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1. WO2021030218 - LIPIDATED CATIONIC PEPTIDE-PEG COMPOSITIONS FOR NUCLEIC ACID DELIVERY

Publication Number WO/2021/030218
Publication Date 18.02.2021
International Application No. PCT/US2020/045508
International Filing Date 07.08.2020
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 47/42 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
42Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
A61K 47/69 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
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
69the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
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
C07K 7/06 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
7Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
04Linear peptides containing only normal peptide links
06having 5 to 11 amino acids
C07K 7/08 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
7Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
04Linear peptides containing only normal peptide links
08having 12 to 20 amino acids
CPC
A61K 47/543
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
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
543Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
A61K 48/0041
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
0008characterised 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
0025wherein the non-active part clearly interacts with the delivered nucleic acid
0041the non-active part being polymeric
C07K 7/06
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
7Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
04Linear peptides containing only normal peptide links
06having 5 to 11 amino acids
C12N 15/88
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
88using microencapsulation, e.g. using ; amphiphile; liposome vesicle
Applicants
  • NUTCRACKER THERAPEUTICS, INC. [US]/[US]
Inventors
  • MCKINLAY, Colin James
Agents
  • CHOING, Stephanie
Priority Data
62/885,02209.08.2019US
62/907,47027.09.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) LIPIDATED CATIONIC PEPTIDE-PEG COMPOSITIONS FOR NUCLEIC ACID DELIVERY
(FR) COMPOSITIONS DE PEPTIDE-PEG CATIONIQUE LIPIDÉ DESTINÉES À L'ADMINISTRATION D'ACIDES NUCLÉIQUES
Abstract
(EN)
The present disclosure relates to complexes and compositions of cationic compounds combined with low mass percentages of PEGylated compounds, such as PEGylated lipids, for the delivery of nucleic acids and other polyanionic cargoes to cells, methods for preparing complexes and compositions comprising one or more cationic compounds and a low mass percentage of PEGylated compounds with polyanionic compounds, and methods for delivering the polyanionic compounds to cells.
(FR)
La présente invention concerne des complexes et des compositions de composés cationiques combinés à de faibles pourcentages en masse de composés pégylés, tels que des lipides pégylés, pour l'administration d'acides nucléiques et d'autres cargaisons polyanioniques à des cellules, des procédés de préparation de complexes et de compositions comprenant un ou plusieurs composés cationiques et un faible pourcentage en masse de composés pégylés avec des composés polyanioniques, et des procédés d'administration des composés polyanioniques à des cellules.
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