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1. WO2020117706 - METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MLH3 ACTIVITY

Publication Number WO/2020/117706
Publication Date 11.06.2020
International Application No. PCT/US2019/064059
International Filing Date 02.12.2019
IPC
C12N 15/113 2010.01
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C07H 21/04 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
04with deoxyribosyl as saccharide radical
A61K 48/00 2006.01
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 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 31/7125 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
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61P 25/28 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
28for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
Applicants
  • TRIPLET THERAPEUTICS, INC. [US]/[US]
Inventors
  • BERMINGHAM, Nessan Anthony
  • BETTENCOURT, Brian R.
  • BIALEK, Peter Edward
Agents
  • ROSE GILLENTINE, Marsha
  • RYGIEL, Mark W.
  • BODENSTEIN, Matthew S.
  • BEZOS, Salvador M.
  • CALVO, Paul A.
  • CAMARCE, Christian
  • CIMBALA, Michele A.
  • COLLER III, Richard D.
  • CONKLIN, Kyle
  • DURKIN, Tracy-Gene G.
  • EISENBERG, Jason D.
  • ELLISON, Eldora L.
  • ESMOND, Robert W.
  • FEATHERSTONE, Donald J.
  • FRUEAUF, Jeremiah B.
  • GOLDSTEIN, Jorge A.
  • HELVEY, Jeffrey T.
  • HICKS, Ross
  • HOLOUBEK, Michelle K.
  • JACKMAN, Peter A.
  • KIM, Ji-Eun
  • LEE, Michael Q.
  • LONGSWORTH, Gaby L.
  • MILLONIG, Robert C.
  • MUTSCHELKNAUS, Joseph
  • NANNENGA-COMBS, Bonnie
  • PICKARD, Byron L.
  • RAY, Michael B.
  • COVERT, John
  • SHEA, JR., Timothy J.
  • SOKOHL, Robert E.
  • SPECHT, Michael D.
  • STEFFE, Eric K.
  • STERLING, Deborah A.
  • STERNE, Robert Greene
  • TUMINARO, Jonathan
  • WRIGHT, Jon E.
  • BANOWIT, Donald R.
  • YONAN, Daniel E.
  • DESAI, Nirav N.
  • POWERS III, R. Wilson
  • GAJEWSKI, Daniel
  • FITZSIMMONS, Jason A.
  • KENTON, JR., Lestin L.
  • BLOCK, Daniel S.
  • RICHARDSON, Ryan C.
  • BEMBEN, Richard M.
Priority Data
62/774,77703.12.2018US
62/877,10422.07.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MLH3 ACTIVITY
(FR) MÉTHODES THÉRAPEUTIQUES POUR LES MALADIES PAR EXPANSION DE RÉPÉTITIONS TRINUCLÉOTIDIQUES ASSOCIÉS À UNE ACTIVITÉ MLH3
Abstract
(EN)
The present disclosure features useful compositions and methods to treat repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with MLH3 activity.
(FR)
La présente invention concerne des compositions et des méthodes utiles pour traiter les maladies par expansion de répétitions trinucléotidiques chez un sujet en ayant besoin. Dans certains modes de réalisation, les méthodes décrites dans la description sont utiles pour traiter les maladies associés à une activité MLH3.
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