Abstract: The invention relates to new 5?mRNA end cap analogs, RNA molecules containing them, their uses and methods for their in vitro synthesis, as well as a method for protein or peptide synthesis in vitro or in cell cultures, which method translates the RNA molecule.

Abstract: The invention relates to new mRNA 5? end cap analogs, RNA molecules containing them, their uses and methods for their in vitro synthesis, as well as a method for protein or peptide synthesis in vitro or in cell cultures, which method encompasses translation of the RNA molecule.

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.

Abstract: The object of the invention is a compound of formula (I), or a stereoisomer or salt thereof, wherein R1 and R2 are selected from the group consisting of N, N+—CH3, N+—C2H5, N+—C3H8, N+—C4H5, N+—CH2C6H5 wherein at least one of R1, R2 is not N. n and m are independently chosen from the group consisting of 0, 1 and 2; X is selected from the group consisting of O, NH, S, CH2 k is 1 or 2 Y is either void or selected from the group of —CH2—, —CH2CH2—, —CH2O—, —CH2S—, —CH2NH—, —CH2CH2O—, —CH2CH2NH—, —CH2CH2S— R3, R4, R5, R6 are selected from the group consisting of H, OH, OCH3, or OCH2CH3; wherein R3 and R4 may be the same or different; R5 and R6 may be the same or different; if either of R3, R4 is different than OH than R5 and R6 are both OH; if either of R5, R6 is different than OH than R3 and R4 are both OH.

Abstract: The object of the invention is a compound of formula (I), or a stereoisomer or salt thereof, wherein R1 and R2 are selected from the group consisting of N, N+—CH3, N+—C2H5, N+—C3H8, N+—C4H5, N+—CH2C6H5 wherein at least one of R1, R2 is not N. n and m are independently chosen from the group consisting of 0, 1 and 2; X is selected from the group consisting of O, NH, S, CH2 k is 1 or 2 Y is either void or selected from the group of —CH2—, —CH2CH2—, —CH2O—, —CH2S—, —CH2NH—, —CH2CH2O—, —CH2CH2NH—, —CH2CH2S— R3, R4, R5, R6 are selected from the group consisting of H, OH, OCH3, or OCH2CH3; wherein R3 and R4 may be the same or different; R5 and R6 may be the same or different; if either of R3, R4 is different than OH than R5 and R6 are both OH; if either of R5, R6 is different than OH than R3 and R4 are both OH.

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.

Abstract: The present invention relates to modification of RNA with 5?-cap analogs of Formula (1): wherein R1-R6 and n are as described herein, in order to improve the stability and increase the expression of said RNA, in particular in immature antigen presenting cells. The present invention provides a vaccine composition comprising said stabilized RNA, immature antigen presenting cells comprising said stabilized RNA, and methods for stimulating and/or activating immune effector cells and for inducing an immune response in an individual using said stabilized RNA.

Abstract: Dinucleotide cap analogs are disclosed, modified at different phosphate positions with a boranophosphate group or a phosphoroselenoate group. The analogs are useful as reagents in the preparation of capped mRNAs and have increased stability both in vitro and in vivo. They may be used as inhibitors of cap-dependent translation. Optionally, the boranophosphate or phosphoroselenoate group has a 2?-O or 3?-O-alkyl group, preferably a methyl group, producing analogs called BH3-ARCAs or Se-ARCAs. ARCAs may be modified with ?-, ?-, or ?-boranophosphate or phosphoroselenoate groups.

Abstract: The present invention relates to modification of RNA with 5?-cap analogs in order to improve the stability and increase the expression of said RNA, in particular in immature antigen presenting cells. The present invention provides a vaccine composition comprising said stabilized RNA, immature antigen presenting cells comprising said stabilized RNA, and methods for stimulating and/or activating immune effector cells and for inducing an immune response in an individual using said stabilized RNA.

Abstract: New RNA cap analogs are disclosed containing one or more phosphorothioates groups. The analogs also contain modifications at the 2?-O position of 7-methylguanosine that prevent them from being incorporated in the reverse orientation during in vitro synthesis of mRNA and that hence are “anti-reverse cap analogs” (ARCAs). The ARCA modification ensures that the S atom is precisely positioned within the active sites of cap-binding proteins in both the translational and decapping machinery. The new S-ARCA analogs are resistant to in vivo decapping enzymes. Some S-ARCAs have a higher affinity for eIF4E than the corresponding analogs not containing a phosphorothioate group. When mRNAs containing the various S-ARCAs are introduced into cultured cells, some are translated as much as five-fold more efficiently than mRNAs synthesized with the conventional analog m7GpppG.

Abstract: Dinucleotide cap analogs are disclosed, modified at different phosphate positions with a boranophosphate group or a phosphoroselenoate group. The analogs are useful as reagents in the preparation of capped mRNAs and have increased stability both in vitro and in vivo. They may be used as inhibitors of cap-dependent translation. Optionally, the boranophosphate or phosphoroselenoate group has a 2?-O or 3?-O-alkyl group, preferably a methyl group, producing analogs called BH3-ARCAs or Se-ARCAs. ARCAs may be modified with ?-, ?-, or ?-boranophosphate or phosphoroselenoate groups.

Abstract: New RNA cap analogs are disclosed containing one or more phosphorothioates groups. The analogs also contain modifications at the 2?-O position of 7-methylguanosine that prevent them from being incorporated in the reverse orientation during in vitro synthesis of mRNA and that hence are “anti-reverse cap analogs” (ARCAs). The ARCA modification ensures that the S atom is precisely positioned within the active sites of cap-binding proteins in both the translational and decapping machinery. The new S-ARCA analogs are resistant to in vivo decapping enzymes. Some S-ARCAs have a higher affinity for eIF4E than the corresponding analogs not containing a phosphorothioate group. When mRNAs containing the various S-ARCAs are introduced into cultured cells, some are translated as much as five-fold more efficiently than mRNAs synthesized with the conventional analog m7GpppG.