WO2024256870 - SMALL MOLECULE LIGANDS AND APTAMERS

National phase entry:
Publication Number WO/2024/256870
Publication Date 19.12.2024
International Application No. PCT/IB2024/000314
International Filing Date 14.06.2024
Title **
[English] SMALL MOLECULE LIGANDS AND APTAMERS
[French] LIGANDS ET APTAMÈRES À PETITES MOLÉCULES
Applicants **
MEIRAGTX GENE REGULATION LIMITED 92 Britannia Walk London N1 7NQ, GB
Inventors
LIU, Kevin, G. c/o MeiraGTx 450 East 29th Street 14th Street New York, NY 10016, US
KIM, Ji-In c/o MeiraGTx 450 East 29th Street 14th Street New York, NY 10016, US
GUO, Xuecui c/o MeiraGTx 450 East 29th Street 14th Street New York, NY 10016, US
FORBES, Alexandria c/o MeiraGTx 450 East 29th Street 14th Street New York, NY 10016, US
Priority Data
63/508,204   14.06.2023   US
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Quotation for National Phase entry

Country StagesTotal
China Filing2351
EPO Filing, Examination8031
Japan Filing593
South Korea Filing575
USA Filing, Examination4060
MasterCard Visa

Total: 15610

The term for entry into the National Phase has expired. This quotation is for informational purposes only

Abstract[English] The present disclosure provides small molecules of Formula (I) that bind to aptamers. Also contemplated are riboswitches and polynucleotide cassettes for regulating the expression of a target gene in response to the small molecule, wherein the polynucleotide cassettes comprise the aptamers disclosed herein. The small molecules disclosed herein that bind to the aptamers disclosed herein are modulators of target gene expression where the target gene contains a riboswitch comprising an aptamer described herein.[French] La présente invention concerne de petites molécules de formule (I) qui se lient à des aptamères. L'invention concerne également des ribocommutateurs et des cassettes polynucléotidiques pour réguler l'expression d'un gène cible en réponse à la petite molécule, les cassettes polynucléotidiques comprenant les aptamères selon l'invention. Les petites molécules divulguées qui se lient aux aptamères selon l'invention sont des modulateurs de l'expression génique cible où le gène cible contient un riborégulateur comprenant un aptamère selon l'invention.
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