Low-generation dendrimers containing a high density of surface hydroxyl groups, and methods of synthesis thereof are provided. In particular, oligo ethylene glycol (OEG)-like dendrimers with a high surface functional groups at relatively low generations (e.g. 120 hydroxyls in the third generation, with a size of just 1-2 nm) is described. Dendrimer formulations including one or more prophylactic, therapeutic, and/or diagnostic agents, and methods of use thereof are also described. The formulations are suitable for topical, enteral, and/or parenteral delivery for treating one or more diseases, conditions, and injuries in the eye, the brain and nervous system (CNS), particularly those associated with pathological activation of microglia and astrocytes.

Provided herein are bioactive molecule conjugates, preparation methods and use thereof, particularly bioactive molecule conjugates (such as antibody-drug conjugates and small molecule drug conjugates) obtained by improved coupling of a drug and a targeting moiety, as well as their preparation method and use for the treatment of a disease associated with an abnormal cell activity.

The present disclosure relates generally to antibody-drug conjugates comprising peptide-containing linkers and to methods of using these conjugates as therapeutics and/or diagnostics. Also disclosed herein are peptide-containing scaffolds useful to conjugate with a targeting moiety (e.g., an antibody), a drug, or both to produce the antibody-drug conjugates.

Described herein are silicon based conjugates capable of delivering one or more payload moieties to a target cell or tissue. Contemplated conjugates may include a silicon-heteroatom core, one or more optional catalytic moieties, a targeting moiety that permits accumulation of the conjugate within a target cell or tissue, one or more payload moieties (e.g., a therapeutic agent or imaging agent), and two or more non-interfering moieties covalently bound to the silicon-heteroatom core.

Low-generation dendrimers containing a high density of surface hydroxyl groups, and methods of synthesis thereof are provided. In particular, oligo ethylene glycol (OEG)-like dendrimers with a high surface functional groups at relatively low generations (e.g. 120 hydroxyls in the third generation, with a size of just 1-2 nm) is described. Dendrimer formulations including one or more prophylactic, therapeutic, and/or diagnostic agents, and methods of use thereof are also described. The formulations are suitable for topical, enteral, and/or parenteral delivery for treating one or more diseases, conditions, and injuries in the eye, the brain and nervous system (CNS), particularly those associated with pathological activation of microglia and astrocytes.

The present invention provides antagonizing antibodies that bind to complement factor D (CFD), conjugates thereof, and methods of using same. The anti-CFD antibodies can be used therapeutically alone or in combination with other therapeutics to treat age related macular degeneration and other diseases.

A macromolecule includes i) a dendrimer comprising a core and at least one generation of building units, the outermost generation of building units having surface amino groups wherein at least two different terminal groups are covalently attached to the surface amino groups of the dendrimer, ii) a first terminal group which is a residue of a pharmaceutically active agent comprising a hydroxyl group, and iii) a second terminal group which is a pharmacokinetic modifying agent. The pharmaceutically active agent is cabazitaxel. The first terminal group is covalently attached to the surface amino group of the dendrimer through a diacid linker, the diacid linker comprising an alkyl chain interrupted by one or more oxygen, sulfur or nitrogen atoms, or a pharmaceutically acceptable salt thereof.

The present invention provides Ligand-Drug Conjugates comprising a PEG Unit in a parallel orientation to the Drug Unit. The invention provides inter alia, Ligand-Drug Conjugates (LDCs), methods of preparing and using them, and intermediates thereof. The Ligand-Drug Conjugates are stable in circulation, yet capable of inflicting cell death on targeted cells or inhibiting proliferation of targeted cells once its drug cargo is released in the vicinity or within targeted cells. In principle embodiments, an LDC of the present invention is represented by the structure of Formula I.

A macromolecule includes i) a dendrimer comprising a core and at least one generation of building units, the outermost generation of building units having surface amino groups wherein at least two different terminal groups are covalently attached to the surface amino groups of the dendrimer, ii) a first terminal group which is a residue of a pharmaceutically active agent comprising a hydroxyl group, and iii) a second terminal group which is a pharmacokinetic modifying agent. The pharmaceutically active agent is cabazitaxel. The first terminal group is covalently attached to the surface amino group of the dendrimer through a diacid linker, the diacid linker comprising an alkyl chain interrupted by one or more oxygen, sulfur or nitrogen atoms, or a pharmaceutically acceptable salt thereof.

Provided herein is dendrimer-targeting agent conjugate comprising: (a) a dendrimer comprising i) a core unit (C); and ii) building units (BU), wherein the dendrimer has from two to six generations of building units; and wherein the core unit is covalently attached to at least two building units; and the dendrimer further comprising: b) a targeting agent which is covalently linked to the dendrimer by a spacer group; c) one or more first terminal groups attached to an outermost building unit of the dendrimer, wherein the first terminal group comprises a complexation group for complexing a radionuclide; and d) one or more second terminal groups attached to an outermost building unit of the dendrimer, wherein the second terminal group comprises a pharmacokinetic-modifying moiety, or a salt thereof. Also provided are compositions comprising the dendrimer-targeting agent conjugates, and methods of using the dendrimer-targeting agent conjugates and compositions comprising them in therapeutic and imaging applications.