MD3386484T2 - Compoziții și metode de livrare a unor agenți terapeutici - Google Patents

Compoziții și metode de livrare a unor agenți terapeutici Download PDF

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Publication number
MD3386484T2
MD3386484T2 MDE20180986T MDE20180986T MD3386484T2 MD 3386484 T2 MD3386484 T2 MD 3386484T2 MD E20180986 T MDE20180986 T MD E20180986T MD E20180986 T MDE20180986 T MD E20180986T MD 3386484 T2 MD3386484 T2 MD 3386484T2
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MD
Moldova
Prior art keywords
lipid
lnp
peg
group
cells
Prior art date
Application number
MDE20180986T
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English (en)
Romanian (ro)
Inventor
Gilles Besin
Stephen Hoge
Joseph Senn
Kerry Benenato
Staci Sabnis
Original Assignee
Modernatx Inc
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Publication date
Application filed by Modernatx Inc filed Critical Modernatx Inc
Publication of MD3386484T2 publication Critical patent/MD3386484T2/ro

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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