WO2024259439A1 - Nanocapsules d'acide nucléique permettant l'administration de médicament - Google Patents

Nanocapsules d'acide nucléique permettant l'administration de médicament Download PDF

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Publication number
WO2024259439A1
WO2024259439A1 PCT/US2024/034387 US2024034387W WO2024259439A1 WO 2024259439 A1 WO2024259439 A1 WO 2024259439A1 US 2024034387 W US2024034387 W US 2024034387W WO 2024259439 A1 WO2024259439 A1 WO 2024259439A1
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aou
werene
nanoarce
aen
nucec
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Jessica ROUGE
Suman PAL
Divya DIVYA
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University of Connecticut
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University of Connecticut
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    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • 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
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6925—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a microcapsule, nanocapsule, microbubble or nanobubble
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6949—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit inclusion complexes, e.g. clathrates, cavitates or fullerenes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5115—Inorganic compounds
    • 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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • C12N2310/16—Aptamers

Definitions

  • rmu nanoarces aou suerrom oaressuesruon, non-sec aonseveoeoae escae enosomesasssosome c ceuar uae, anna o ecen ormuaons s an,n some cases, areeaooxc. ona, mannoocaus,ave aowoan caac anavesaeno oxze orroze ereore more eeecmne overa sa oeormuaon ane m caro.
  • arce comrsn non-omerc am cec acans covaen aaceo a am es are arraneowarearceens,n wceroocrous oe am es are aearce surace an areeror, anerocrous oe one or morenraceuar or exraceuar reeas cero assenns.en w some on eem oro momreensn,eres cearvae rous oe am es are crossnerou arazoe,oeer, or a oc w one or more ce en suerou n anoerav aaseec,neers.
  • ew ceane assem o a m acever vece.rs, se assem o ao_-, a mu-aere nucec m.ex, suraca cna coonncuaoessomes a sureraocrmaneafug w acre a are eenc,a wsueraeen aenonc nucreoc aoccs.aesxn,e-rmcaeaenmoaezoeo asnom 1 za 2 n ocu saer aaree ar,ereseonn neave care osome.saer sazeseosomeoreve orm a cae aroune ase m o_ senormuaenremaure ruureurn amnsraon.
  • scaears are nm.. emeraure ramn exerence mee mrou anoueeor smnuaesos oe m o_ s, ne amoun o .o. sonw s ae cna aracoveerz -ao °n o we manann sa. . ase ronamc sze o -osomes m, - moaeoao_eo-sos.
  • conu s maes o caoncosomes wncreasn suracan suracaane c cooncenraonsrom o .esusncaeaa were a suaneu sazee a snou saone a eneouo aonru mremr en scoauson oosomes,e caoncosomes consrucon ver.sus coonro.os somoews. a. ana mss oua oo asno. eme cso oescosuren saes o m oae moeosomes..
  • a. uer .oowereo a conon a wce aer soueroonaceae suseuenerae.oa m reease was vsuaze a rev e an assa run on an e.. enaurn eenrsecarannscreon m sreaes weno_-s are exoseoo eserase reease o .
  • acaoweearso a arcemno.n m-ceorrro euxneresrseae as rer we reons amonarerra reonsneac on aears as cacuaons sowneowe conocamaesnca an wemae.. nenseseween coroune an non-corouneereaeear saemese.rencee ssnnucoarnecsece wnacse caeor .. snoswsu.ore,s *ceno.ae, **., ***..
  • exermens weres reresenncea wvaes o o a ses an same ses.ereeararam o snca nera an consern- cesromerene consern.e . caeoor.. snoswon.oc, * ., **., ***.. merr exressona r a mcroscome course assaman o ea or cesn coreressuoen wne sne sruamm aese we mree reportedly an a ser °um ovreeern mea an coreaen merr exressonne nons. aernc a . an . or . an .
  • eren ses o sam –..xermens wereerormereemes w e a ° a.en seos cw a asn emao meno s an oca m ce ncues.
