C07F9/65616

Modified nucleic acid

Provided is an oligonucleic acid analog which contains, as at least one structural unit thereof, a modified nucleic acid monomer compound which is a ring-open nucleoside having a cleaved carbon-carbon bond between the 2′ and 3′ positions and a substituent hydroxymethyl group at the 4′ position. When used as siRNA, the oligonucleic acid analog exhibits superior biological stability and target gene expression inhibiting activity. The oligonucleic acid analog can be used in antisense methods, ribozyme methods, and decoy methods, etc., can be used as a nucleic acid aptamer, and can also be used as a nucleic acid probe or molecular beacon, etc., or in genetic diagnostics, etc.

Methods for treating Filoviridae virus infections

Provided are compounds, methods, and pharmaceutical compositions for treating Filoviridae virus infections by administering ribosides, riboside phosphates and prodrugs thereof, of Formula IV: ##STR00001##
The compounds, compositions, and methods provided are particularly useful for the treatment of Marburg virus, Ebola virus and Cueva virus infections.

PHOSPHOROUS DERIVATIVES AS KINASE INHIBITORS

The invention features compounds of the general formula (I) in which the variable groups are as defined herein, and to their preparation and use.

##STR00001##

PHOSPHONATE NUCLEOSIDES USEFUL IN THE TREATMENT OF VIRAL DISEASES
20170218001 · 2017-08-03 ·

The present invention relates to a compound of formula (I), or a pharmaceutically acceptable salt or prodrug thereof, and their use in medicine particular as anti-viral agents;

##STR00001##

wherein: X is selected from O and NR.sup.11; Y is selected from O, S and NR.sup.12; A is selected from —(CR.sup.1R.sup.2)n-, —(CR.sup.9R.sup.10)—, —(CR.sup.9R.sup.10)—(CR.sup.1R.sup.2)n-, —(CR.sup.1R.sup.3)—(CR.sup.2R.sup.4)—(CR.sup.1R.sup.2)n-, —CR.sup.3═CR.sup.4—(CR.sup.1R.sup.2)n- and —C≡C—(CR.sup.1R.sup.2)n-; R.sup.1 and R.sup.2 are independently selected from H, alkyl, hydroxyl, hydroxymethyl and halogen; R.sup.3 and R.sup.4 are independently selected from H and alkyl, or R.sub.3 and R.sub.4 together with the carbon atoms to which they are attached form a mono or bicyclic ring system selected from cycloalkyl, cycloalkenyl, heterocycloalkyl, heterocycloalkenyl, aryl and heteroaryl; R.sup.5 is selected from H, P(═O)(OH).sub.2 and P(═O)(OH)—O—P(═O)(OH).sub.2; R.sup.6 is selected from H and alkyl; R.sup.7 and R.sup.8 are independently selected from H, alkyl, halogen and hydroxymethyl R.sup.9 and R.sup.19 together with the carbon atoms to which they are attached form a mono or bicyclic ring system selected from cycloalkyl, cycloalkenyl, heterocycloalkyl and heterocycloalkenyl; R.sup.11 is selected from H and alkyl; R.sup.12 is selected from H and alkyl; m is 0, 1, 2 or 3; n is 1, 2 or 3; p is 0 or 1; q is 0, 1, 2 or 3; r is 0, 1, 2, 3, 4 or 5; s is 0 or 1; Base is a natural or non-natural nucleobase, and wherein each alkyl, cycloalkyl, cycloalkenyl, heterocycloalkyl, heterocycloalkenyl, aryl and heteroaryl may be optionally substituted as described herein.

Phospholipidation of imidazoquinolines and oxoadenines

The present invention relates to a process for phospholipidation of imidazoquinolines and oxoadenines. More particularly, the present invention relates to a high-yielding and scalable procedure for the phospholipidation of imidazoquinolines and oxoadenines which obviates the need to isolate unstable phosphoramidite intermediates. This process may be used for the phospholipidation of toll-like receptor 7 (TLR7)-active and toll-like receptor (TLR8)-active imidazoquinolines and oxoadenines.

MODULATORS OF 5'-NUCLEOTIDASE, ECTO AND THE USE THEREOF

Compounds that modulate the conversion of AMP to adenosine by 5′-nucleotidase, ecto, and compositions containing the compounds and methods for synthesizing the compounds, are described herein. The use of such compounds and compositions for the treatment and/or prevention of a diverse array of diseases, disorders and conditions, including cancer- and immune-related disorders, that are mediated by 5′-nucleotidase, ecto is also provided.

Isoindoline or isoquinoline compounds, a process for their preparation and pharmaceutical compositions containing them

Compounds of formula (I): ##STR00001##
wherein Het, R.sub.3, R.sub.4, R.sub.5, R.sub.7, R.sub.8, R.sub.9, T, p, p′, q, and q′ are as defined in the description. Medicinal products containing the same which are useful in treating pathologies involving a deficit in apoptosis, such as cancer, auto-immune diseases, and diseases of the immune system.

Nucleoside phosphonate salts

The present invention relates to compounds and methods for treating viral diseases. Some compounds of the invention are described by Formula I: ##STR00001## wherein M.sup.+ is Na.sup.+, Li.sup.+, K.sup.+, Ca.sup.2+, Mg.sup.2+, or NR.sub.cR.sub.dR.sub.eR.sub.f.sup.+ and R.sub.c, R.sub.d, R.sub.e and R.sub.f are each independently hydrogen or C.sub.1-5 alkyl, or a stereoisomer, a diastereomer, an enantiomer or racemate thereof.

COMPOSITIONS FOR CONTROLLED RELEASE OF CYSTEAMINE AND SYSTEMIC TREATMENT OF CYSTEAMINE SENSITIVE DISORDERS
20220040127 · 2022-02-10 ·

The invention features compositions, methods, and kits containing (i) one or more cysteamine precursor compounds convertible to cysteamine in vivo, and (ii) optionally agents to enhance that conversion, formulated to produce a spectrum of pharmacokinetic profiles of cysteamine that can be tailored to individual patients and diseases. The invention also features varying modes of administration of the therapeutic substances in the treatment of cystinosis and other cysteamine sensitive disorders. In particular, formulations combining active ingredient(s) with pharmaceutical excipients that permit sustained cysteamine plasma concentrations are featured.

Substituted polycyclic carbamoyl pyridone derivative prodrug

The present invention provides a compound having antiviral effects, particularly having growth inhibitory activity on influenza viruses, a preferred example of the compound being a substituted 3-hydroxy-4-pyridone derivative prodrug having cap-dependent endonuclease inhibitory activity.