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1. WO2021058492 - NANOPARTICLE COMPOSITIONS FOR GENE THERAPY

Publication Number WO/2021/058492
Publication Date 01.04.2021
International Application No. PCT/EP2020/076449
International Filing Date 22.09.2020
IPC
A61K 38/43 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
C12N 15/88 2006.01
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 liposome vesicle
CPC
A61K 9/5138
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
513Organic macromolecular compounds; Dendrimers
5138obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyvinyl pyrrolidone, poly(meth)acrylates
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
C12N 15/90
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
90Stable introduction of foreign DNA into chromosome
Applicants
  • UNIVERSITY COLLEGE DUBLIN [IE]/[IE]
Inventors
  • WANG, Wenxin
  • A, Sigen
  • SAEZ, Irene, Lara
  • XU, Qian
  • O’KEEFFE AHERN, Jonathan
Agents
  • PURDY, Hugh, Barry
Priority Data
19199697.425.09.2019EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) NANOPARTICLE COMPOSITIONS FOR GENE THERAPY
(FR) COMPOSITIONS DE NANOPARTICULES POUR LA THÉRAPIE GÉNIQUE
Abstract
(EN)
A nanoparticulate composition comprises a gene editing ribonucleoprotein system complexed within a cationic polymer. The cationic polymer may be a Poly-beta amino ester hyperbranched polymer, especially a 4-branching hyperbranched polymer. The gene editing ribonucleoprotein system may be a CRISPR-Cas9 gene editing system configured to excise a mutation or exon in a gene, replace a mutation in a gene, or produce a knock-down or knock-out of a gene, and in particular configured to excise exon 80 of the COL7A1 gene which codes for the collagen VII protein. Data shows that using
(FR)
L'invention concerne une composition nanoparticulaire comprenant un système de ribonucléoprotéine d'édition de gène complexé à l'intérieur d'un polymère cationique. Le polymère cationique peut être un polymère hyperramifié à base de poly-bêta-aminoester, en particulier un polymère hyperramifié à 4 branches. Le système de ribonucléoprotéine d'édition de gène peut être un système d'édition de gène CRISPR-Cas9 configuré pour exciser une mutation ou un exon dans un gène, remplacer une mutation dans un gène ou produire un knock-down ou un knock-out d'un gène, et en particulier configuré pour exciser l'exon 80 du gène COL7A1 qui code pour la protéine de collagène VII.
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