  • arre e wxresseona w ceens mo oae sames were mea.eere a ° or roomemeraure, e rrn serumree o_-s waseran weneverexroreusso cna o m evere souon.s resu suorse resus sow oncosomes orn erormereemes w eren sesnn.
  • sowess.caears arem. reee a roomemeraure amaenn oca memerers a enxrenscsuona wee wn m coeaseo sramresn w seerruem e or-an we mneeav.erxeresrsooun o ca monecrr mosome orenvere souoon_ even a-ers waas ere was no sncan emsson o merr exresson aer s. resus sownn.
  • ceseoosomes an m ranseceno ces usn o_ s ca werereae w eua concenraons o merroaencuae oncosomes an merr m noecamne.ames were was seen e wven a cesor.n cronoraosw,eosomes arneamraenns.eceoo mxc o soweeoox_c a-ows concenraons.ower concenraon oo_ s was sc er crossneormuaon as sucower conce reeneor aee eren.ere, unreae ces were se asnr caeo vnsa were u asne asor coneron vovro sus exeseescre rrorars aree averae oreeeren exermen rmen.
  • sucuaneous - seeeum eomoeneousever o m rouou oeumors are orrown one o mce.woerenssue secons mare as we reoxreessen weere cearecu anae meanmuaoreesc seonwnareen.s vaue os areneren areas mare areas.esus sowa m exressons reaso cae aove wenevere usn aamerunconazeonaomoeneoussrue seuenceunconazearces so _-s.n conras, scrameumor. weareerencesn exressonevesrououe .
  • eneraennc,e m sua-aer aen nanoar cocnerso“enan reoceaasseu oes” arerovea oeraren an aroone see.anocasues oesc nucec ac caroe.., m , encasuae nucec ac caro, a osure comrse aosomaaero osome, an a nucec ac na mea orancramewor aero sazee e nanocasue a nocasue oueraero, e..,nuenceearen o as we as reaen comeosveron os a nnuc meec aocs c oar usoe., ouesr, sen nancaonca asuvaes asescreeren, ever ssems.
  • s va m renesr,s moono mceoreceus,esac canncueas, waxes, seros, cos, caonc or anoncs,ervaze eres,osos, snos, eow.
  • an a meos onvenone meos noeen s or ce are conemae accornoe so consrue.eans onroucone anre o wne an-noawrncuoar moseean ss oennrouec aro,n a anns nooe exemeeren.
  • rovn meansvn, amnsern, s aso are orro or no, manuacurn, como en,srun,ranserrn s useeren,eerm “arumnacneu, ocras aecncsn.
  • aooca conon,sorer, orsease e.., arr amen”ncuenne aooca conon,sorer, orsease o esn or reucneeveomen oe conon,sorer, orsease, e rs cnca smoms or reevneaooca sease ors .., causn reresson oeaooca conon,sorer, or conon,s cornecr,a or smseaosme as.re aremaemoernae me oarns o anerw waseeene smcaom as oere a.areoeorca suecn nee ohariamens a mamma, anarcuar auman,u a, a eneeow.reamen aso meansrovn an acve comou
  • reersoe amoun o aera wcs suc or “eraeuca eecve amoun” uraon o asorer or one eno reuce or ameoraee sever anor sorer, cau or more smomsereo,n orrevene assemblemen o a roresson ose o rneer oerss moonre o s amsoomrser a,ssnocae or wrev aensoer reercurrence,eveomen, onse ormroveeroacc oreraeuc eecs ,eec asorer, or enance or eraeuc aen.n eecve amoun can r o anoererae..,roacc orerm “roaxs” reersore eure morean oneose.s useeren,e sasca
  • “reerence” or “con “reerence” are use wcs emoe as aasene o ro”eve mae areeermne vaue or rane, “onro” aso re rencmar aans wco assess a measure resu. eeremrss "o ceornaroeu exc”e orrm "enes or conro ces.
  • ncaeeren or oer eerormen a suae orer uness oerwse ex wse cear conrace conex.e use o an an a exames, or noemosaer aamnuaaoen oen.., “es succo aes o”,sennevneneon m uerneesso oeeerusraeenvenon anoes seccaon soue consrue asncan rwse came.oanuaene oenvenon as u an non-came eemen as essenaoeracce erenavee samese meaenrenn.
