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1. WO2020069718 - RNA PARTICLES COMPRISING POLYSARCOSINE

Publication Number WO/2020/069718
Publication Date 09.04.2020
International Application No. PCT/EP2018/076633
International Filing Date 01.10.2018
IPC
A61K 47/34 2017.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
34Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 31/195 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
19Carboxylic acids, e.g. valproic acid
195having an amino group
C07K 1/06 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
1General processes for the preparation of peptides
06using protecting groups or activating agents
C08G 69/10 2006.01
CCHEMISTRY; METALLURGY
08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
69Macromolecular compounds obtained by reactions forming a carboxylic amide link in the main chain of the macromolecule
02Polyamides derived from amino carboxylic acids or from polyamines and polycarboxylic acids
08derived from amino carboxylic acids
10Alpha-amino-carboxylic acids
CPC
A61K 31/195
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic, hydroximic acids
19Carboxylic acids, e.g. valproic acid
195having an amino group
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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 9/5123
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules ; having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
51Nanocapsules; Nanoparticles;
5107Excipients; Inactive ingredients
5123Organic compounds, e.g. fats, sugars
C08G 69/10
CCHEMISTRY; METALLURGY
08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
69Macromolecular compounds obtained by reactions forming a carboxylic amide link in the main chain of the macromolecule
02Polyamides derived from amino-carboxylic acids or from polyamines and polycarboxylic acids
08derived from amino-carboxylic acids
10Alpha-amino-carboxylic acids
C08L 77/04
CCHEMISTRY; METALLURGY
08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
LCOMPOSITIONS OF MACROMOLECULAR COMPOUNDS
77Compositions of polyamides obtained by reactions forming a carboxylic amide link in the main chain
04Polyamides derived from alpha-amino carboxylic acids
Applicants
  • JOHANNES GUTENBERG-UNIVERSITÄT MAINZ [DE]/[DE]
  • BIONTECH RNA PHARMACEUTICALS GMBH [DE]/[DE]
Inventors
  • BARZ, Matthias
  • WEBER, Benjamin
  • HAAS, Heinrich
  • HELLER, Philipp
  • NOGUEIRA, Sarah
  • SCHLEGEL, Anne
Agents
  • SCHNAPPAUF, Georg
Priority Data
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) RNA PARTICLES COMPRISING POLYSARCOSINE
(FR) PARTICULES D'ARN COMPRENANT DE LA POLYSARCOSINE
Abstract
(EN)
The present disclosure relates to RNA particles for delivery of RNA to target tissues after administration, in particular after parenteral administration such as intravenous, intramuscular, subcutaneous or intratumoral administration, and compositions comprising such RNA particles. The RNA particles in one embodiment comprise single-stranded RNA such as mRNA which encodes a peptide or protein of interest, such as a pharmaceutically active peptide or protein. The RNA is taken up by cells of a target tissue and the RNA is translated into the encoded peptide or protein, which may exhibit its physiological activity.
(FR)
La présente invention concerne des particules d'ARN destinées à être transmises à des tissus cibles après administration, en particulier après administration parentérale telle qu'une administration intraveineuse, intramusculaire, sous-cutanée ou intratumorale, et des compositions comprenant de telles particules d'ARN. Dans un mode de réalisation, les particules d'ARN comprennent un ARN simple brin tel qu'un ARNm qui encode un peptide ou une protéine d'intérêt, tel qu'un peptide ou une protéine pharmaceutiquement actif. L'ARN est absorbé par les cellules du tissu cible et l'ARN est traduit en peptide ou protéine codée, qui peut présenter son activité physiologique.
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