7- or 8-hydroxy-isoquinoline and 7- or 8-hydroxy-quinoline derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD).

The present disclosure provides compounds, pharmaceutically acceptable compositions thereof, and methods of using the same.

The present invention relates to quinoline derived small molecule inhibitors of nicotinamide N-methyltransferase (NNMT), the preparation thereof and uses thereof.

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In vitro or ex vivo methods for screening a quinoline derivative, or anyone of its pharmaceutically acceptable salt, presumed effective in treating and/or preventing an inflammatory disease, the method including: providing an eukaryotic cell, bringing into contact said cell with a quinoline derivative, measuring an expression of miR-124 in the cell, and selecting the candidate presumed effective in treating and/or preventing an inflammatory disease when the level of expression of miR-124 measured is increased relatively to a reference value.

In vitro or ex vivo methods for screening a quinoline derivative, or anyone of its pharmaceutically acceptable salt, presumed effective in treating and/or preventing an inflammatory disease, the method including: providing an eukaryotic cell, bringing into contact said cell with a quinoline derivative, measuring an expression of miR-124 in the cell, and selecting the candidate presumed effective in treating and/or preventing an inflammatory disease when the level of expression of miR-124 measured is increased relatively to a reference value.

A compound, or a pharmaceutically acceptable salt, solvate or prodrug thereof, having the chemical structure of formula I as defined in the text and methods of using these compounds to inhibit topoisomerase IIα and treat or prevent metastasis of cancer in a subject.

The present disclosure includes in one aspect substituted arylmethyl ureas, substituted heteroarylmethyl ureas, or analogues thereof, and compositions comprising the same, that can be used to treat or prevent hepatitis B virus (HBV) and/or hepatitis D virus (HDV) infections in a patient.

The present disclosure includes in one aspect substituted arylmethyl ureas, substituted heteroarylmethyl ureas, or analogues thereof, and compositions comprising the same, that can be used to treat or prevent hepatitis B virus (HBV) and/or hepatitis D virus (HDV) infections in a patient.

The invention provides immunoconjugates of Formula (I) comprising an antibody linked by conjugation to one or more aminoquinoline derivatives. The invention also provides aminoquinoline derivative intermediate compositions of Formula (III) comprising a reactive functional group. Such intermediate compositions are suitable substrates for formation of the immunoconjugates through a linker or linking moiety. The invention further provides methods of treating cancer with the immunoconjugates.

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecules having a phenyl-sulfonic amide (or similar) structure which function as agonists of mucolipin 1 (ML1), and their use as therapeutics for the treatment of Duchenne muscular dystrophy (DMD) and related disorders.