HRP20200251T1 - Proizvodnja i korištenje vitro-sintetizirane ssrna za uvođenje u ćelije sisavaca za proizvodnju biološkog ili biokemijskog učinka - Google Patents

Proizvodnja i korištenje vitro-sintetizirane ssrna za uvođenje u ćelije sisavaca za proizvodnju biološkog ili biokemijskog učinka Download PDF

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HRP20200251T1
HRP20200251T1 HRP20200251TT HRP20200251T HRP20200251T1 HR P20200251 T1 HRP20200251 T1 HR P20200251T1 HR P20200251T T HRP20200251T T HR P20200251TT HR P20200251 T HRP20200251 T HR P20200251T HR P20200251 T1 HRP20200251 T1 HR P20200251T1
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Croatia
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protein
encodes
ssrna
mrna
ribonucleic acid
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HRP20200251TT
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Judith Meis
Anthony D. PERSON
Cynthia S. Chin
Jerome Jendrisak
Gary A. Dahl
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Cellscript, Llc
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Claims (15)

1. Obrađeni sastav ribonukleinske kiseline (dalje u tekstu:RNA), koji sadrži in vitro sintetiziranu ssRNA ili mRNA, gdje je manje od 0,01%, poželjno manje od 0,001%, nadalje, poželjno manje od 0,0002% mase RNA u spomenutom tretiranom sastavu RNA dvolančana RNA (dsRNA) veličine veće od oko 40 duljina parova; pri čemu je navedeni obrađeni sastav ribonukleinske kiseline dobiven obradom ssRNA sintetiziranom in vitro s proteinom specifičnim za dsRNA endoribonukleaza III u puferiranoj vodenoj otopini koja sadrži katione magnezija u koncentraciji od oko 1-4 mM, te soli koja daje ionsku snagu ekvivalentnu barem oko 50 mM kalijevog acetata ili kalijevog glutamata.
2. Obrađeni sastav ribonukleinske kiseline prema zahtjevu 1, naznačen time da spomenuto liječenje nadalje obuhvaća pročišćavanje ili čišćenje navedene ssRNA ili mRNA uklanjanjem reakcijskih komponenti endoribonukleaze III i produkta digestije dsRNA.
3. Obrađeni sastav ribonukleinske kiseline prema bilo kojem od zahtjeva 1 ili 2, naznačena time, da je navedena ssRNA ili mRNA karakterizirana najmanje jednim od sljedećeg: i) to je proizvod in vitro transkripcije DNA obrasca RNA polimerazom; ii) da je mRNA ili ssRNA prekursor mRNA prije njenog ograničenja i / ili poliadenilacije; iii) kodira najmanje jedan protein; iv) kodira faktor transkripcije; v) kodira CD protein, što znači protein identificiran u grupi diferencijacijskog sustava; vi) kodira enzim; vii) kodira protein u super imunolobulinskoj obitelji; viii) kodira citokin ili hemokin; ix) kodira protein receptora stanične površine; x) kodira protein u staničnoj signalnoj stanici; xi) kodira antitijelo; xii) kodira receptor T stanica; xiii) kodira reporterski protein; xiv) sadrži jedan ili više modificiranih ribonukleozida odabranih iz skupine koja sadrži pseudouridin, 1-metilpseudouridin, 5-metiluridin, 2'-O-metiluridin i 2-tiouridin umjesto barem dijela odgovarajućih nemodificiranih uridinskih ribonukleozidosa i 5-metilcitidin umjesto barem dijela odgovarajućih nepromijenjenih citidin-ribonukleozida; xv) ima kapu od 5 ′; xvi) ima kapu sa strukturom Cap 1; xvii) ima poli A rep; xviii) ne sadrži modificirane ribonukleozidoze koji nisu ribonukleozidi koji sadrže 5 'strukturu poklopca, ako je prisutan poklopac od 5', uključujući 5 'predzadnji nukleozid kada ssRNA sintetizirana in vitro pokazuje strukturu kapice 1; xix) ima najmanje jedan heterologni niz izabran između: 5 'UTR sekvence, Kozak sekvence, IRES sekvence i 3' UTR sekvence; xx) ne kodira protein ili polipeptid, već umjesto toga sadrži najmanje jednu dugu nekodirajuću RNK (ncRNA); xxi) kodira protein; xxii) kodira funkcionalni protein; xxiii) kodira protein koji je prisutan na staničnoj membrani ili u njoj; xxiv) kodira protein imunog efektora; xxv) kodira proteinski komplement kralježnjaka/kičmenjaka xxvi) kodira protein koji sadrži receptor za signalni put.
