C07H19/16

Substituted nucleosides, nucleotides and analogs thereof

Disclosed herein are nucleotide analogs, methods of synthesizing nucleotide analogs and methods of treating diseases and/or conditions such as a HCV infection with one or more nucleotide analogs.

Substituted nucleosides, nucleotides and analogs thereof

Disclosed herein are nucleotide analogs, methods of synthesizing nucleotide analogs and methods of treating diseases and/or conditions such as a HCV infection with one or more nucleotide analogs.

Synthesis of Protected 3'-amino Nucleoside Monomers
20230089101 · 2023-03-23 ·

Orthogonally protected 3′-amino nucleoside monomers and efficient methods for their synthesis are described. The methods employ selective protection of the 3′-amino group in the presence of the unprotected nucleoside base.

Synthesis of Protected 3'-amino Nucleoside Monomers
20230089101 · 2023-03-23 ·

Orthogonally protected 3′-amino nucleoside monomers and efficient methods for their synthesis are described. The methods employ selective protection of the 3′-amino group in the presence of the unprotected nucleoside base.

RNA-editing oligonucleotides and uses thereof

The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

RNA-editing oligonucleotides and uses thereof

The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

ADENOSINE COMPOUND, ITS PHARMACEUTICALLY ACCEPTABLE SALT OR ITS STEREOISOMER AND USE THEREOF
20220332750 · 2022-10-20 ·

The present invention relates to a compound represented by Formula I, its pharmaceutically acceptable salt, its stereoisomer, its pharmaceutically acceptable hydrate or solvate, or its pharmaceutically acceptable ester, wherein R is selected from heteroaryl, substituted heteroaryl, aryl, substituted aryl, cycloalkyl, substituted cycloalkyl, alkyl, and substituted alkyl, wherein the substituted heteroaryl, the substituted aryl, the substituted cycloalkyl and the substituted alkyl are each independently substituted with one or more hydroxyalkylene groups. The compound or its pharmaceutically acceptable salt, its stereoisomer, its pharmaceutically acceptable hydrate or solvate, its pharmaceutically acceptable ester of the present invention has agonistic activity on A.sub.2A adenosine receptor and can improve the permeability of blood-brain barrier to promote the delivery of drug across blood-brain barrier, and can also prevent or treat a disease associated with A.sub.2A adenosine receptor agonistic activity.

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ADENOSINE COMPOUND, ITS PHARMACEUTICALLY ACCEPTABLE SALT OR ITS STEREOISOMER AND USE THEREOF
20220332750 · 2022-10-20 ·

The present invention relates to a compound represented by Formula I, its pharmaceutically acceptable salt, its stereoisomer, its pharmaceutically acceptable hydrate or solvate, or its pharmaceutically acceptable ester, wherein R is selected from heteroaryl, substituted heteroaryl, aryl, substituted aryl, cycloalkyl, substituted cycloalkyl, alkyl, and substituted alkyl, wherein the substituted heteroaryl, the substituted aryl, the substituted cycloalkyl and the substituted alkyl are each independently substituted with one or more hydroxyalkylene groups. The compound or its pharmaceutically acceptable salt, its stereoisomer, its pharmaceutically acceptable hydrate or solvate, its pharmaceutically acceptable ester of the present invention has agonistic activity on A.sub.2A adenosine receptor and can improve the permeability of blood-brain barrier to promote the delivery of drug across blood-brain barrier, and can also prevent or treat a disease associated with A.sub.2A adenosine receptor agonistic activity.

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Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12
20230132391 · 2023-04-27 ·

Codon optimized nucleic acid sequences for RDH12 are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequence for functional RDH12. Recombinant vectors are provided that express the codon optimized, functional RDH12. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence, deficiency or inappropriate expression of RDH12. Other compositions and methods are providing for correcting a non-functional, defective or inadequately expressed native RDH12.

POLYMERIC SOLID SUPPORT FOR OLIGONUCLEOTIDE SYNTHESIS

Embodiments of the present application relate to porous polymeric beads solid support for use in oligonucleotide synthesis and the method of producing and using the same. Further embodiments relate to porous polymeric beads and method for preparing and using the same.