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1. WO2021055956 - METHODS OF TREATMENT OF NEUROFIBROMATOSIS TYPE 1 (NF1) AND NF1-MEDIATED CONDITIONS AND COMPOSITIONS FOR USE IN SUCH METHODS

Publication Number WO/2021/055956
Publication Date 25.03.2021
International Application No. PCT/US2020/051827
International Filing Date 21.09.2020
IPC
A61K 31/7088 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
A61K 31/7105 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/711 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
C12N 15/113 2010.01
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
CPC
C12N 15/113
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
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
C12N 2310/11
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
10Type of nucleic acid
11Antisense
C12N 2310/20
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2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 2310/3145
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
30Chemical structure
31of the backbone
314Phosphoramidates
3145with the nitrogen in 3' or 5'-position
C12N 2310/3233
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2310Structure or type of the nucleic acid
30Chemical structure
32of the sugar
323modified ring structure
3233Morpholino-type ring
C12N 2320/33
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2320Applications; Uses
30Special therapeutic applications
33Alteration of splicing
Applicants
  • THE UAB RESEARCH FOUNDATION [US]/[US]
  • ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE, ROYAL HOLLOWAY UNIVERSITY OF LONDON [GB]/[GB]
Inventors
  • WALLIS, Deeann
  • KESTERSON, Robert
  • KORF, Bruce
  • LEIER, Andre
  • LAMBERT, Laura
  • POPPLEWELL, Linda
  • DICKSON, George
Agents
  • PETERSON, Thomas G.
Priority Data
62/903,52120.09.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS OF TREATMENT OF NEUROFIBROMATOSIS TYPE 1 (NF1) AND NF1-MEDIATED CONDITIONS AND COMPOSITIONS FOR USE IN SUCH METHODS
(FR) PROCÉDÉS DE TRAITEMENT DE LA NEUROFIBROMATOSE DE TYPE 1 (NF1) ET D'AFFECTIONS MÉDIÉES PAR NF1 ET COMPOSITIONS DESTINÉES À ÊTRE UTILISÉES DANS DE TELS PROCÉDÉS
Abstract
(EN)
The present disclosure provides methods and compositions for the treatment of NF-1 and NF-1 mediated conditions. The present disclosure further provides for methods of exon skipping and exon retention and compositions for use in such methods. Such methods of exon skipping and exon retention may be used in the methods of treatment discussed herein. The present disclosure further provides new therapeutic compounds, particularly oligonucleotides, including antisense oligonucleotides, for use in the methods described herein.
(FR)
La présente invention concerne des procédés et des compositions pour le traitement de la NF-1 et d'affections médiées par la NF-1. La présente invention concerne également des procédés de saut d'exon et de rétention d'exon, et des compositions destinées à être utilisées dans ces procédés. De tels procédés de saut d'exon et de rétention d'exon peuvent être utilisés dans les procédés de traitement décrits ici. La présente invention concerne en outre de nouveaux composés thérapeutiques, en particulier des oligonucléotides, notamment des oligonucléotides antisens, destinés à être utilisés dans les procédés de l'invention.
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