Novel compounds, compositions, and methods of using and preparing the same, which maybe useful for treating alpha-1 antitrypsin deficiency (AATD).

Novel compounds, compositions, and methods of using and preparing the same, which maybe useful for treating alpha-1 antitrypsin deficiency (AATD).

Compositions and methods for visualizing tissue under illumination with near-infrared radiation, including compounds comprising near-infrared, closed chain, sulfo-cyanine dyes and prostate specific membrane antigen ligands are disclosed.

Compositions and methods for visualizing tissue under illumination with near-infrared radiation, including compounds comprising near-infrared, closed chain, sulfo-cyanine dyes and prostate specific membrane antigen ligands are disclosed.

Described herein are compounds and compositions characterized, in certain embodiments, by conjugation of various groups, such as lipophilic groups, to an amino or amide group of an amino acid, a linear or cyclic peptide, a linear or cyclic polypeptide, or structural isomer thereof, to provide compounds of the present invention, collectively referred to herein as “APPLs”. Such APPLs are deemed useful for a variety of applications, such as, for example, improved nucleotide delivery. Exemplary APPLs include, but are not limited to, compounds of Formula (I), (II), (III), (IV), (V), and (VI), and salts thereof, as described herein: ##STR00001##
wherein m, n, p, R′, R.sup.1, R.sup.2, R.sup.3, R.sup.4, R.sup.5, R.sup.8, Z, W, Y, and Z are as defined herein.

Described herein are compounds and compositions characterized, in certain embodiments, by conjugation of various groups, such as lipophilic groups, to an amino or amide group of an amino acid, a linear or cyclic peptide, a linear or cyclic polypeptide, or structural isomer thereof, to provide compounds of the present invention, collectively referred to herein as “APPLs”. Such APPLs are deemed useful for a variety of applications, such as, for example, improved nucleotide delivery. Exemplary APPLs include, but are not limited to, compounds of Formula (I), (II), (III), (IV), (V), and (VI), and salts thereof, as described herein: ##STR00001##
wherein m, n, p, R′, R.sup.1, R.sup.2, R.sup.3, R.sup.4, R.sup.5, R.sup.8, Z, W, Y, and Z are as defined herein.

Provided herein are small molecules that bind to ASH1L and inhibit ASH1L activity, and methods of use thereof for the treatment of disease, including acute leukemia, solid cancers and other diseases dependent on activity of ASH1L.

Provided herein are small molecules that bind to ASH1L and inhibit ASH1L activity, and methods of use thereof for the treatment of disease, including acute leukemia, solid cancers and other diseases dependent on activity of ASH1L.

The present invention relates to compounds that inhibit expression of Id1 and/or Id3, as well as uses thereof in the treatment of cancer and other diseases and conditions associated with Id1 and/or Id3 expression.

Compounds useful in the modulation of ion channel activity in cells are disclosed herein. This disclosure also relates to use of these compounds in the treatment of pain, and pharmaceutical compositions containing these compounds and methods for their preparation.