The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Jul. 20, 2021
Filed:
Jan. 27, 2017
Uniwersytet Warszawski, Warsaw, PL;
Jacek Jemielity, Warsaw, PL;
Kaja Fac-Da̧browska, Warsaw, PL;
Blażej Wojtczak, Pabianice, PL;
Marek Baranowski, Warsaw, PL;
Anna Nowicka, Legionowo, PL;
Joanna Kowalska, Warsaw, PL;
Paweł Sikorski, Warsaw, PL;
Marcin Warmiński, Warsaw, PL;
UNIWERSYTET WARSZAWSKI, Warsaw, PL;
Abstract
The present invention relates to nucleotides, analogs of mRNA 5'-end (cap) containing sulfur atom at the position 5′ of 7-methylguanosine nucleoside. The disclosed compounds are recognized (bound and non-hydrolyzed) by DcpS enzyme (Decapping Scavenger), and thus may find therapeutic use as inhibitors thereof. DcpS is cap-specific enzyme with pyrophosphatase activity, which was identified as a therapeutic target in the treatment of spinal muscular atrophy (SMA). Some of the compounds disclosed have additional modifications in the phosphate chain, which modulate their affinity for DcpS enzyme. The present invention also relates to mRNAs modified at the 5′ end with mRNA 5′-end (cap) analogs containing 5′-phosphorothiolate moiety, which mRNAs have an increased stability and translational activity in cellular conditions, to a method of their preparation, their uses, and to a pharmaceutical formulation containing them, wherein Land Lare independently selected from the group comprising O and S, wherein at least one of Land Lis not O.