  • menss asoscose an an eemens cane --- meo o reeecrranonn ov ranuaes oo v eaacues seearerane a Erasmus mueereanne wneno seerve as a soranncaeeren, an eac substitutee vaues rane, uness oerwsenvua rece.or exame, ncororaenoe seccaon as were erees, ma a sze ranes sae as nmo nmor concenraons, nm ss amouns, anee,snenea vaues suc as nmo nm, nmo s s,ec ncmaoon.
  • nemse are n omno exam nme,s o or w nmaos se ncm,c eac., anree enxere,s asn e practiseraen comnaons o numerca vaueseween anncuneowes osse enumerae areoe consereoe exress vaue anees vaue urermore, wen "aou”, "aroxm saaee”n asscosure.
  • cnnroucn eemens oeresenscosure ore asecs an emomen eemens.mares, "a,”e “ aane,”c anve “ “aneo” aerre,”n wenene useo moeannroaucere are one or more oe mean one or m e an eemen,sneneo eorer easeem "ens.
  • escomnaons oese comonensn an e s useeren
  • “oona” or “oona” meansae suseuenescre saven ev oern cr ocru cmrscuamncsea mncae o or ma no occur, anaeescronncuesnsances were s useeren, “accu”rs r annsances wereoes no.
  • romo ca eerso a sra orrance canrocaron conann u are nomreono a, momes un,e ess o, ne-rwrose s,esco-ero.e, nr-esuena,ve exames o ancue, n-en,soen, neoen, n-ex, sec-u,so-u,er-u, n-e, n-oc, n-non, an n -meex,,-meen,,-meen, oer moees,en ma aso-ee ac sr.aen or anr “aance” croaun esx aamnensncrouue,euw aereen nwo meo --, - -, - -, an - -.
  • aox reerso an a rou
  • aseneeren aeneoe u are nooecmuare moo,e merooxu, e ano oxx,eron aoxom,.-eroreosexnave exames o aoxncue, exox. ,uox,er-uox,enox, an us usseuenerse anre, “naeon”e or “oaoen” reerso -, -r, - or -.
  • oruerrner,moure s,meermors cornsv,e sneceoe nno ce,o e acr.e e,n a re osc e use ae un uerareessn a oree c , no oon mexeannocaeensoe an ovnoe seccs oe suec maer,scoseerenr exame,aers.
  • muuncona nanoarcesor nanocasues,e are muaere, nanoarcesa are caae o enc nceorescre as muuncona sma moecuerus,oocs, oraessu ora ran oneu onrc moorneazeera weuc aereanesucacr
  • saliva aereanesucacr
  • se mu saeeureenc,e m,u or auncaromnaace nuaca acceae sa,rae are caae o nucec acever anaree noarces oescosure nanoarces caneareeo sec enenocown.ese muuncona ase nucec acans.or examce, c cutec renc-e, aonrsnexnn
  • roocrous oe amens,ees are arnrcanuees noowna-romeerarc acmeneroers,.en., woce , anerocrous oe am es are arraneoware ware an are crossne w one or morener arce surace exraceuar reease aenssuc aseasse c,earvoaeasee, es oenrea oser, morenraceuar or crossne mcee ,o w or easase,oorm a surace o c one or more nucec acans are covaen aaceo rromu a ara.zone so,meo eemeor, omr aenesn,e sureorouc wrou osn oe am es are crossne n some emomens,e muu e or more ceavaeners
  • oenraerncues an ac-aerou rosaene a ac ea, oroeezronreo,u sesm, ocra ar caozmone,naoonsemecreaorazone, cs-aconc rous.
  • oer ac-ae emomenns someen emero excmuenes,enerncues asuerou, wen some conons.
  • rocrous oe am cec acans are covaen aaceoe emomens, esrou aoeer or aen suerou.n some rocroue ooee aemr or aene re ssuuseromrou a re aacaconn o ae nnuec oerc a acene mansoe rocrou oe am ee.., one ammonumrou a oe one s one nuc n ao moee.., n someec e amcomanen.
