An exemplary embodiment relates to novel protein modulators capable of altering function of the mutant CFTR protein and their use for treating diseases associated with CFTR protein malfunction. An exemplary embodiment provides compositions, pharmaceutical preparations and methods of correcting the cellular alteration of a mutant CFTR protein wherein the CFTR mutation is a mutation F508-CFTR, or another mutation of class II.

The present invention provides therapeutic strategies for treatment of severe debilitating diseases associated with IFN-I due to cGAS activation. In one aspect, the invention provides compounds of Formula (I): [Formula should be inserted here] and pharmaceutical uses thereof. In another aspect, the invention provides methods for treating an autoimmune disease or a monogenic disorder by administering an effective amount of a compound of Formula (I). ##STR00001##

The present invention provides therapeutic strategies for treatment of severe debilitating diseases associated with IFN-I due to cGAS activation. In one aspect, the invention provides compounds of Formula (I): [Formula should be inserted here] and pharmaceutical uses thereof. In another aspect, the invention provides methods for treating an autoimmune disease or a monogenic disorder by administering an effective amount of a compound of Formula (I). ##STR00001##

The invention concerns ionic compounds in which the anionic load has been delocalized. A compound disclosed by the invention is comprised of an amide or one of its salts, including an anionic portion combined with at least one cationic portion M.sup.+m in sufficient numbers to ensure overall electronic neutrality; the compound is further comprised of M as a hydroxonium, a nitrosonium NO.sup.+, an ammonium NH.sub.4.sup.+, a metallic cation with the valence m, an organic cation with the valence m, or an organometallic cation with the valence m. The anionic portion matches the formula R.sub.FSO.sub.xN.sup.?Z, where R.sub.F is a perflourinated group, x is 1 or 3, and Z is an electroattractive substituent. The compounds can be used notably for ionic conducting materials, electronic conducting materials, colorants and the catalysis of various chemical reactions.

The invention concerns ionic compounds in which the anionic load has been delocalized. A compound disclosed by the invention is comprised of an amide or one of its salts, including an anionic portion combined with at least one cationic portion M.sup.+m in sufficient numbers to ensure overall electronic neutrality; the compound is further comprised of M as a hydroxonium, a nitrosonium NO.sup.+, an ammonium NH.sub.4.sup.+, a metallic cation with the valence m, an organic cation with the valence m, or an organometallic cation with the valence m. The anionic portion matches the formula R.sub.FSO.sub.xN.sup.?Z, where R.sub.F is a perflourinated group, x is 1 or 3, and Z is an electroattractive substituent. The compounds can be used notably for ionic conducting materials, electronic conducting materials, colorants and the catalysis of various chemical reactions.

We disclose novel asymmetric bis-acridines with antitumor activity. These compounds are useful for use in pharmaceuticals, particularly in the treatment or the prevention of neoplasms.

We disclose novel asymmetric bis-acridines with antitumor activity. These compounds are useful for use in pharmaceuticals, particularly in the treatment or the prevention of neoplasms.

The present invention provides for novel acridine-like class of compounds that have demonstrated efficiency in treating cancer. The compounds of the present invention have demonstrated efficacy in binding to and antagonizing the activity of the p53 repressor, HDMX. Once administered to a cell, the compounds of the present invention bind HDMX, thereby allowing p53 to induce apoptosis of the cancerous cell. A combination of this class of compounds along with Nutlin3 provides a novel approach to treat cancers.

The present invention provides for novel acridine-like class of compounds that have demonstrated efficiency in treating cancer. The compounds of the present invention have demonstrated efficacy in binding to and antagonizing the activity of the p53 repressor, HDMX. Once administered to a cell, the compounds of the present invention bind HDMX, thereby allowing p53 to induce apoptosis of the cancerous cell. A combination of this class of compounds along with Nutlin3 provides a novel approach to treat cancers.

Described herein are compounds that inhibits the membrane proximal external region (MPER) of a viral envelope (Env), as well as and compositions thereof. Further provided herein are methods for treating or preventing a viral infection comprises administering to a subject in need thereof an agent or compound that inhibits the MPER of a viral Env. In certain embodiments, the viral infection is an HIV infection.