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1. WO2013120629 - NUCLEIC ACID COMPRISING OR CODING FOR A HISTONE STEM-LOOP AND A POLY(A) SEQUENCE OR A POLYADENYLATION SIGNAL FOR INCREASING THE EXPRESSION OF AN ENCODED THERAPEUTIC PROTEIN

Publication Number WO/2013/120629
Publication Date 22.08.2013
International Application No. PCT/EP2013/000461
International Filing Date 15.02.2013
IPC
C12N 15/68 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
67General methods for enhancing the expression
68Stabilisation of the vector
A61K 39/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
A61K 39/35 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
35Allergens
C12N 15/117 2010.1
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
11DNA or RNA fragments; Modified forms thereof
117Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
CPC
A61K 39/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
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 48/0066
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
0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
A61P 11/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
A61P 3/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
A61P 31/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
Applicants
  • CUREVAC GMBH [DE]/[DE]
  • THEβ, Andreas [DE]/[DE] (US)
  • SCHLAKE, Thomas [DE]/[DE] (US)
  • PROBST, Jochen [DE]/[DE] (US)
Inventors
  • THEβ, Andreas
  • SCHLAKE, Thomas
  • PROBST, Jochen
Agents
  • GRAF VON STOSCH, Andreas
Priority Data
PCT/EP2012/00067115.02.2012EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) NUCLEIC ACID COMPRISING OR CODING FOR A HISTONE STEM-LOOP AND A POLY(A) SEQUENCE OR A POLYADENYLATION SIGNAL FOR INCREASING THE EXPRESSION OF AN ENCODED THERAPEUTIC PROTEIN
(FR) ACIDE NUCLÉIQUE COMPRENANT OU CODANT POUR UNE TIGE-BOUCLE D'HISTONE ET UNE SÉQUENCE POLY(A) OU UN SIGNAL DE POLYADÉNYLATION POUR AUGMENTER L'EXPRESSION D'UNE PROTÉINE THÉRAPEUTIQUE CODÉE
Abstract
(EN)
The present invention relates to a nucleic acid sequence, comprising or coding for a coding region, encoding at least one peptide or protein comprising a therapeutic protein or a fragment, variant or derivative thereof, at least one histone stem-loop and a poly(A) sequence or a polyadenylation signal. Furthermore the present invention provides the use of the nucleic acid for increasing the expression of said encoded peptide or protein, particularly for the use in gene therapy. It also discloses its use for the preparation of a pharmaceutical composition, e.g. for use in gene therapy, particularly in the treatment of diseases which are in need of a treatment with a therapeutic peptide or protein, preferably as defined herein. The present invention further describes a method for increasing the expression of a peptide or protein comprising a therapeutic protein or a fragment, variant or derivative thereof, using the nucleic acid comprising or coding for a histone stem-loop and a poly(A) sequence or a polyadenylation signal.
(FR)
La présente invention concerne une séquence d'acide nucléique, comprenant ou codant pour une région codante, codant pour au moins un peptide ou au moins une protéine, comprenant une protéine thérapeutique ou un fragment, variant ou dérivé de celle-ci, au moins une tige-boucle d'histone et une séquence poly(A) ou un signal de polyadénylation. En outre, la présente invention concerne l'utilisation de l'acide nucléique pour augmenter l'expression dudit peptide codé ou de ladite protéine codée, en particulier pour l'utilisation en thérapie génique. L'invention concerne également son utilisation pour la préparation d'une composition pharmaceutique, par exemple pour l'utilisation en thérapie génique, en particulier dans le traitement de maladies qui ont besoin d'un traitement par un peptide thérapeutique ou une protéine thérapeutique, de préférence tel que défini présentement. La présente invention concerne en outre un procédé pour augmenter l'expression d'un peptide ou d'une protéine, comprenant une protéine thérapeutique ou un fragment, variant ou dérivé de celle-ci, à l'aide de l'acide nucléique comprenant ou codant pour une tige-boucle d'histone et une séquence poly(A) ou un signal de polyadénylation.
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