  • surace ane sno,m aer eomeno,mens, nucec acans are caae o seecvenno a ce e nucec acan see ac cavreonraseo, o aroro ae nucec ac.n some emomensn wc conssn oumor-marers,nerns, ce suracen r,eceeororsens seeceromerou cannes, memraneransorroen, enzmes, anoes, a,ransmemraneroens,on emomensn wce nu n cmercroens.n some caro cec acan seecvenso a blazee,e cocarxa.ens so semeec eemorommerou conssn ocoroens, suar resues, an ac,e nucec acncuesennsn
  • -memazoe cues one or more o-memazoe, -me--erazoem, oer-m-a-zmocaarzooen,rme.azoe,enzmazoe,-me-,,-razoe, ncues-memaz n some emomens,e orancan en oe,-memazoe,-me--mazoe,mazoe, or mezmmazoazeo.en, a snomen e smomoe emmenos,me orancan comrses-memazoe, or- emomens,e meaonsn 2+ , anenes, orean orcanacnasn-mse-memamzoazoe.n ceran emomens,e orme n 2+ an-mema e, ann some ramewor- zoes aeocmaz
  • am nuceoes reace we non-omerc ncues oneea ora a mrornon e oee c aec erra c aeeuem ucs c ar nuecn re esac c a,o cnn s.
  • omanerermne emosmens ,eceroraeucm aens, a nucec ac comrsn one or more rozm nsenseonuceoes s, ereo.es, aamers, m,ce-wcn onuceoes s, or an comnaon auoroorne, s aom rae eomaoeeme nnucs,eoe nea,n ao raarocseo coomerses aanosc aen, wcncues or an eecronensea ,on,ocoero, coesero, a sero, n an asec, a an com a mea ceaor, or an comnaonereo.
  • ncun amns eo orean oranosn asease orsorersrove, escosure,n werencoe a snueercs cne naeveaeereo on aen o er meocreven armacoeunu oare nanoarce o reease aenresenne suec,us reeasn or exraceuar n some emomens, a meo oreeeraeuc aen oranosc aen.
  • smom assocae we c an orrevenn asease, asorer, or ene exres onons wc reure m anus reure anncreasennuenza orson.n ceoran c eamnceor omre an cso,men caonononerenoc.unes c communcaeseases suc assevererou arocess o vaccnao eran emomens,e meo n ceran emomens,e men,oo mr eoxaumae, o vaccne.
  • n s no morean aou , no morean aou , no more eners ceaveo mnor aen exanra aceouuar e,n nvoro mnomreen,a ann aonu some, e or no morean aou o aou , noessan aou , noessan ao momens, noessan aou , or noessan aou oenu , noessan aou , noessan n some emom ers ceaven annraceuar envronmen.
  • aers wcosso com ame nes esn,nenera are serca vesces comose o one or moreaerse.., am c mo casuae aueous souons.osomes arecaorme s veesacraeseesrnoecu eoces en a, cn suc a asuarsoe anosc o s evoemn rane esr.c or aea n uoa cnur aca o enor s,somnesa ar ceomosrsvee sa caare moecuese nucec acs, e.., an .
  • zwn- osecraoonncc,oesoncuose,au arec noonmeeo, nseeuarrao,,o-soaeoc-sno-necero csoaeaneoaamnonaemne,a,mooeoo -- o e-oososaaecanooanmene-,-maamemooomeeo-- mcoresxoane--caroxae -ma,amoosa eanoamne , n soosoeanoamne , anee, or a mxureereo.
  • n comnaon wme e omeor emxcense,n sus,core exaemre, csa maonce use aone,u arereera use ercenae oeer n aosome ma s.n some emomens,e ,reaeran,reae ereaeran,reaeran,reaeran we.n som ran,reaeran,reaeran, orreaeran aou e emomens,eeer s consueeween aou- e.., osome- m,o aeouraco-n.n, s aomoue em-om,e anosu,e-eer, aoue.-.,, an conesuee os aoeu --- rao,.
