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1. WO2021247800 - TREATMENT OF NEUROLOGICAL DISEASES USING MODULATORS OF GENE TRANSCRIPTS

Publication Number WO/2021/247800
Publication Date 09.12.2021
International Application No. PCT/US2021/035603
International Filing Date 03.06.2021
IPC
C12N 15/113 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
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7115 2006.1
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
7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
A61K 31/7125 2006.1
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
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 31/712 2006.1
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
712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
Applicants
  • QURALIS CORPORATION [US]/[US]
Inventors
  • ELBAUM, Daniel
  • HINCKLEY, Sandra
Agents
  • ZHANG, Clark
  • BIEKER, Kristina
  • BRODOWSKI, Michael, H.
  • ASHRAF, Shovon
  • ARGENTIERI, Steven, R.
Priority Data
63/033,92603.06.2020US
63/119,71701.12.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) TREATMENT OF NEUROLOGICAL DISEASES USING MODULATORS OF GENE TRANSCRIPTS
(FR) TRAITEMENT DE MALADIES NEUROLOGIQUES À L'AIDE DE MODULATEURS DE TRANSCRITS DE GÈNES
Abstract
(EN) Disclosed herein are STMN2 oligonucleotides with one or more spacers. In various embodiments, STMN2 oligonucleotides with spacer(s) reduce STMN2 transcripts with cryptic exon and increase full length STMN2 transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).
(FR) L'invention concerne des oligonucléotides STMN2 comprenant au moins un espaceur. Dans divers modes de réalisation, les oligonucléotides STMN2 comprenant au moins un espaceur réduisent des transcrits STMN2 avec un exon cryptique et augmentent la longueur totale des transcrits STMN2, conférant ainsi une efficacité thérapeutique contre des maladies neurologiques, telles que la sclérose latérale amyotrophique (ALS), la démence frontotemporale (FTD) ou la maladie d'Alzheimer (AD).
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