4. Obrađeni sastav ribonukleinske kiseline prema bilo kojem od zahtjeva 1-3, naznačen time što navedeni sastav ribonukleinske kiseline ili ssRNA ili mRNA kodira najmanje jedan protein odabran iz skupine koja se sastoji od: proteina koji smanjuje ili suzbija urođeni imuni odgovor koji sadrži proizvodnju interferona (IFN). ili odgovora, poput mRNA koja kodira B18R protein ili E3L ili K3L protein vaccinia virusa, ili funkcionalni fragment ili varijantu bilo kojeg od njih.
5. Obrađeni sastav ribonukleinske kiseline u skladu s bilo kojim od patentnih zahtjeva 1-3, naznačen time što navedeni sastav ribonukleinske kiseline ili ssRNA ili mRNA kodira najmanje jedan protein odabran iz skupine koja se sastoji od: eritropoetin (EPO); enzim koji se može detektirati odabran između luciferaze krijesnica, Renilla luciferaze, bakterijske beta-galaktozidaze (lacZ) i_zelene fluorescentne proteine (GFP); faktor rasta ili citokin odabran iz skupine koja se sastoji od faktora rasta koji potiče iz trombocita (PDGF), vaskularnog endotelnog faktora rasta (VEGF), transformirajući faktor rasta-betal (TGF-beta1), faktora rasta sličnog inzulinu (IGF), alfa- melanocit-stimulirajući hormon (alfa-MSH); inzulinu sličan faktor rasta-I (IGF-I); IL-4; IL-13; i IL-10; inducibilna sintaza dušičnog oksida (iNOS); protein toplinskog šoka; i regulatora provođenja transmembranske cistične fibroze (CFTR), ili funkcionalni fragment ili varijantu bilo kojeg od njih; enzim s antioksidacijskom aktivnošću odabran između katalaze, fosfolipid hidroperoksida glutation peroksidaze, superoksid dismutaze-1 i superoksid dismutaze-2; Brutonova tirozin kinaza; adenozin deaminaza; i ekto-nukleozid-trifosfat-difosfidrolaze, ili funkcionalni fragment ili varijanta bilo kojeg od njih.
6. Obrađeni sastav ribonukleinske kiseline u skladu s bilo kojim od patentnih zahtjeva 1-3, naznačen time što navedeni sastav ribonukleinske kiseline sadrži ssRNA ili mRNA molekule koje kodiraju jedan ili više proteina odabranih iz bilo koje od skupina A) do F) ispod: A) MYOD; ili B) ASCL1, MYT1L, NEUROD1 i POU3F2 (AMNP) ili funkcionalni fragment ili varijanta bilo kojeg od njih; ili C) ETS2, MESP1, GATA4, HAND2, TBX5 i MEF2C, ili funkcionalni fragment ili varijanta bilo kojeg od njih; ili D) ASCL1, EN1, FOXA2, LMX1A, NURR1 i PITX3, ili funkcionalni fragment ili varijanta bilo kojeg od njih; ili E) HNF1α, HNF4α, FOXA1, FOXA2, FOXA3 i GATA4, ili funkcionalni fragment ili varijanta bilo kojeg od njih; ili F) OCT4, SOX2, KLF4, LIN28, NANOG i MYC protein odabran između c-MYC, c-MYC (T58A) i L-MYC, ili funkcionalnog fragmenta ili varijante bilo kojeg od prethodnih.
