Patent classifications
C12N15/1131
Long Acting Pharmaceutical Compositions For Hepatitis C
The present Invention relates to long acting pharmaceutical compositions useful in the treatment or prevention or cure of viral infections, such as HCV infections, and diseases associated with such infections.
COMBINATION LONG ACTING COMPOSITIONS AND METHODS FOR HEPATITIS C
The present Invention relates to pharmaceutical compositions useful in the treatment or prevention or cure of viral infections, such as HCV infections, and diseases associated with such infections.
RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (SINA)
The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
ANTISENSE ANTIVIRAL COMPOUNDS AND METHODS FOR TREATING A FILOVIRUS INFECTION
The present invention provides antisense antiviral compounds, compositions, and methods of their use and production, mainly for inhibiting the replication of viruses of the Filoviridae family, including Ebola and Marburg viruses. The compounds, compositions, and methods also relate to the treatment of viral infections in mammals including primates by Ebola and Marburg viruses. The antisense antiviral compounds include phosphorodiamidate morpholino oligonucleotides (PMOplus) having a nuclease resistant backbone, about 15-40 nucleotide bases, at least two but typically no more than half piperazine-containing intersubunit linkages, and a targeting sequence that is targeted against the AUG start site region of Ebola virus VP35, Ebola virus VP24, Marburg virus VP24, or Marburg virus NP, including combinations and mixtures thereof.
PHARMACEUTICAL COMPOSITION FOR TREATING A VIRAL INFECTION
The invention concerns a pharmaceutical composition for treating a viral infection caused by a member of the Reoviridae family; a method of treatment involving the use of same and use of the anti-viral to treat said viral infection. The agent has use in both humans and animals.
HBV BINDING OLIGONUCLEOTIDES AND METHODS OF USE
Oligonucleotides that target hepatitis B virus (HBV) viral sequences, such as rcDNA, cccDNA, and HBV transcripts, are described herein. In addition, compositions and kits comprising such oligonucleotides are further described. Further disclosed herein are uses of such oligonucleotides and compositions to reduce rcDNA to cccDNA conversion, reduce cccDNA levels, and/or treat an HBV infection.
Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
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).
TARGET SEQUENCE OF RNA VIRUS AND USE THEREOF
The present invention provides a target sequence of an RNA virus. The target sequence is a nucleic acid sequence fragment in the gene sequence in the RNA virus containing 20-40 bases and having not less than 95% similarity to genome sequence of human or related species such as livestock and poultry. The above-mentioned target sequence of the RNA virus is selected from SEQ ID NO. 1 - SEQ ID NO. 615. The present invention also relates to a primer composition for constructing the above-mentioned target sequence, biomaterials such as antisense RNA related to the above-mentioned target sequence, and related uses such as design of a vaccine lacking the target sequence. The virus fragment with the above-mentioned sequence constructed in the present invention has the function of interacting with human genomic DNA and is similar to viral miRNA. Moreover, the effect of overexpression of the target sequence of the RNA virus on the expression level of surrounding genes is verified, and a new concept that the above-mentioned target fragment is an important pathogenic substance of the RNA virus is proposed. The above-mentioned target sequence has important application value for the detection and diagnosis of RNA viruses, drug screening, as well as the treatment of diseases caused by RNA viruses and the design/optimization of vaccines and methods.
MICROMIRs
The present invention relates to very short heavily modified oligonucleotides which target and inhibit microRNAs in vivo, and their use in medicaments and pharmaceutical compositions.
Functionally-modified oligonucleotides and subunits thereof
Functionally-modified oligonucleotide analogs comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.