  • aarne as, caosrsm on cr asoarounse, mareeer-ams ceonmsonoase o craemmewcaor an.
  • s arenownorer surace area,unaeore szes, an exceona 3 + ourecremsaor s maea.
  • onarousneenen reresens arocaronrou conannoenzenencue aenas so omnee e omrooumse cnosn,ssen coo or —naonunconarousne orancans memazoe, anor-memazoe.
  • memazooem,ee onz emmoazomee,nms,aezo orea annc-aamnnsoesr seeeceacr aocm,- amnen sommazoe,- e sommeo emmeonsmeen osr,anec oraans seecerom-memazoe an-memeazoe.n n some ncans-mem 2+ azoe.
  • r meoscrereeren are crossnerou am es oescosure as oerwse ners.n ceran emomens, rou.n ceran em escreeren are crossnerou arazoe cr omens, am es oescosure as oerwseescreeren are oosesrwnseeescrorue aereone areer crroosus.nen cerraoun em ano amenens s, am es oescosure as emomens,erazoe,oeer, or aen sue uerou.n ceran o ane or aene moe one crossnnrou resusrom a reacon rou an an aze oro mo rocrou oe am ee.., one ammonum rom e onener.n one exame,erazoe crossner resus ammo an
  • na anre emerovemernoms,e non-omerc am es oescosure as oerwse --- or s eren are creorocrous oe am es oescosure as oerwseescre exraceuassne w one or morenersa are ceavae one or morenraceuar or mmore enzmr reease aens.or exame,n one emomen,eners ceavae one or e es, suc as,u nomeo,eases,roeases, eserases, or easases.n some ascoosumreen ass, oeerwnseeress ccreaevaeeren a anre es ceeraavsae.en cer oanen enmracoemueanr osr, exeranceerusa or reeease cee man.n ceran em
  • ncue asuero s oescosure as oerwseescreeren excuensu ceraenr eomu uo. or amneonse,reroun ceresa ovaeescosure as oerwseescreeren eners oescosure mae seec uner reucn conons.
  • emomen no morean ao ve ceave.or exame,n one morean aou u , no morean aou , no morean aou , non an exraceuar en,v noro mnmoreen.ann a anoouer e,m oro nom meonre, noane aou oeners ceave aou , noessan aou , noessan ssan aou , noessan aou oeners ceav aou, noessan aou, or noessan exn ceran emomenes on an exraceuar envronmen.
  • escos omens,e nucec acans anaren nucec acans o m ure as oerwseescreeren are caae o seecvenn , , ano comenao,n moe , zmes, rozmes, m, s , mcro, s , escosnur ceer asas on ee emreo rwo.
  • rwosemeesncrs,ee neurceenc ar aec caaane os anenear reeuna nounc.eocr a ecxamaen,sn o s reomuea emono canmeen ss,e n,ucecz acme, ranozsm ane, marcreon nu,c ore ac acans caae oene oonuceoesncun ansense oonuceoes n oereraeuc escosnur ceer asa on eemrwosemeesncrs,ee neurceenc ca ance naan.
  • w n,eescoscu areenrosvesa one or moreeraeuc aenss encase , or encose nosome comarmen o e muuncona nanoarce.
  • raosooe,on,ocoeroo,r c exoaemsereo, a, au soerroo,o aren, e ae rcaroonaeee nuceoe, a or an comn nsea an a mea ceaor, aarmoanereo.
  • cosen envuaeae exacosae o nanocasues oescosures amnsraon are aascann vew oeaenoereae.ca,osae an enrea useorove an eecve amoun oe nanocasuesoeaen nanocasuees.