7. Obrađeni sastav ribonukleinske kiseline u skladu s bilo kojim od patentnih zahtjeva 1-3, naznačen time što navedeni sastav RNA ili ssRNA ili mRNA kodira najmanje jedan protein odabran iz skupine koja sadrži: CD1a; CD1b; CD1c; CD1d; CD1e; CD2; CD3d; CD3e; CD3g; CD4; CD5; CD6; CD7; CD8a; CD8b; CD9; CD10; CD11; CD 11; CD11c; CD11d; CDw12; CD14; CD16a; CD16b; CD18; CD 19; CD20; CD21; CD22; CD23; CD24; CD25; CD26; CD27; CD28; CD29; CD30; CD31; CD32; CD33; CD34; CD35; CD36; CD37; CD38; CD39; CD40; CD41; CD42a; CD42b; CD42c; CD42d; CD44; CD45; CD46; CD47; CD48; CD49a; CD49b; CD49c; CD49d; CD49e; CD49f; CD50; CD51; CD52; CD53; CD54; CD55; CD56; CD57; CD58; CD59; CD61; CD62E; CD62L; CD62P; CD63; CD64; CD66a; CD66b; CD66c; CD66d; CD66e; CD66f; CD68; CD69; CD70; CD71; CD72; CD74; CD79a; CD79b; CD80; CD81; CD82; CD83; CD84; CD85a; CD85c; CD85d; CD85e; CD85f; CD85g; CD85h; CD85i; CD85j; CD85k; CD86; CD87; CD88; CD89; CD90; CD91; CD92; CD93; CD94; CD95; CD96; CD97; CD98; CD99; CD100; CD101; CD102; CD103; CD104; CD 105; CD106; CD107a; CD107b; CD108; CD109; CD110; CD111; CD112; CD113; CD114; CD115; CD116; CD117; CD118; CD119; CD120a; CD120b; CD121a; CD121b; CD122; CD123; CD124; CD125; CD126; CD127; CD129; CD130; CD131; CD132; CD133; CD134; CD135; CD136; CD137; CD138; CD139; CD140a; CD140b; CD141; CD142; CD143; CD144; CD146; CD147; CD148; CD150; CD151; CD152; CD153; CD154; CD155; CD156a; CD156b; CD157; CD158a; CD158b1; CD158b2; CD158c; CD158d; CD158e; CD158f1; CD158g; CD158h; CD158i; CD158j; CD158k; CD158z; CD159a; CD159c; CD160; CD161; CD162; CD163; CD163b; CD164; CD165; CD166; CD167a; CD167b; CD168; CD169; CD170; CD171; CD172a; CD172b; CD172g; CD173; CD177; CD178; CD179a; CD179b; CD180; CD181; CD182; CD183; CD184; CD185; CD186; CD191; CD192; CD193; CD194; CD195; CD196; CD197; CDw198; CDw199; CD200; CD201; CD202b; CD203a; CD203c; CD204; CD205; CD206; CD207; CD208; CD209; CD210; CDw210b; CD212; CD213a1; CD213a2; CD214; CD215; CD217; CD218a; CD218b; CD220; CD221; CD222; CD223; CD224; CD225; CD227; CD228; CD229; CD230; CD231; CD232; CD233; CD234; CD235a; CD235b; CD236; CD238; CD239; CD240CE; CD240D; CD241; CD242; CD243; CD244; CD245; CD246; CD247; CD248; CD249; CD252; CD253; CD254; CD256; CD257; CD258; CD261; CD262; CD263; CD264; CD265; CD266; CD267; CD268; CD269; CD270; CD271; CD272; CD273; CD274; CD275; CD276; CD277; CD278; CD279; CD280; CD281; CD282; CD283; CD284; CD286; CD288; CD289; CD290; CD292; CDw293; CD294; CD295; CD296; CD297; CD298; CD299; CD300a; CD300b; CD300c; CD300d; CD300e; CD300f; CD300g; CD301; CD302; CD303; CD304; CD305; CD306; CD307a; CD307b; CD307c; CD307d; CD307e; CD309; CD312; CD314; CD315; CD316; CD317; CD318; CD319; CD320; CD321; CD322; CD324; CD325; CD326; CD327; CD328; CD329; CD331; CD332; CD333; CD334; CD335; CD336; CD337; CD338; CD339; CD340; CD344; CD349; CD350; CD351; CD352; CD353; CD354; CD355; CD357; CD358; CD360; CD361; CD362; i CD363, ili funkcionalni fragment ili varijanta bilo kojeg od prethodnih.
