Cationic steroidal antimicrobial (CSA) compounds having a structure of Formula I, II or III, or salt thereof, wherein at least one of R.sub.1-R.sub.18, (e.g., R.sub.18) includes a terpenyl group attached via an ester or amide linkage and at least one R.sub.1-R.sub.18, (e.g., R.sub.3, R.sub.7 and R.sub.12) is an amino acid linked to the steroidal backbone by an ester or amide linkage:

##STR00001## R.sub.18 has the following structure:

—R.sub.19—(C═O)—X—R.sub.20 where R.sub.19 is omitted or alkyl, alkenyl, alkynyl, or aryl, X is oxygen or nitrogen, and R.sub.20 is a terpenyl group. R.sub.3, R.sub.7 and R.sub.12 have the following structure:

R.sub.22R.sub.23N—R.sub.21—(C═O)—X— where R.sub.21 is substituted or unsubstituted alkyl, X is oxygen or nitrogen, and R.sub.22 and R.sub.23 are independently hydrogen, alkyl, alkenyl, alkynyl, or aryl.

Cationic steroidal antimicrobial (CSA) compounds having a structure of Formula I, II or III, or salt thereof, wherein at least one of at least one of R.sub.1-R.sub.18, (e.g., R.sub.3, R.sub.7 and R.sub.12) is linked to the steroidal backbone by a urethane group:

##STR00001##

At least one of R.sub.1-R.sub.18, (e.g., R.sub.3, R.sub.7 and R.sub.12) has the following urethanyl structure:

—O—(C═O)—NR.sub.19R.sub.20

where R.sub.19 and R.sub.20 are independently hydrogen, alkyl, alkenyl, alkynyl, aryl, aminoalkyl, aminoalkenyl, aminoalkynyl, or aminoaryl, provided that at least one of R.sub.19 or R.sub.20 includes an amino group (e.g., R.sub.19 is hydrogen and R.sub.20 is (C.sub.2-C.sub.6)aminoalkyl).

R.sub.18 can have the following structure:

—R.sub.21—(C═O)—NR.sub.22R.sub.23

where R.sub.21 is omitted or alkyl, alkenyl, alkynyl, or aryl, and R.sub.22 and R.sub.23 are hydrogen, alkyl, alkenyl, alkynyl, or aryl, provided that at least one of R.sub.22 or R.sub.23 is not hydrogen.

The invention relates to sterol esters and methods for producing purified sterols that can be utilized in methods for producing a lipid nanoparticle.

The present disclosure provides LNPs comprising payload molecules, e.g., mRNA therapeutics, for the treatment of diseases or disorders, which would benefit from delivery of payload molecules to airway cells.

Described herein is the chemical structure of neurosteroid derivative compounds, methods of synthesizing the derivatives, and their uses in treating disorders, including those of the central nervous system. These compounds are readily synthesized and have improved pharmaceutical properties, including water solubility, compared to known neurosteroids.

The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.

Provided are androstane and dihydrotestosterone compounds functionalized with carbocyclic groups or heterocyclic groups that may be saturated or unsaturated. The compounds may be used in methods of inhibiting cell growth of malignant cells and/or hyperplastic cells and/or treating individuals having diseases associated with malignant cell growth (e.g., cancer, such as, for example, prostate cancer) and/or hyperplastic cell growth and/or molecular imaging of malignant cells and/or hyperplastic cells and/or inducing degradation of a target protein. Also provided are compositions.

Disclosed are novel compounds that accumulate in Gram-negative bacteria. Also disclosed are method of antimicrobial treatment using the compounds.

Disclosed herein are cationic steroidal antimicrobials (“CSA compounds” or “ceragenins”) and methods of making the same. Particularly advantageous methods are identified for the synthesis of CSA compounds. CSA compounds may be formulated for treating subjects with ailments responsive to CSA compounds, including but not limited to treating bacterial infections. Accordingly, some embodiments include formulations and methods of administering CSA compounds.

The present invention relates to a method for preparing a modified hydrophobic excipient including the following steps: Step A. obtaining intermediate I or II from a hydrophobic natural compound with one to three hydroxyl groups or a hydrophobic synthetic compound with one to three hydroxyl groups and amino acid derivatives with amino protecting groups and different chain lengths acting as raw material; Step B. obtain intermediate III from an amino acid derivative and N-hydroxysuccinimide or 1-hydroxybenzotriazole acting as raw material and a dehydrating agent; and Step C. reacting the intermediate II and the intermediate III as raw material with an acid-binding agent under a dark condition to generate a modified hydrophobic excipient.