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1. WO2020117705 - METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MLH1 ACTIVITY

Publication Number WO/2020/117705
Publication Date 11.06.2020
International Application No. PCT/US2019/064058
International Filing Date 02.12.2019
IPC
A61K 31/713 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
713Double-stranded nucleic acids or oligonucleotides
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 35/28 2015.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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
A61P 37/02 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
37Drugs for immunological or allergic disorders
02Immunomodulators
C12N 5/079 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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07Animal cells or tissues
071Vertebrate cells or tissues, e.g. human cells or tissues
079Neural cells
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
62/774,77103.12.2018US
62/778,68612.12.2018US
62/876,98722.07.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MLH1 ACTIVITY
(FR) MÉTHODES POUR LE TRAITEMENT DE TROUBLES D'EXPANSION DE RÉPÉTITIONS TRINUCLÉOTIDIQUES ASSOCIÉS À UNE ACTIVITÉ MLH1
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 MLH1 activity.
(FR)
La présente invention concerne des compositions et des méthodes utiles pour traiter des troubles d'expansion de répétitions, par exemple, chez un sujet en ayant besoin. Dans certains aspects, les compositions et les méthodes décrites dans la description sont utiles dans le traitement de troubles associés à l'activité MLH1.
Latest bibliographic data on file with the International Bureau