Phosphonate linkers and their use for delivering compounds with passive cell permeability into a cell wherein the phosphonate group facilitates cellular retention of the compound are described.

This invention relates to pharmaceutical formulations of gemcitabine-[phenyl-benzoxy-L-alaninyl)]-phosphate, a monophosphate derivative of the well-known oncology drug gemcitabine. In particular, the invention relates to formulations which comprise a polar aprotic solvent, preferably dimethyl acetamide (DMA). Formulations comprising these solvent provide therapeutically effective treatments of gemcitabine-[phenyl-benzoxy-L-alaninyl)]-phosphate. The invention also relates to methods of using said formulations and kits comprising said formulations.

Described herein are prostate specific membrane antigen (PSMA) binding conjugates that are useful for delivering therapeutic, diagnostic and imaging agents. Also described herein are pharmaceutical composition containing them and methods of using the conjugates and compositions. Also described are processes for manufacture of the conjugates and the compositions containing them.

The present invention provides phosphorylcholine conjugates and pharmaceutical compositions comprising same for the prevention or treatment of autoimmune diseases. In particular, the conjugates of the present invention are effective in treating autoimmune diseases associated with pathological inflammation.

The present invention relates to a mixed-charge nanoparticle for inducing selective death of cancer cells and a use thereof. The mixed-charge nanoparticle of the present invention is localized and crystallized specifically in cancer cell lysosomes through a pH-dependent aggregation behavior due to the balance between positively charged ligands and negatively charged ligands on the surface thereof and can induce lysosomal membrane permeabilization (LMP) and lysosomal cell death mediated thereby, like cationic amphiphilic drugs (CADs) Exhibiting a cancer cell-specific death effect, the nanoparticles of the present invention can surmount the limited medical use of conventional cationic nanoparticles due to the non-specific cytotoxicity thereof. Particularly, the nanoparticles of the present invention do not exhibit toxicity to the human body and normal cells, thus finding useful applications in medical and medicinal uses such as for prevention and treatment of solid cancer, blood cancer, and tumors.

Provided is a drug containing a liver targeting specific ligand and a thyroid hormone receptor agonist in its structure, which is a new drug structure formed by linking the liver targeting specific ligand with the thyroid hormone receptor agonist through a branched chain, a linker and a linking chain. Thyroid hormone receptors (TRs) are divided into two subtypes, TR-α and TR-β, wherein, TR-β is mainly expressed in liver, and TR-α is mainly expressed in heart, nervous system, etc. In certain embodiments, it is envisaged that the provided drug has the action of liver targeting, can specifically bring the thyroid hormone receptor agonist into liver, without entering heart and other issues, and may thereby avoid side effects caused by the action of the thyroid hormone receptor agonist on other issues, and maintain its therapeutic efficacy in the treatment of lipid metabolism disorders and related complications.

Disclosed are compositions comprising: a) an extract of Curcuma longa, curcumin or phospholipid-complexed curcumin; b) a lipophilic extract of Zingiber officinale; and c) a lipophilic extract obtained from plants containing isobutylamides of polyunsaturated fatty acids selected from the group consisting of Echinacea spp. extract, Zanthoxylum spp. extract and Acmella oleracea (Spilanthes oleracea) extract.

The present invention provides capsular polysaccharides from Streptococcus pneumoniae serotypes identified using NMR. The present invention further provides polysaccharide-protein conjugates in which capsular polysaccharides from one or more of these serotypes are conjugated to a carrier protein such as CRM197. Polysaccharide-protein conjugates from one or more of these serotypes may be included in multivalent pneumococcal conjugate vaccines having polysaccharides from multiple additional Steptococcus pneumoniae serotypes.

The invention relates to a liquid oral formulation of bumetanide for the treatment of autism, more particularly for the improvement of the Autism Spectrum Disorder (ASD) core symptoms, tuberous sclerosis complex, fragile X syndrome, Rett syndrome, Down syndrome, cancer and particularly gliomas, spinal cord lesions, chronic pain, brain trauma, cerebrovascular infarcts, various types of epilepsies, and also acute lung injury such as pneumonias or Severe Acute Respiratory Disease due to Coronavirus-2 (SARS-CoV-2). Said formulation is especially designed for paediatric populations. The invention relates also to a posology for the administration of said liquid oral formulation.

The present invention provides compositions and methods for providing controllable local delivery of a conjugate of bortezomib (Btz) and a bisphosphonate to promote bone formation. In certain embodiments, the invention is used as a treatment for a subject with diseases and disorders characterized by bone loss.