Described herein are splice modifying compounds affecting splicing of mRNA, such as pre-mRNA, expressed from the FOXM1 gene, compositions comprising thereof, and methods using the same.

This invention relates to compounds that are agonists of the muscarinic M.sub.1 and M4 receptor and which are useful in the treatment of diseases mediated by the muscarinic M.sub.1 and/or M4 receptors. Also provided are pharmaceutical compositions containing the compounds and the therapeutic uses of the compounds. Compounds provided are of formula (1): and salts thereof.

##STR00001##

A method of administering to a patient in need thereof or contacting with a biological sample, a compound related to Formula I-e ##STR00001##
or pharmaceutically acceptable compositions thereof, is useful to inhibit activity of TLR7/8 or a mutant thereof and/or to treat a TLR7/8-mediated disorder.

A method of administering to a patient in need thereof or contacting with a biological sample, a compound related to Formula I-e ##STR00001##
or pharmaceutically acceptable compositions thereof, is useful to inhibit activity of TLR7/8 or a mutant thereof and/or to treat a TLR7/8-mediated disorder.

The present invention provides PRMT5 inhibitors of Formula (I), wherein R.sub.1 is a non-hydrogen monovalent group; W is a direct bond or —NH—; T, U, and V are independently of each other selected from C and N; R.sub.2 is H or a halo; m is 1 or 2; X is a carbon, a nitrogen, or an oxygen; Y is C or N; Z is a direct bond or a carbon; R.sub.3 is H, a non-hydrogen monovalent group, an oxo group, a bivalent spiro ring-forming group, or a bivalent bridge-forming group; n is 1 or 2; and Formula (II) stands for a single bond or a double bond. Pharmaceutical products comprising the PRMT5 inhibitors and use thereof in treating proliferative disorders such as cancer, metabolic disorders, blood disorders, autoimmune diseases, and inflammatory diseases are also provided. ##STR00001##

Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.

Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.

The present disclosure provides tricyclic compounds of formula (I) that are histone methyltransferases G9a and/or GLP inhibitors and are therefore useful for the treatment of diseases treatable by inhibition of G9a and/or GLP such as cancers and hemoglobinopathies (e.g., beta-thalassemia and sickle cell disease). Also provided are pharmaceutical compositions containing such compounds and processes for preparing such compounds. ##STR00001##

The present invention provides a genus of polycyclic amines that are useful as opioid receptor modulators. The compounds of the invention are useful in both therapeutic and diagnostic methods, including for treating pain, neurological disorders, cardiac disorders, bowel disorders, drug and alcohol addiction, drug overdose, urinary disorders, respiratory disorders, sexual dysfunction, psoriasis, graft rejection or cancer.

The present invention provides a genus of polycyclic amines that are useful as opioid receptor modulators. The compounds of the invention are useful in both therapeutic and diagnostic methods, including for treating pain, neurological disorders, cardiac disorders, bowel disorders, drug and alcohol addiction, drug overdose, urinary disorders, respiratory disorders, sexual dysfunction, psoriasis, graft rejection or cancer.