8. Obrađeni sastav ribonukleinske kiseline prema zahtjevu 1, naznačen time, da ima najmanje jedno od sljedećeg: i) spomenuta sol u navedenoj puferiranoj vodenoj otopini daje ionsku čvrstoću koja je najmanje visoka kao kalijev acetat u koncentraciji od oko 50-300 mM; ii) navedeno obrađivanje nadalje obuhvaća pročišćavanje ili čišćenje ssRNA ili mRNA u sastavu RNK taloženjem soli, PAGE, elektroforezom gela od agaroze, kromatografijom na spin kolonama ili HPLC, pri čemu se uklanjaju probavljene onečišćujuće molekule dsRNA; iii) navedeno obrađivanje nadalje uključuje pročišćavanje ili čišćenje ssRNA ili mRNA u tretiranom sastavu ribonukleinske kiseline koristeći najmanje jedan korak odabran iz skupine koja se sastoji u kontaktu otopine s jednom ili više deoksirikonukleaza, ekstrahiranjem preparata RNA s fenolom i / ili kloroform, taloženje alkohola, taloženje ssRNA s amonijevim acetatom i ispiranje RNA taloga s 70% -tnim etanolom, i ne uključuje upotrebu kromatografije na stupcu, uključujući gravitacijski protok, HPLC ili FPLC; iv) navedena ssRNA ili mRNA sadrži najmanje jedan modificirani ribonukleozid, izabran iz skupine koja sadrži pseudouridin, 1-metilpseudouridin, 5-metilcitidin, 5-metiluridin, 2'-O-metiluridin i 2-tiouridin umjesto najmanje dio odgovarajućeg nemodificiranog kanonskog ribonukleozida, koji smanjuje indukciju ili aktivaciju RNA senzora ili urođenog puta imunog odgovora u stanici; v) navedena ssRNA ili mRNA ne sadrži modificirane ribonukleozidoze, osim onih ribonukleozida koji sadrže 5 'nukleotidnu strukturu kape ako je prisutan 5' poklopac, uključujući 5 'predzadnjeg nukleozida kada in vitro sintetizirani ssRNA pokazuje kapkastu strukturu kapica, ako je prisutan.
9. Obrađeni sastav ribonukleinske kiseline u skladu s bilo kojim od patentnih zahtjeva 1-8, naznačen time što navedena ssRNA ili mRNA kodiraju terapeutski protein za upotrebu u nadomjesnoj enzimi.
10. Obrađeni sastav ribonukleinske kiseline prema bilo kojem od zahtjeva 1-8, naznačen time, da se upotrebljava u liječenju regenerativnom medicinom, reprogramiranjem stanica, terapijom na stanici, nadomjesnom enzimskom terapijom, presađivanjem ili popravkom stanica, tkiva i organa, inženjeringom tkiva ili organa ili imunoterapijom.
11. Učinkovita doza obrađenog sastava ribonukleinske kiseline prema bilo kojem od zahtjeva 1-8, naznačena time, da se upotrebljava za smanjenje ili uklanjanje simptoma ili bolesti kod čovjeka ili životinje.
12. Upotreba obrađenog sastava ribonukleinske kiseline prema bilo kojem od zahtjeva 1-8, u pripravi lijeka za liječenje regenerativnim lijekom, reprogramiranje stanica, terapiju na bazi stanica, nadomjesnu terapiju enzimom, transplantaciju stanica, tkiva i organa ili popravak, inženjering tkiva ili organa ili imunoterapija.
13. Uporaba učinkovite doze obrađenog sastava ribonukleinske kiseline prema bilo kojem od zahtjeva 1-8, naznačen time, da se koristi u pripremi lijeka za smanjenje ili uklanjanje simptoma ili bolesti kod čovjeka ili životinje.
14. Upotreba puferirane vodene otopine koja sadrži protein specifičan za dsRNA endoribonukleaza III, kationi magnezij u koncentraciji od oko 1-4 mM, te sol koja daje ionsku snagu ekvivalentnu barem oko 50 mM kalijevog acetata ili kalijevog glutamata za liječenje pripravak RNA koji sadrži ili mRNA ili ssRNA prekursor za spomenutu mRNA prije njenog zatvaranja i / ili poliadeniliranja tako da količina dsRNA koja je veća od oko 40 baznih parova nakon spomenutog tretmana bude manja od 0,01%, poželjno manja od 0,001 %, nadalje poželjno manje od 0,0002% mase RNA u navedenom obrađenom sastavu RNA.
15. Upotreba puferirane vodene otopine koja sadrži protein specifičan za dsRNA endoribonukleaza III, kationi magnezij u koncentraciji od oko 1-4 mM, te sol koja daje ionsku snagu ekvivalentnu barem oko 50 mM kalijevog acetata ili kalijevog glutamata za liječenje pripravak RNA koji sadrži ili mRNA ili ssRNA koja kodira funkcionalni protein za nadomjesnu enzimsku terapiju, pri čemu je količina dsRNA veća od oko 40 baznih parova nakon spomenutog liječenja manja od 0,01%, poželjno manja od 0,001%, nadalje poželjno manje više od 0,0002% mase RNA u navedenom obrađenom sastavu RNA.
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