  • n o cancer an e esrove.n som n o cancere..,rosae,reas amnsern aeraeuc e emomens,ereamen o cancerncues comosoner a eecve amoun o e nanoarcesescre eren, or amnseren seuoc, aom ao suunse acnaovrn suc a canmceer a.ssa nneoceasrsacres, or comosonsereo, canencunrean, aevan, ameoran, re o aceveeesre resu as orrow o,n n e mraon o, reuevn,ean onse o,n n roresson or more smoms ore cn sever o, anor reucn
  • eoecovre aremvoeunnn cancersrove.uc meoso aea nvua.e., a suec woo o a nanoarce or comosonereo as noeenanose w can es no sa an smoms o cancer anor wo annvenvearee cer.or exame,ea nvuas ma e "mmunze" w cancer a rsnvuaarscee..,roareonsev weoomaevne a oa cmancer asnoror o on csaenc oer smaenomss ca orrn --- onoe omro mroresmen aessocc mauea wons asesvoecoaeme wn o ceveomen o canceraensavn aenec weveomen o cance anceraen
  • ra ans cae uonorrovnor asecs omsson o maeras mae useor comnaons o maeras anor eeacnseren.
  • eua oveor ae emomensa are wne scoe o euero c o anarcuar eemenorracce oeeacnserenso smarnermeseeearrse,c anve su oc amesaneorn,s m aarneuacurer, seer, user, ssem oeraor or oer enerese oeerceve accornoe sanars o consrue asearo.own examesurerusraeeresennvenonu sou noe xnam ane wamns scoe.
  • an m ormuaonsoae are neor szaaoen a oe mmer o_ -s ueoensa oeormuaonse an m su aures warmeran - °,narcuar,averoveoe uns sce o nucease.osomes, conons, areoxco ces.ereaoree, weren ce cuaaorn encv sruornamceen csa arne, usn oeern ce mran mocaon w an oerans an sncan eors are unerwa ase rouencororaon oeren earousa cana oareosomes ssues an orans.owever, even w s socazaono secc cocesorasnoc urace mocaonsor sazaon anmrove suerromme e azaono sec
  • reess merenerra a ca rne auressesemaons.. a caoncosomeormuaon, co orescenroen,soae wn caramocoeser nsrucerom--','-meamnoeane- osoeanoaorocore-oesero an,-oeo-sn-cero-- ormecne ares va sme eecrosac an roocneracons.
  • coonssomruecso wner oeo_-s,osomes werersreare as moar raon coroormoowe snresnze uner anacnner amos anere-oeseron a: e overn uner vacuum, .e mxure wasen mn oowe resersonn m- waer anurerou a eerm exnreuer o an amraorae szeer memrane.uccessu sness oosomes was .. oonuceoen s.eueenrceesesn uave nanoscae caracerzaon are sownn.
  • nma was vere - oenaecameess neave- . was oserveae m o_s surace suracan conuaeans.neres. mueoe aer ane masn oe o_ arces cane vsuazen, unee orancosoma core,e m mearamew n .
  • wou uran aceae sannueoe nanocasueaosr.,.ensa saer,eo_ was encasuae wn a nucec ac m o_.o aceevens weeman o 12 suracan moecues one o_ n orerou enecassu,aeme o 12 suracans aeo mm o maersorme,s crossnenac m o_ corearce.ncee mceencororaenoe e usn aazo eser crossner wcs covaen u cemsrarce surace usn coer caaze aze ane ccoaon m o_-.e r.esu os ses ao_ surace crossne mbibrce resennvenors’revous sewinaocee cro nsuscence ac m
  • eresence o enzmesa are eevaeurn sec e arceseraen a secc enzmee..nc csease saesueo overexresson o cros rease exressonurnnammaon an cancer.e eser o essenraseer wreasse sne wccanee esnonseomoes en oa mnoces e cneoss.omeaeraaon, asere are evesncororae ae surace oe mcee re,e eser crossner was exe m o_-s were enucraesu vaae szeo_s usnu cemsr.
  • seaex coumn.earcesa a sze o.. e nxmcu anson w a -- . m,ncane successuormaon oe c aosve suraceoena o arce surace.e sneea se aonc suracanaer asseme ae serseouaon oea enno , an zeaoena reans sues a mono anreasersereeaaree mrces a ev or seueoe n_r se ceo-. convesr weer a menunroe.reun.
