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1. WO2022003362 - THERAPEUTIC NUCLEIC ACIDS, PEPTIDES AND USES II

Publication Number WO/2022/003362
Publication Date 06.01.2022
International Application No. PCT/GB2021/051678
International Filing Date 01.07.2021
IPC
C07K 14/005 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
C12N 15/861 2006.1
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
861Adenoviral vectors
CPC
A61K 38/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
A61K 48/005
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
A61P 25/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
C07K 14/4702
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
4701not used
4702Regulators; Modulating activity
C07K 2319/02
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
01containing a localisation/targetting motif
02containing a signal sequence
C07K 2319/10
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
01containing a localisation/targetting motif
10containing a tag for extracellular membrane crossing, e.g. TAT or VP22
Applicants
  • IMPERIAL COLLEGE INNOVATIONS LIMITED [GB]/[GB]
Inventors
  • ISALAN, Mark
  • MIELCAREK, Michal
Agents
  • MOORE, Michael
  • AHMAD, Sheikh Shakeel
  • BAKER, Philip
  • BEETZ, Joeri
  • CALDWELL, Judith
  • CALLAGHAN, Mark
  • CHRISTIE, Gemma
  • CREASE, Devanand
  • FOUNTAIN, Sullivan
  • GOODACRE, Jonathan
  • HOPLEY, Joanne
  • KEHOE, Laura
  • KENT, Peter
  • LAWRENCE, Richard
  • CUMMINGS, Sean
  • PATERSON, Colin
  • RICHARDSON, Mark
  • WALKER-SMITH, Samantha
  • WEAL, Emily
Priority Data
2010075.601.07.2020GB
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) THERAPEUTIC NUCLEIC ACIDS, PEPTIDES AND USES II
(FR) ACIDES NUCLÉIQUES, PEPTIDES ET UTILISATIONS II THÉRAPEUTIQUES
Abstract
(EN) Disclosed herein are polypeptides for use in treating diseases associated with pathogenic genomic repeat sequences, such as neurological disorders. Also disclosed are nucleic acid molecules and vectors that encode such polypeptides. Therapeutic uses and methods for treating such diseases are also disclosed; in particular, therapeutic uses and methods comprising complementary pairs and combinations of therapeutic polypeptides, nucleic acids or vectors. Also disclosed is a method and associated peptides and nucleic acids for active, long-term delivery of therapeutic molecules to target cells in vivo or in vitro.
(FR) La divulgation concerne des polypeptides destinés à être utilisés dans le traitement de maladies associées à des séquences de répétition génomique pathogènes, telles que des troubles neurologiques. La divulgation concerne également des molécules d'acide nucléique et des vecteurs codant de tels polypeptides. La divulgation concerne en outre des utilisations thérapeutiques et des méthodes de traitement de telles maladies ; en particulier, des utilisations thérapeutiques et des méthodes comprenant des paires complémentaires et des combinaisons de polypeptides thérapeutiques, d'acides nucléiques ou de vecteurs. La divulgation concerne enfin une méthode ainsi que des peptides et des acides nucléiques associés pour une administration active et à long terme de molécules thérapeutiques dans des cellules cibles in vivo ou in vitro.
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