HRP20220652T1 - Pripravci i postupci unosa terapijskih sredstava - Google Patents

Pripravci i postupci unosa terapijskih sredstava Download PDF

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Publication number
HRP20220652T1
HRP20220652T1 HRP20220652TT HRP20220652T HRP20220652T1 HR P20220652 T1 HRP20220652 T1 HR P20220652T1 HR P20220652T T HRP20220652T T HR P20220652TT HR P20220652 T HRP20220652 T HR P20220652T HR P20220652 T1 HRP20220652 T1 HR P20220652T1
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HR
Croatia
Prior art keywords
lnp
lipid
mir
dose
peg
Prior art date
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HRP20220652TT
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English (en)
Inventor
Gilles BESIN
Stephen Hoge
Joseph Senn
Kerry BENENATO
Staci SABNIS
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Modernatx, Inc.
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Publication of HRP20220652T1 publication Critical patent/HRP20220652T1/hr

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    • 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
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Claims (19)

1. Lipidna nanočestica (LNP) koja inkapsulira mRNA koja kodira terapijski protein, pri čemu LNP sadrži kationski lipid, pomoćni lipid, strukturni lipid, te stabilizator, pri čemu je kationski lipid lipid koji se može ionizirati, pri čemu je stabilizator PEG-ilirani lipid, te pri čemu je PEG-ilirani lipid spoj formule (V-OH): [image] ili njegova farmaceutski prihvatljiva sol, pri čemu: r je cijeli broj između, i uključujući, 1 i 100; i R5 je izborno supstituirani C10-40 alkil, izborno supstituirani C10-40 alkenil, ili izborno supstituirani C10-40 alkinil; i pri čemu LNP ne izaziva ubrzani klirens krvi.
2. LNP u skladu s patentnim zahtjevom 1, pri čemu lipid koji se može ionizirati ima formulu (XI): [image] ili njegova farmaceutski prihvatljiva sol, pri čemu: 1 se bira između 1, 2, 3, 4, te 5; M i M’ se neovisno bira između -C(O)O-, -OC(O)-, -C(O)N(R’)-, -P(O)(OR’)O-, -S-S-, arilne skupine, te heteroarilne skupine; M1 je veza ili M’; R2 i R3 se neovisno bira iz skupine koju čine H, C1-14 alkil, te C2-14 alkenil; R4 je nesupstituirani C1-3 alkil, ili -(CH2)nQ, u kojem se n bira između 2, 3 ili 4, a Q je OH, -NHC(S)N(R)2, -NHC(O)N(R)2, -N(R)C(O)R, -N(R)S(O)2R, -N(R)R8, -NHC(=NR9)N(R)2, -NHC(=CHR9)N(R)2, -OC(O)N(R)2, -N(R)C(O)OR, heteroaril ili heterocikloalkil; R’ se neovisno bira iz skupine koju čine C1-8 alkil, C2-18 alkenil, -R*YR”, -YR”, te H; R” se neovisno bira iz skupine koju čine C3-14 alkil i C3-14 alkenil; R* se neovisno bira iz skupine koju čine C1-12 alkil i C2-12 alkenil; i Y je neovisno C3-6 karbocikl.
3. LNP u skladu s patentnim zahtjevom 2, pri čemu spoj formule (XI) ima strukturu spoja 18: [image] ili njegova farmaceutski prihvatljiva sol.
4. LNP u skladu s bilo kojim od patentnih zahtjeva 1-3, pri čemu je spoj formule (V-OH): [image] ili njegova farmaceutski prihvatljiva sol.
5. LNP u skladu s bilo kojim od patentnih zahtjeva 1-4, pri čemu PEG-ilirani lipid sadrži molekulu PEG prosječne molekulske težine od: (i) 2000 Da; ili (ii) manje od 2000 Da, izborno oko 1500 Da, oko 1000 Da, ili oko 500 Da.
6. LNP u skladu s bilo kojim od patentnih zahtjeva 1-2, pri čemu je spoj formule (V-OH) HO-PEG2000-ester-C18.
