In an embodiment of the invention, a composition for treating a cell population comprises an Affinity Medicant Conjugate (AMC). The medicant moiety can be a toxin including an acylfulvene or a drug moiety. The affinity moiety can be an antibody, a binding protein, a steroid, a lipid, a growth factor, a protein, a peptide or non peptidic. The affinity moiety can be covalently bound to the medicant via a linker. Novel linkers that can be directed to cysteine, arginine or lysine residues based on solution pH allow greater flexibility in preserving and/or generating specific epitopes in the AMC.

A hyperbranched polymer includes a hyperbranched, hydrophobic molecular core, respective low molecular weight polyethyleneimine chains attached to at least three branches of the hyperbranched, hydrophobic molecular core, and respective polyethylene glycol chains attached to at least two other branches of the hyperbranched, hydrophobic molecular core. Examples of the hyperbranched polymer may be used to form hyperbranched polyplexes, and may be included in DNA or RNA delivery systems.

The present disclosure generally relates to therapeutic nanoparticles. Exemplary nanoparticles disclosed herein may include about 0.1 to about 40 weight percent of a therapeutic agent and about 10 to about 90 weight percent a diblock poly(lactic) acid-poly(ethylene)glycol copolymer or a diblock poly(lactic)-co-poly(glycolic) acid-poly(ethylene)glycol copolymer, wherein the diblock poly(lactic) acid-poly(ethylene)glycol copolymer comprises poly(lactic) acid having a number average molecule weight of about 30 kDa to about 90 kDa or the diblock poly(lactic)-co-poly(glycolic) acid-poly(ethylene)glycol copolymer comprises poly(lactic)-co-poly(glycolic) acid having a number average molecule weight of about 30 kDa to about 90 kDa.

Poly(amine-co-ester) polymers, methods of forming active agent-load polyplexes and particles therefrom, and methods of using them for delivery of nucleic acid agents with optimal uptake have been developed. Examples demonstrate critical molecular weights in combination with exposed carboxylic and/or hydroxyl groups, and methods of making. Typically, the compositions are less toxic, more efficient at drug delivery, or a combination thereof compared to a control other transfection reagents. In some embodiments, the compositions are suitable for in vivo delivery, and can be administered systemically to a subject to treat a disease or condition. For poly(amine-co-ester) polymers with specific amine or hydroxyl group containing end-groups in admixture with PEGylated poly(amine-co-ester) polymers, in vivo delivery to the lung by inhalation has been shown.

The present disclosure relates to nanoemulsions of 5-amino-[4-(4-chlorobenzoyl)-3, 5-dichlorobenzyl]-1,2,3-triazole-4-carboxamide (carboxy-amido-triazole or CAI), methods of preparing thereof, and their use in the treatment of inflammatory optic neuropathies.

A polymer-prostaglandin conjugate comprising: a polymer backbone comprising a plurality of moieties of formula (I): where: T represents a triazole moiety; Q is independently selected at each occurrence and may be present or absent and when present represents a linking group; R is selected from the group consisting of linear or branched hydrocarbon; D is selected from prostaglandins; and L is a group of formula (II) wherein R.sup.5 is selected from hydrogen and C.sub.1 to C.sub.6 alkyl; (R) indicates the end of the group bonded to the R group; and (D) indicates the end of the group attached to the group D. ##STR00001##

The present invention relates to a nanoparticle comprising a nanomaterial and at least a first ligand and a second ligand tethered to the nanoparticle. The present invention further relates to a nanoparticle for use as a medicament or diagnostic agent. The present invention also relates to a nanoparticle for use in a method of preventing or treating a disease selected from diabetic nephropathy, glomerulonephritis, glomerular VEGF A dysregulation, endothelial VEGF A dysregulation, diabetic retinopathy, rheumatoid arthritis, age-related macular degeneration, and cancer such as breast cancer. Furthermore, the present invention relates to a method of preparing a nanoparticle.

The present disclosure generally provides complexes including a pharmaceutically active agent and a functionalized polymer, wherein the functionalized polymer includes repeat units, the repeat units including ionizable repeat units having at least one ionizable side group and/or ionizable end group, a plurality of the at least one ionizable groups forming non-covalent bonds with the pharmaceutically active agent. Polymers which may be used to form such complexes as well as methods of making and using the complexes and related compositions are also provided.

Provided in the present invention is a bispecific antibody which comprises an anti-MUC1 VHH antibody fragment and an anti-CD16 VHH antibody fragment. The antibody fragment used in the present invention is a variable region sequence derived from a heavy chain camelid antibody and has a high binding affinity to the antigen. Antibody fragments recognizing MUC1 and CD16 are constructed in the same antibody molecule by the invention, so that the antibody molecule can specifically bind to MUC1 and CD16 molecules to promote the killing effect of NK cells on MUC1-positive expression cells and has an inhibiting effect on the growth of MUC1-positive tumors. Also provided is a conjugate of the bispecific antibodies, a related pharmaceutical composition and use.

Methods for synthesizing and purifying oligopeptide-modified poly-beta-amino-esters (OM-PBAEs) and related polymers without using DMSO as a solvent yield OM-PBAEs with improved storage stability in biocompatible buffers. The polymers can be stored for extended periods and used to encapsulate nucleic acids and viral vectors losing transfection or transduction efficiency.