  • conrmne aon oe aer ae surace oearce.e anaavess- m na sze o.. nm.ese m o_- racons wereen usenocra ae a aunraesrs ex oeremseenasrsceeesn.ca,e a s.,n ancana ameou.n o-,, co,une an wresen eenmene mau- ---aeere anar acnes.
  • mesuss ar reeease uer a .. cearan a asesonseeaeor oowe aceae conrms m reeaseses weno conon rmer an- esn areresen, raeran w s, acc an enzmaceraaon roms m reease asesnarenaeca we oan ees Kirrase- oare orne aacreaceuer mine.esusnroucen aooca ssem uns enoco e woue sae wen exoseo enosoma es sewere accaon occurs an o_- erases.oessoess, anea ces werereae w o- mea w ans c wonaonun merr exressn m n an n- an corounere-reamen.esusroms assasee.
  • ncaeansncan amouns o m were reeaserome o__--ss cnomoaere meoa aonsomes.er amouonna o m,. werneceaevsereao no cnsoesocc --- r ceomeaseex o moocas e mnvnrmonam, suesn aecrease cance oremaure m reeasen m o_ -:oenes o.e aernanroenxressonrom s n orero assessse ae sa ananrove e aer, a cereuam wenas.
  • ooso semruesm o arn m serum, wreeere m seoreao ar ° an overrnoo awneen w aaseno oeea c oers an cmeasen resreare waer souon o merr m oe same concenraon n.noearces was use as a neave conro merr cao a wasoaeoae ae same m concenraon were use as aoncosomesm oreamens are summarzen.,., an sve conro.esus oese emsson canrsneuannoe weerae ane reave ceevse.s o m.
  • a son o merr was comaraeo, ann mannsances morean, s o mae saiseerro me soraoea coenoosnosm aen as s saeen o eea mceanormuuoraesocnen –ce sanens anass. overnn areezer, wc,oreosome maave mae mes weree maaveen aece sa n serum.ne cas earcesess unorm an nuceases aonseoorransecon w e oe m, susce o serum orsme exresson.
  • m oaeo_- s aon w ea cenesnes se arnum m ae- meoeac.amesunes
  • w su vmarmanrzeosen were.nc,u wae w an ano cenese m o_-snuce no snca ere resusncae an assa.moran,o-- wasessoxca noxc asncae m-oecamnerearaons wenese a e ne m-osome an amerunc :eeceoreccuarvaeenn co oncenora_ons.-s va s oencouao oena uez nrcea aoro nen amzeeo esxoammes oaree m ever.sse case asaren nanocarrerso secc ceeess.
  • woes-s, c mesn seruom_ree m-easorunucsona mzneu weso o mr wnmozue a aamer were seccnn annernazaona ma occur va scav n non- wase an aoweo sor ener receors.e ces wereenransaenoroen.
  • a meaersaan aeern vvo smrnuseraonroueorueseneceu omo ar.amse ar s anowscanreareorenmnn c oan annexn receorsa are orurououeumor,e aamer moeor cereraeucsseever overcomeeroem oeeroenouse muaons oescosure ma s aso noaeae amouver o oonuceoeeraeucsoumors.
  • escreeren was one ore n o m aeverenearceormuaons eraureo useunres o nanor orams o mcoram.seer u mnoeuxseec weere saus,sans caom emnoanncnemeen o --- m conven soana m aearso ao wornever o orers o manueess m a aeeroass.
  • mm rossneo_- ssoven o o o -ee was souo .
  • souon was aeoo_s an 12 suracan een aeons m woe w o ms resuesor aenseneo uneerrmna ens,rraeaeoneors a meno.esnrcoourso oraeeno suer maccaene an suracee voa aroone cc reaconeweene aneea o_- 12 s useor sness wus oes resues.e - m na,earces we ere use wou anurerurcaon.
  • aamer an aamer asroows mxen a mcrocenrueue aneae ean were cona a °or mns.noer souon wasreare waenr.nereaseuer, o m , o ase an o m- secon souonoa onane arnsa so vouuon, was cooeo roomemeraure an mxe we r.
