Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).

Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).

The present invention relates to a method for producing an ?-aryl substituted carbonyl compound (e.g., an ?-aryl substituted cyclic ketone) from a carbonyl compound (e.g., a cyclic ketone) using an aryl halide or a heteroaryl halide and a photocatalyst (e.g., acridinium, helicenium, angulenium, or a combination thereof) in the presence of an amine compound. The method of the present invention is particularly useful in producing an ?-aryl substituted carbonyl compound (e.g., an ?-aryl substituted cyclic ketone) from an unactivated carbonyl compound (e.g., an unactivated cyclic ketone).

Provided herein are compounds of Formula (I). Methods for the preparation of the compounds of Formula (I) and intermediates useful in the preparation of the compounds of Formula (I) are described herein. The compounds of Formula (I) may be useful as inhibitors of the MYST family of lysine acetyltransferases (KATs) for the treatment of and/or prophylaxis of hyperproliferative diseases, disorders or conditions such as cancer. In particular, the compounds of Formula (I) are useful for the inhibition of KAT6A and KAT6B which are enzymes frequently mutated, overexpressed, amplified and/or translocated in cancer altering their normal expression, activity and function. The use of the compounds of Formula (I) in the manufacture of pharmaceutical compositions or for treating cancers is further described, including for treating cancer in combination with other anti-cancer agents.

The invention provides a compound having a GLUT9 inhibitory activity. The compound is of Formula [I]

##STR00001##

wherein each symbol is as defined in the specification, or a pharmaceutically acceptable salt thereof.

The invention provides a compound having a GLUT9 inhibitory activity. The compound is of Formula [I]

##STR00001##

wherein each symbol is as defined in the specification, or a pharmaceutically acceptable salt thereof.

Provide herein are compounds and pharmaceutical compositions suitable as modulators of hemoglobin, methods and intermediates for their preparation, and methods for their use in treating disorders mediated by hemoglobin and disorders that would benefit from tissue and/or cellular oxygenation.

Methods and compositions for treating lysosomal storage disorders are disclosed. The methods involve administering a genus of benzenesulfonamides, particularly N-[3-(aminosulfonyl)phenyl]-benzamides and heteroarylamides. A genus of suitable compounds is shown in formula 1: ##STR00001##

Methods and compositions for treating lysosomal storage disorders are disclosed. The methods involve administering a genus of benzenesulfonamides, particularly N-[3-(aminosulfonyl)phenyl]-benzamides and heteroarylamides. A genus of suitable compounds is shown in formula 1: ##STR00001##

The present disclosure relates to a novel lipid compound and a lipid nanoparticle composition including the same and, more specifically, the lipid nanoparticle composition includes ionized lipid, helper lipid, PEG-lipid, and additives, and may mitigate changes and side effects in the delivery mechanism and enhance the protein expression efficiency by including a biofriendly vitamin-based novel lipid compound and helper lipids including neutral lipids.