Abstract: Oligonucleotides comprising modifications at the 2? and/or 3? positions(s) along with methods of making and use against Alzheimer disease and other tauopathies are disclosed.

Abstract: The disclosure includes antisense oligonucleotides (ASOs), including gapmer ASOs, and methods of making and using the same.

Abstract: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2?-O-methyl nucleotides and 2?-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5?-stabilized end cap; and (b) a short interfering nucleic acid (siNA).

Abstract: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2?-O-methyl nucleotides and 2?-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5?-stabilized end cap; and (b) a short interfering nucleic acid (siNA).

Abstract: Provided herein are novel synthetic routes to amines through an oxime intermediate, e.g., 3?-N nucleosides and novel and intermediate compounds produced during these synthetic procedures.

Abstract: Described are short interfering nucleic acid (siNA) molecules comprising modified nucleotides, compositions, and methods and uses thereof.

Abstract: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2?-O-methyl nucleotides and 2?-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5?-stabilized end cap; and (b) a short interfering nucleic acid (siNA).

Abstract: The present invention is in the field of pharmaceutical compounds and preparations and method of their use in the treatment of disease. Described are short interfering nucleic acid (siNA) molecules comprising modified nucleotides, compositions containing the same, and uses thereof for treating or preventing coronavirus infections. In particular, the present invention is in the field of siNA molecules effective against a broad spectrum of coronaviruses, and especially the ?-coronaviruses, including SARS-CoV-2, the causative agent of COVID-19.

Abstract: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2?-O-methyl nucleotides and 2?-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5?-stabilized end cap; and (b) a short interfering nucleic acid (siNA).

Abstract: The disclosure includes antisense oligonucleotides, including gapmer antisense oligonucleotides, along with methods of making and use, e.g., against HBV.

Abstract: Oligonucleotides comprising modifications at the 2? and/or 3? positions(s) along with methods of making and use against Alzheimer disease and other tauopathies are disclosed.

Abstract: Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2?-O-methyl nucleotides and 2?-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5?-stabilized end cap; and (b) a short interfering nucleic acid (siNA).

Abstract: Disclosed herein is an oligonucleotide conjugate or complex thereof, comprising a modified oligonucleotide having sequence independent antiviral activity against hepatitis B, a liver targeting agent, and a linker connecting the liver targeting agent to a 3? end and/or a 5? end of the modified oligonucleotide or complex thereof. The oligonucleotide conjugate may be incorporated into a pharmaceutical composition for treating a liver disease or disorder such as hepatitis B.

Abstract: Various embodiments provide STOPS™ polymers that are S-antigen transport inhibiting oligonucleotide polymers, processes for making them and methods of using them to treat diseases and conditions. In some embodiments the STOPS™ modified oligonucleotides include an at least partially phosphorothioated sequence of alternating A and C units having modifications as described herein. The sequence independent antiviral activity against hepatitis B of embodiments of STOPS™ modified oligonucleotides, as determined by HBsAg Secretion Assay, is greater than that of a reference compound.

Abstract: A method of making a compound represented by Formula (I): includes a reducing step, a protecting step, and a phosphitylating step, where X is NH; Y is OCF3, —O(CR42)aCR43, —O(CR42)bOCR43, or —O(CR42)b—CR4?CR42; Z is H; B is adenine (A), guanine (G), thymine (T), cytosine (C), uracil (U), 5-methylcytosine (5-me-C), or a protected version of A, G, T, C, U, or 5-me-C; each R1 is independently C1-6 alkyl or cycloalkyl; R2 is CH2CH2CN or C1-6 alkyl; or R1 and R2 together form an optionally substituted C1-6 cycloalkyl; R3 is H or PG; PG is a protecting group; R4 is independently in each instance H or F; a is 1 or 2; and b is 1, 2, or 3.

Abstract: The present disclosure relates to compounds and compositions containing 5?-phosphoramidite nucleoside monomers of formulae (I) and (II), and methods of making and use, wherein the substituents are as defined in the appended claims.

Abstract: Various embodiments provide STOPS™ polymers that are S-antigen transport inhibiting oligonucleotide polymers, processes for making them and methods of using them to treat diseases and conditions. In some embodiments the STOPS™ modified oligonucleotides include an at least partially phosphorothioated sequence of alternating A and C units having modifications as described herein. The sequence independent antiviral activity against hepatitis B of embodiments of STOPS™ modified oligonucleotides, as determined by HBsAg Secretion Assay, is greater than that of a reference compound.

Abstract: The present disclosure relates to the field of pharmaceutical compounds and preparations and method of their use in the treatment of disease. Described are antisense oligonucleotide (ASO) molecules, compositions containing the same, and uses thereof for treating or preventing coronavirus infections. In particular, the present disclosure provides specific ASOs that are effective against a broad spectrum of coronaviruses, and especially the ?-coronaviruses, including SARS-CoV-2, the causative agent of COVID-19.

Abstract: Various embodiments provide STOPS™ polymers that are S-antigen transport inhibiting oligonucleotide polymers, processes for making them and methods of using them to treat diseases and conditions. In some embodiments the STOPS™ modified oligonucleotides include an at least partially phosphorothioated sequence of alternating A and C units having modifications as described herein. The sequence independent antiviral activity against hepatitis B of embodiments of STOPS™ modified oligonucleotides, as determined by HBsAg Secretion Assay, is an EC50 that is less than 100 nM.

Abstract: Oligonucleotides comprising modifications at the 2 and/or 3? positions(s) along with methods of making and use against Alzheimer disease and other tauopathies are disclosed.