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1. WO2014089239 - FORMULATIONS DE PARTICULES LIPIDIQUES D'ACIDE NUCLÉIQUE AMÉLIORÉES

Numéro de publication WO/2014/089239
Date de publication 12.06.2014
N° de la demande internationale PCT/US2013/073181
Date du dépôt international 04.12.2013
CIB
A61K 9/127 2006.01
ANÉCESSITÉS COURANTES DE LA VIE
61SCIENCES MÉDICALE OU VÉTÉRINAIRE; HYGIÈNE
KPRÉPARATIONS À USAGE MÉDICAL, DENTAIRE OU POUR LA TOILETTE
9Préparations médicinales caractérisées par un aspect particulier
10Dispersions; Emulsions
127Liposomes
A61K 48/00 2006.01
ANÉCESSITÉS COURANTES DE LA VIE
61SCIENCES MÉDICALE OU VÉTÉRINAIRE; HYGIÈNE
KPRÉPARATIONS À USAGE MÉDICAL, DENTAIRE OU POUR LA TOILETTE
48Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
CPC
A61K 31/713
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
A61K 9/0019
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
0012Galenical forms characterised by the site of application
0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
A61K 9/1272
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
1272with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
A61K 9/1617
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
14Particulate form, e.g. powders, ; Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
16Agglomerates; Granulates; Microbeadlets ; ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
1605Excipients; Inactive ingredients
1617Organic compounds, e.g. phospholipids, fats
A61K 9/1641
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
14Particulate form, e.g. powders, ; Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
16Agglomerates; Granulates; Microbeadlets ; ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
1605Excipients; Inactive ingredients
1629Organic macromolecular compounds
1641obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poloxamers
C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
Déposants
  • ALNYLAM PHARMACEUTICALS, INC. [US]/[US]
Inventeurs
  • AKINC, Akin
  • MAIER, Martin
  • KUMAR, Varun
Mandataires
  • LESSLER, Jay, P.
Données relatives à la priorité
61/734,73607.12.2012US
Langue de publication anglais (EN)
Langue de dépôt anglais (EN)
États désignés
Titre
(EN) IMPROVED NUCLEIC ACID LIPID PARTICLE FORMULATIONS
(FR) FORMULATIONS DE PARTICULES LIPIDIQUES D'ACIDE NUCLÉIQUE AMÉLIORÉES
Abrégé
(EN)
The present invention relates to lipid nanoparticles containing a biodegradable cationic lipid which provide improved delivery of active pharmaceutical ingredients, such as siRNA. In addition to the biodegradable cationic lipid the nanoparticles comprise a non-cationic lipid, polyethylne glycol dipalmitoylglycerol (PEG-DPG) and optionally a sterol.
(FR)
La présente invention concerne des nanoparticules lipidiques contenant un lipide cationique biodégradable qui permet une administration améliorée d'ingrédients pharmaceutiques actifs, comme un siARN. Outre le lipide cationique biodégradable, les nanoparticules comprennent un lipide non cationique, du polyéthylène glycol dipalmitoylglycérol (PEG-DPG) et éventuellement un stérol.
Également publié en tant que
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