  • na concenraon s were aeo serumree o- mea a a remove an unoun oarce asn anncueae anoorer.
  • mns.nen ° ce ans were was aemosoroeur o or m exresson.es wereenmae conoc 2 eo aow croscoe a mcrosco usn aoncse eona conoca.
  • namcro scanaemrnc sazce aenrza a snuron zaec.eoena o snesze maeras were caracerze szes were aoena usnaverneaszerano-.xac cacua monea osur aever oane a szeer amnose ursnecnamae sorwaroe.oso omerae a eoowe suseuen anaze scannn eecron mcro a- s were anass waserorme on aermossco an-aracon .
  • auma -raracomeeruae,r ovaano oeran ae. e, a curren o m an a scan see o . , w a worn voae o -ase mass secrom ° mn- was useo oan aerns.- was c ercneave moeeecon oe suracan conuae ssovenonu mce a omnm anonurm araceaxeoor resuce ane amaacnsu os soa.on. sames were

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Abstract

La présente divulgation décrit une nanocapsule d'acide nucléique à structure (MOF) organométallique liposomale multifonctionnelle multicouche capable de stabiliser une charge d'acide nucléique thérapeutique, en particulier l'ARNm. Plus particulièrement, la présente divulgation se rapporte à une nanoparticule multifonctionnelle qui fonctionne en tant que matériel d'administration d'acide nucléique qui peut à la fois administrer l'ARNm à des cellules et libérer l'ARNm des cellules d'une manière spécifique aux enzymes; et des procédés d'utilisation de telles nanoparticules dans le cadre du traitement et/ou du diagnostic de maladies et de pathologies.
PCT/US2024/034387 2023-06-16 2024-06-17 Nanocapsules d'acide nucléique permettant l'administration de médicament Pending WO2024259439A1 (fr)

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Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160271268A1 (en) * 2013-11-08 2016-09-22 Dana-Farber Cancer Institute, Inc. Nucleic acid nanostructures for in vivo agent delivery
US20210038528A1 (en) * 2017-06-23 2021-02-11 University Of Connecticut Nucleic Acid Nanocapsules for Drug Delivery and Targeted Gene Knockdown
US20210236651A1 (en) * 2017-07-13 2021-08-05 Northwestern University General and Direct Method for Preparing Oligonucleotide-Functionalized Metal-Organic Framework Nanoparticles

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160271268A1 (en) * 2013-11-08 2016-09-22 Dana-Farber Cancer Institute, Inc. Nucleic acid nanostructures for in vivo agent delivery
US20210038528A1 (en) * 2017-06-23 2021-02-11 University Of Connecticut Nucleic Acid Nanocapsules for Drug Delivery and Targeted Gene Knockdown
US20210236651A1 (en) * 2017-07-13 2021-08-05 Northwestern University General and Direct Method for Preparing Oligonucleotide-Functionalized Metal-Organic Framework Nanoparticles

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GAVITT TYLER D., HARTMANN ALYSSA K., SAWANT SHRADDHA S., MARA ARLIND B., SZCZEPANEK STEVEN M., ROUGE JESSICA L.: "A GATA3 Targeting Nucleic Acid Nanocapsule for In Vivo Gene Regulation in Asthma", ACS NANO, vol. 15, no. 7, 27 July 2021 (2021-07-27), US , pages 11192 - 11201, XP093255227, ISSN: 1936-0851, DOI: 10.1021/acsnano.0c07781 *
HERBERT FABIAN C., ABEYRATHNA SAMEERA S., ABEYRATHNA NISANSALA S., WIJESUNDARA YALINI H., BROHLIN OLIVIA R., CARRARO FRANCESCO, AM: "Stabilization of supramolecular membrane protein–lipid bilayer assemblies through immobilization in a crystalline exoskeleton", NATURE COMMUNICATIONS, vol. 12, no. 1, UK, pages 1 - 13, XP093255225, ISSN: 2041-1723, DOI: 10.1038/s41467-021-22285-y *

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