The present invention provides a nucleic acid-containing lipid nanoparticle comprising an analog of a fatty acid ester of glycerol, and a nucleic acid, wherein the analog is not hydrolyzable by a lipase.

Disclosed is a sulfanyl sulfonic acid compound represented by the following formula (1) or a metal salt thereof. In the formula, m represents an integer of 2 to 7, n represents an integer of 3 to 10, and R.sup.1 and R.sup.2 each independently represent a hydrogen atom or an alkyl group.

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The invention relates to the compounds or its pharmaceutical acceptable polymorphs, solvates, enantiomers, stereoisomers and hydrates thereof. The pharmaceutical compositions comprising an effective amount of compounds of formula I, formula II, formula III, formula IV and formula V and the methods for the treatment of xerostomia may be formulated for oral, buccal, rectal, topical, transdermal, transmucosal, lozenge, spray, intravenous, oral solution, buccal mucosal layer tablet, parenteral administration, syrup, or injection. Such compositions may be used to treatment of oral mucosal inflammatory, dry mouth or oral dry mouth mediated infectious diseases.

The invention relates to the compounds or its pharmaceutical acceptable polymorphs, solvates, enantiomers, stereoisomers and hydrates thereof. The pharmaceutical compositions comprising an effective amount of compounds of formula I, formula II, formula III, formula IV and formula V and the methods for the treatment of xerostomia may be formulated for oral, buccal, rectal, topical, transdermal, transmucosal, lozenge, spray, intravenous, oral solution, buccal mucosal layer tablet, parenteral administration, syrup, or injection. Such compositions may be used to treatment of oral mucosal inflammatory, dry mouth or oral dry mouth mediated infectious diseases.

The disclosure relates to the synthesis of amide containing compounds inflow. In particular, the disclosure relates to the synthesis of polypeptides via native chemical ligation inflow. The disclosure also relates to selective desulfurization or deselenization of amide containing compounds comprising a thiol, disulfide, selenol or diselenide functional group respectively, particularly polypeptides.

The present disclosure relates to prodrugs, double prodrugs, derivatives and analogues of 3-(methylamino)-2-((methylamino)methyl)propane-1-thiol. The compounds of this disclosure also relate to formula I:

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The use of these compounds as radio- and chemo-protectors is also described.

The present invention relates to a new, enhanced process for the manufacture of Cysteamine Bitartrate (I) and of its key intermediate thiazolidine (II).

Furthermore, it relates to a new processes for the manufacture of crystalline anhydrous Cysteamine Bitartrate (polymorph L2) and monohydrate Cysteamine Bitartrate (polymorph L1). The crystalline anhydrous Cysteamine Bitartrate (polymorph L2) so obtained is characterized by particularly fine particle size and good appearance.

The present invention relates to a new, enhanced process for the manufacture of Cysteamine Bitartrate (I) and of its key intermediate thiazolidine (II).

Furthermore, it relates to a new processes for the manufacture of crystalline anhydrous Cysteamine Bitartrate (polymorph L2) and monohydrate Cysteamine Bitartrate (polymorph L1). The crystalline anhydrous Cysteamine Bitartrate (polymorph L2) so obtained is characterized by particularly fine particle size and good appearance.

The invention features methods for the treatment of cystinosis and other cysteamine sensitive disorders in a subject including administration of a disulfide convertible to cysteamine in vivo. The methods can include the separate administration of a reducing agent to the subject to increase the bioavailablity and extend the plasma pharmacokinetic profile of the cysteamine produced following administration of the disulfide. The methods permit sustained cysteamine plasma concentrations in a subject.

Provided herein are lipids having the Formula (I): and pharmaceutically acceptable salts thereof, wherein R.sup.1, R.sup.1′, R.sup.2, R.sup.2′, R.sup.3, R.sup.3′, R.sup.4, R.sup.4′, R.sup.5, and R.sup.5′, are as defined herein. Also provided herein are lipid nano article (LNP) compositions comprising lipid having the Formula (I) and a capsid-free, non-viral vector (e.g., ceDNA). In one aspect, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).