna
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
WO2016197132A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics Inc. Treating hepatitis b virus infection using crispr
WO2016201377A1 (en) 2015-06-10 2016-12-15 Moderna Therapeutics, Inc. Targeted adaptive vaccines
WO2017019891A2 (en) 2015-07-29 2017-02-02 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis b virus gene expression
JP2018527003A (ja) 2015-09-17 2018-09-20 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 安定化尾部領域を含むポリヌクレオチド
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
US20190054112A1 (en) 2015-09-18 2019-02-21 Moderna Therapeutics, Inc. Polynucleotide formulations for use in the treatment of renal diseases
WO2017102010A1 (en) 2015-12-17 2017-06-22 Biontech Rna Pharmaceuticals Gmbh Novel cytokine fusion proteins
WO2017108087A1 (en) 2015-12-21 2017-06-29 Curevac Ag Inlay for a culture plate and corresponding method for preparing a culture plate system with such inlay
EP3701963A1 (en) 2015-12-22 2020-09-02 CureVac AG Method for producing rna molecule compositions
US11248223B2 (en) 2015-12-23 2022-02-15 Curevac Ag Method of RNA in vitro transcription using a buffer containing a dicarboxylic acid or tricarboxylic acid or a salt thereof
USD787703S1 (en) 2016-04-26 2017-05-23 Curevac Ag Inlay for a culture plate

Also Published As

Publication number Publication date
EP3757570B1 (en) 2023-10-11
HUE039189T2 (hu) 2018-12-28
US20160151409A1 (en) 2016-06-02
ES2795249T3 (es) 2020-11-23
EP3388834B1 (en) 2020-04-15
US10646504B2 (en) 2020-05-12
US20200390797A1 (en) 2020-12-17
EP2972360B1 (en) 2018-03-07
PT2972360T (pt) 2018-05-25
NO3014045T3 (sl) 2018-08-04
RS57316B1 (sr) 2018-08-31
HRP20180749T1 (hr) 2018-08-10
DK2972360T3 (en) 2018-05-22
ES2670529T3 (es) 2018-05-30
EP3757570A1 (en) 2020-12-30
US10130649B2 (en) 2018-11-20
EP4332576A3 (en) 2024-04-24
ES2967701T3 (es) 2024-05-03
LT2972360T (lt) 2018-09-10
EP2972360A1 (en) 2016-01-20
EP3388834A1 (en) 2018-10-17
CY1120507T1 (el) 2019-07-10
EP3757570C0 (en) 2023-10-11
US20190216843A1 (en) 2019-07-18
EP4332576A2 (en) 2024-03-06
DK3388834T3 (da) 2020-05-04
WO2014144196A1 (en) 2014-09-18

Similar Documents

Publication Publication Date Title
SI2972360T1 (sl) Sinergistično izboljšanje dostave nukleinskih kislin preko mešanih formulacij
AU2019201924B2 (en) Lipid nanoparticle compositions and methods for mrna delivery
US20220054602A1 (en) mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
US11254936B2 (en) Nuclease resistant polynucleotides and uses thereof
ES2663360T3 (es) Lípidos escindibles
JP6561378B2 (ja) 非肺標的細胞へのｍＲＮＡの経肺送達
ES2762873T3 (es) Lípidos catiónicos ionizables
JP2019533708A (ja) メッセンジャーｒｎａの皮下送達
AU2013237874A1 (en) Lipid-derived neutral nanoparticles
Estiri et al. Therapeutic effects of mesenchymal stem cells expressing erythropoietin on cancer-related anemia in mice model
NZ618275B2 (en) Lipid nanoparticle compositions and methods for mrna delivery