7. LNP u skladu s bilo kojim od patentnih zahtjeva 1-6, pri čemu je spoj formule (V-OH): [image] ili njegova farmaceutski prihvatljiva sol.
8. LNP u skladu s bilo kojim od patentnih zahtjeva 1-7, pri čemu je pomoćni lipid nekationski pomoćni lipid, a strukturni lipid je kolesterol, te pri čemu LNP ima molarni omjer od otprilike 45-65 % kationskog lipida, otprilike 0,15-15 % PEG-iliranog lipida, otprilike 15-45 % kolesterola i otprilike 5-25 % nekationskog pomoćnog lipida.
9. LNP u skladu s patentnim zahtjevom 8, pri čemu LNP sadrži manje od 0,5 % (tež./tež.) PEG-iliranog lipida.
10. LNP u skladu s patentnim zahtjevom 8, pri čemu LNP sadrži manje od 0,25 % (tež./tež.) PEG-iliranog lipida.
11. LNP u skladu s bilo kojim od patentnih zahtjeva 1-7, pri čemu je pomoćni lipid: (i) nekationski pomoćni lipid koji sadrži najmanje jedan masnokiselinski lanac od najmanje 8C i najmanje jedan ostatak skupine polarne glave; (ii) dvojnoionski nekationski pomoćni lipid; (iii) lipid koji nije fosfatidilkolin (PC); ili (iv) 1,2-distearoil-sn-glicero-3-fosfokolin (DSPC).
12. LNP u skladu s bilo kojim od patentnih zahtjeva 1-7 ili 11, pri čemu pomoćni lipid: (i) kompetitivno inhibira vezanje fosfatidilkolina na CD36; ili (ii) se ne veže ili ima nisku aktivnost vezanja na CD36.
13. LNP u skladu s bilo kojim od patentnih zahtjeva 1-7, pri čemu strukturni lipid je sterol, npr. kolesterol.
14. LNP u skladu s bilo kojim od prethodnih patentnih zahtjeva, pri čemu mRNA sadrži (a) kemijski modificirane baze ili šećere, te modifikacije okosnice; i/ili (b) mjesto vezanja miR, pri čemu mjesto vezanja miR može: (i) se birati između miR 126, miR 155 i miR 142 3p; ili (ii) sadržavati miR-122 mjesto vezanja i također sadržavati jedan ili više mjesta vezanja miR koja se bira iz skupine koju čine miR-142, miR-146, miR-155, miR-126, miR-16, miR-21, miR-223, miR-24, miR-27.
15. LNP u skladu s bilo kojim od prethodnih patentnih zahtjeva, naznačen time što se LNP slabije veže na stanice B1a i/ili ima smanjenu aktivnost aktiviranja stanica B1a.
16. Lipidna nanočestica (LNP), namijenjena uporabi u postupku unosa terapijske razine proteina od interesa na subjektu, pri čemu LNP je LNP u skladu s bilo kojim od prethodnih patentnih zahtjeva, pri čemu se postupak sastoji u primjeni prve doze i druge doze LNP na subjektu; i pri čemu prva doza LNP ne izaziva imunološki odgovor koji pospješuje ubrzani klirens krvi (ABC) prilikom primjene druge doze LNP.
17. LNP, namijenjen uporabi u skladu s patentnim zahtjevom 16, pri čemu imunološki odgovor je anti-PEG IgM ili anti-PEG IgG odgovor.
18. LNP, namijenjen uporabi u skladu s bilo kojim od patentnih zahtjeva 16-17, pri čemu prva doza LNP ne aktivira signalni put ovisan o CD36 u imunosnoj stanici prilikom primjene druge doze LNP.
19. LNP, namijenjen uporabi u skladu s bilo kojim od patentnih zahtjeva 16-18, pri čemu interval između prve doze i druge doze je otprilike 21 dan ili manje, i/ili pri čemu se postupak dodatno sastoji u primjeni na subjektu dodatnog sredstva koje inhibira imunosne odgovore.
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EP3386484A1 (en) 2018-10-17
DK3386484T3 (da) 2022-07-04
JP2022031359A (ja) 2022-02-18
LT3386484T (lt) 2022-06-10
ES2924407T3 (es) 2022-10-06

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