The invention relates to compound shown with formula (I) or a pharmaceutically acceptable derivative thereof for use as a novel inhibitor of MDM2 activity.

The invention relates to compound shown with formula (I) or a pharmaceutically acceptable derivative thereof for use as a novel inhibitor of MDM2 activity.

Disclosed are (i) compounds of formula I, or pharmaceutically acceptable salts thereof; and (ii) lipidoid nanoparticles comprising compound of formula I or pharmaceutically acceptable salts thereof, as well as their use as vehicles for drug delivery across the blood-brain barrier.

Disclosed are (i) compounds of formula I, or pharmaceutically acceptable salts thereof; and (ii) lipidoid nanoparticles comprising compound of formula I or pharmaceutically acceptable salts thereof, as well as their use as vehicles for drug delivery across the blood-brain barrier.

In one aspect, the present disclosure provides compounds of the formula (I): wherein the variables are as defined herein. In another aspect, the present disclosure provides compounds of the formula (V): wherein the variables are as defined herein. In another aspect, the present disclosure also provides methods of preparing the compounds disclosed herein. In another aspect, the present disclosure provides antibody drug conjugates comprising compounds of the present disclosure. In another aspect, the present disclosure also provides pharmaceutical compositions and methods of use of the compounds and anti-body drug conjugates disclosed herein. ##STR00001##

In one aspect, the present disclosure provides compounds of the formula (I): wherein the variables are as defined herein. In another aspect, the present disclosure provides compounds of the formula (V): wherein the variables are as defined herein. In another aspect, the present disclosure also provides methods of preparing the compounds disclosed herein. In another aspect, the present disclosure provides antibody drug conjugates comprising compounds of the present disclosure. In another aspect, the present disclosure also provides pharmaceutical compositions and methods of use of the compounds and anti-body drug conjugates disclosed herein. ##STR00001##

Indole derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD).

The object of the present invention concerns a hair restructuring association comprising or alternatively consisting of: (a) a quaternary ammonium salt of formula (I) and (b) at least one sulpho-derivative of vegetable fatty acids, wherein R.sub.5, R.sub.6 are independently chosen between hydrogen and a radical R.sub.0, R.sub.0 consists of the following structure of formula (II), R.sub.1 is chosen from the group consisting of: hydrogen, methyl, isopropyl, sec-butyl, isobutyl, ethylenemethylthio, benzyl, para-hydroxybenzyl and 3-methylene-1H-indole, R.sub.2, R.sub.3, R.sub.4 are independently chosen from the group consisting of: methyl, ethyl, n-propyl, isopropyl, n-butyl, isobutyl, sec-butyl and tert-butyl, mX.sup.− is chosen from the group consisting of: formic acid, acetic acid, unsaturated monocarboxylic acids, adipic acid, aldaric acid, oxalic acid, phthalic acid, azelaic acid, sebacic acid, malonic acid, succinic acid, tartaric acid, glutaric acid, pimelic acid, maleic acid, malic acid, fumaric acid and suberic acid, isocitric acid, citric acid, fatty acids, acidic amino acids, keto acids and aromatic carboxylic acids, m is an integer number comprised between 1 and 22, n is comprised between 2 and 20.

The present invention provides compounds, compositions thereof, and methods of using the same for the inhibition of USP30, and the treatment of USP30-mediated disorders.

The present invention provides compounds, compositions thereof, and methods of using the same for the inhibition of USP30, and the treatment of USP30-mediated disorders.