The invention described herein pertains to folate-receptor targeted agents comprising therapeutic agents useful for the treatment of inflammatory disease, including folate-receptor targeted liposomes (folate-targeted liposomes) containing entrapped therapeutic agents and folate-receptor targeted dendrimers conjugated to therapeutic agents (folate-targeted dendrimer conjugates), useful for the treatment of inflammatory disease, including auto-immune disease, as well as to folate-targeted liposomes containing entrapped imaging agents and dendrimer conjugates conjugated to imaging agents, for use in the diagnosis and monitoring of treatment in such disease.

Methods and compositions for enhancing the immune response to an IRM compound by depositing within a localized tissue region an IRM depot preparation that provides an extended residence time of active IRM within the localized tissue region.

Lipid-based nanoparticle compositions are provided. The compositions generally comprise lipid-hydrophilic polymer-amyloid binding ligand conjugates, and may be liposomal compositions. The compositions, including the liposomal compositions, may be useful for imaging and/or the treatment of amyloid-β plaque deposits characteristic of Alzheimer's Disease.

The present disclosure relates to a method for in vivo targeting of a nanoparticle via bioorthogonal copper-free click chemistry, more particularly to a method for in vivo targeting of a nanoparticle, including: injecting a precursor capable of being metabolically engineered in vivo when injected into a living system and having a first bioorthogonal functional group into the living system; and injecting a nanoparticle having a second bioorthogonal functional group which can perform a bioorthogonal copper-free click reaction with the first bioorthogonal functional group attached thereto into the living system. In accordance with the present disclosure, accumulation of nanoparticles at a target site in a living system can be increased remarkably and the biodistribution of the nanoparticles can be controlled since the nanoparticles bound to a cell surface are taken up into the cell with time.

TNFα antisense oligonucleotides are provided herein. Methods of treating TNFα diseases or Initial Screen of Phosphodiester disorders using the TNFα antisense oligonucleotides and related products are provided.

The invention relates to a nanostructured delivery system comprising at least one polymer and/or at least one lipid and at least one polymethine dye, wherein the at least one polymethine dye acting as a targeting unit brings about the targeted transport of the nanostructured delivery system into a target issue. The invention also relates to a pharmaceutical composition and the uses of the nanostructured delivery system for transporting said system and, optionally, a pharmaceutical active ingredient into the target tissue, as well for treating liver and/or kidney diseases.

The present invention is related to a lipid composition contained in and/or containing a carrier comprising at least a first lipid component, at least a first helper lipid, and a shielding compound which is optionally removable from the lipid composition under in vivo conditions, whereby the lipid composition containing carrier has an osmolarity of about 50 to 600 mosmole/kg, preferably about 250-350 mosmole/kg, and more preferably about 280 to 320 mosmole/kg, and/or whereby liposomes formed by the first lipid component and/or one or both of the helper lipid and the shielding compound in the carrier have a particle size of about 20 to 200 nm, preferably about 30 to 100 nm, and more preferably about 40 to 80 nm.

The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.

Graft copolymer polyelectrolyte complexes are disclosed for the efficient delivery of anionic, cationic or polyelectrolyte therapeutic agents into biological cells, and for maintaining the biological activity of these molecules while in serum and other aqueous environments are provided. Such complexes comprise (1) an anionic graft copolymer containing an anionic polymer backbone, with pendent carboxylic acid groups and pendant chains containing amphipathic or hydrophilic polymers covalently bonded to a portion of the pendant carboxylic acid groups, (2) one or more anionic, cationic or polyelectrolyte therapeutic agents, and (3) optionally a liposome optionally containing an additional therapeutic agent. Also disclosed are functional nanoparticles containing the complexes.

Pharmaceutical compositions comprising stabilized self-assembling amphiphilic molecular structure (SAMS) having average diameters of 120 nm or less with a low zeta potential of less than 30 mV to be used for Total Parenteral Nutrition (TPN) or Total Parenteral Alimentation (TPA), increase blood pressure, renal dialysis, transport and release hydrophobic gases, exchange transfusion to replace blood, extracorporeal membrane oxygenation (ECMO), open constricted or blocked blood vessels, treat septic shock and irreversible shock due to sepsis and/or severe blood loss irreversible septic shock, treat dilutional coagulopathy of severe blood loss and bleeding with loss of clotting factors, and replace lost volume in childhood diseases that cause hypovolemia or severe hypovolemia, treat traumatic brain injury, burns, diagnosis of diseases and methods of making and using said compositions.