HRP20221536T1 - Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina - Google Patents

Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina Download PDF

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Publication number
HRP20221536T1
HRP20221536T1 HRP20221536TT HRP20221536T HRP20221536T1 HR P20221536 T1 HRP20221536 T1 HR P20221536T1 HR P20221536T T HRP20221536T T HR P20221536TT HR P20221536 T HRP20221536 T HR P20221536T HR P20221536 T1 HRP20221536 T1 HR P20221536T1
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compound according
straight
carbon atoms
alkyl chain
delivery
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HRP20221536TT
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Steven M. Ansell
Xinyao Du
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Acuitas Therapeutics Inc.
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    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/24—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having more than one carboxyl group bound to the carbon skeleton, e.g. aspartic acid
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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    • A61K48/0041—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 polymeric
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    • 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
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    • C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C219/08—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having at least one of the hydroxy groups esterified by a carboxylic acid having the esterifying carboxyl group bound to an acyclic carbon atom of an acyclic unsaturated carbon skeleton
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    • C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C219/10—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having at least one of the hydroxy groups esterified by a carboxylic acid having the esterifying carboxyl group bound to an acyclic carbon atom of a carbon skeleton containing rings
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    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
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    • C07C229/16—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
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    • C07C235/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C235/06—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
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Claims (9)

1. Spoj koji ima sljedeću strukturu (II): [image] ili njegova farmaceutski prihvatljiva sol, tautomer ili stereoizomer, naznačen time što: R10 i R11 su svaki neovisno, ravan, zasićen alkil lanac koji sadrži od 12 do 16 atoma ugljika; i z ima srednju vrijednost koja se kreće od 30 do 60.
2. Spoj prema patentnom zahtjevu 1, naznačen time što je z 45.
3. Spoj prema patentnom zahtjevu 1, naznačen time što su R10 i R11, svaki neovisno, ravan, zasićen alkil lanac koji sadrži 12 atoma ugljika.
4. Spoj prema patentnom zahtjevu 1, naznačen time što su R10 i R11, svaki neovisno, ravan, zasićen alkil lanac koji sadrži 14 atoma ugljika.
5. Spoj prema patentnom zahtjevu 1, naznačen time što su R10 i R11, svaki neovisno, ravan, zasićen alkil lanac koji sadrži 16 atoma ugljika.
6. Spoj prema patentnom zahtjevu 1, naznačen time što je R10 ravan, zasićen alkil lanac koji sadrži 12 atoma ugljika i R11 je ravan, zasićen alkil lanac koji sadrži 14 atoma ugljika.
7. Spoj prema patentnom zahtjevu 1, koji ima sljedeću strukturu: [image] naznačen time što n ima srednju vrijednost koja se kreće od 40 do 50.
8. Spoj prema patentnom zahtjevu 1, koji ima sljedeću strukturu: [image] naznačen time što n ima srednju vrijednost koja se kreće od 40 do 50.
9. Pripravak koji sadrži spoj prema bilo kojem od patentnih zahtjeva 1-8 i kationski lipid.
HRP20221536TT 2014-06-25 2015-06-05 Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina HRP20221536T1 (hr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462016839P 2014-06-25 2014-06-25
EP20195502.8A EP3766916B1 (en) 2014-06-25 2015-06-05 Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids

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HRP20221536T1 true HRP20221536T1 (hr) 2023-02-17

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US (5) US9738593B2 (hr)
EP (3) EP4148083A1 (hr)
JP (4) JP6594421B2 (hr)
CN (2) CN106795096B (hr)
AU (4) AU2015280499B2 (hr)
CA (2) CA2953341C (hr)
ES (2) ES2834556T3 (hr)
HR (1) HRP20221536T1 (hr)
HU (1) HUE060907T2 (hr)
IL (5) IL298516A (hr)
LT (1) LT3766916T (hr)
PL (1) PL3766916T3 (hr)
PT (1) PT3766916T (hr)
RS (1) RS63848B1 (hr)
SI (1) SI3766916T1 (hr)
WO (1) WO2015199952A1 (hr)

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