Alios Biopharma, Inc.

Aligos Therapeutics, Inc.

Janssen Biopharma, Inc.

Janssen Pharmaceutica Nv

Vivek Kumar Rajwanshi

Leonid Beigelman

Jin Hong

Rajendra K Pandey

David Bernard Smith

Sergei M Gryaznov

Guangyi Wang

Jerome Deval

Natalia Dyatkina

Markus Hossbach

Saul Martinez Montero

Laxman Eltepu

N Tilani S De Costa

Yi Jin

Antitsa Dimitrova Stoycheva

Lakshmipathi Pandarinathan

Bharat Baral

Lawrence M Blatt

Andreas Ebneth

Constantin Van Outryve D'Ydewalle

Saúl Martinez Montero

Constantin Van Outryve D'YDEWALLE

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

Modified short interfering nucleic acid (siNA) molecules and uses thereof

The present disclosure relates to compounds and compositions containing 5'-phosphoramidite nucleoside monomers, and methods of making and use.

Modified nucleoside phosphoramidites

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).

Modified oligonucleotides comprising modifications at the 2' and/or 3′ position(s) along with methods of making and use, e.g., against HBV are disclosed.

Modified oligonucleotides and methods of use

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 OCF, —O(CR)CR, —O(CR)OCR, or —O(CR)—CR═CR; 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 Ris independently Calkyl or cycloalkyl; Ris CHCHCN or Calkyl; or Rand Rtogether form an optionally substituted Ccycloalkyl; Ris H or PG; PG is a protecting group; Ris independently in each instance H or F; a is 1 or 2; and b is 1, 2, or 3.

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&#x2032;-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5&#x2032;-stabilized end cap; and (b) a short interfering nucleic acid (siNA).

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

Modified gapmer oligonucleotides and methods of use

Oligonucleotides comprising modifications at the 2' and/or 3&#x2032; positions(s) along with methods of making and use against Alzheimer disease and other tauopathies are disclosed.

Modified oligonucleotides for use in treatment of tauopathies

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. 

Various embodiments provide STOPS&#x2122; 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&#x2122; 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&#x2122; modified oligonucleotides, as determined by HBsAg Secretion Assay, is greater than that of a reference compound.

S-antigen transport inhibiting oligonucleotide polymers and methods

Modified oligonucleotides comprising modifications at the 2' and/or 3&#x2032; positions(s) along with methods of making and use, e.g., against HBV are disclosed.

Disclosed herein are nucleosides, nucleotides and analogs thereof, pharmaceutical compositions that include one or more of nucleosides, nucleotides and analogs thereof, and methods of synthesizing the same. Also disclosed herein are methods of ameliorating and/or treating a paramyxovirus viral infection, with a nucleoside, a nucleotide and an analog thereof.

Substituted nucleosides, nucleotides and analogs thereof

Disclosed herein are cyclic nucleotide analogs, methods of synthesizing cyclic nucleotide analogs and methods of treating diseases and/or conditions such as viral infections, cancer, and/or parasitic diseases with cyclic nucleotide analogs.

Growing community of inventors

Cupertino, CA, United States of America

Vivek Kumar Rajwanshi