The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

The present application provides that synthetic retinoid compounds are useful in methods of treating bacterial infections, such as a bacterial infection caused by S. aureus, methicillin-resistant S. aureus (MRSA), vancomycin-resistant S. aureus (VRSA), E. faecalis, E. faecium, B. subtilis, and B. anthracis. The present application also provides a tricyclic fluoroquinolone compound, Z3060, useful in methods of treating bacterial infections, such as a bacterial infection caused by S. aureus, methicillin-resistant S. aureus (MRSA), vancomycin-resistant S. aureus (VRSA), E. spp., K. pneumoniae, P. aeruginosa, A. baumannii, E. faecium, and E. faecalis. Also provided herein is a gold compound, auranofin, useful in treating bacterial and fungal infections, such as a fungal infection caused by C. albicans, C. parapsilosis, C. tropicalis, C. glabrata, and C. neoformans.

The present invention relates to methods for treating uremic pruritus with anti-pruritic compositions, wherein the method provides a therapeutic effect in a patient.

Some embodiments of the invention include inventive compounds (e.g., compounds of Formula (I)). Other embodiments include compositions (e.g., pharmaceutical compositions) comprising the inventive compound. Still other embodiments of the invention include compositions (e.g., pharmaceutical compositions) for treating, for example, certain diseases using the inventive compounds. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as cancer). Further embodiments include methods for making the inventive compounds. Additional embodiments of the invention are also discussed herein.

This invention provides formulations for use in distributing RNAi molecules targeted to a human GST-π for treating a malignant tumor in a subject. The formulation can include nanoparticles composed of an ionizable lipid, a DSPE lipid, and additional lipids. A drug product can be made by lyophilization of the formulation. This invention further provides methods for ameliorating or treating a malignant tumor by administering a therapeutically effective amount of a formulation containing the RNAi agents.

The present invention provides isolated monoclonal antibodies, particularly human antibodies, that bind to IP-10 with high affinity, inhibit the binding of IP-10 to its receptor, inhibit IP-10-induced calcium flux and inhibit IP-10-induced cell migration. Nucleic acid molecules encoding the antibodies of the invention, expression vectors, host cells and methods for expressing the antibodies of the invention are also provided. Immunoconjugates, bispecific molecules and pharmaceutical compositions comprising the antibodies of the invention are also provided. The invention also provides methods for inhibiting IP-10 activity using the antibodies of the invention, including methods for treating various inflammatory and autoimmune diseases.

The present invention provides a method of treating and/or preventing pulmonary hypertension in a subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of a bioactive peptide preparation derived from at least one botanical source.

A method of treating congenital cytomegalovirus (cCMV) induced hearing loss includes the step administering a composition to the mammal, wherein the composition consists essentially of a biologically effective amount of vitamin A, vitamin E, vitamin C, a vasodilator comprising magnesium, and, optionally, a withanolide, and/or resveratrol.

A method of treatment of a neuropathic pain by administering an andrographolide to the subject is provided. The andrographolide treatment may relief the symptom of neuropathic pain by reducing the astrocytic activity.

The present invention provides a pharmaceutical composition comprising a binary conjugate, DC009, which is a conjugate of a thrombolytic peptide (Pro-Ala-Lys) and a tetrahydroisoquinoline compound having two C1-4 alkyl groups via a lysine linking arm, and a pharmaceutical acceptable carrier. The composition has a pH less than 6.5, preferably has a pH about pH 2-5.5 The composition may comprise a pharmaceutical acceptable excipient such as mannitol, sorbitol, sucrose, lactose, or trehalose.