Provided herein include compositions, methods and systems for delivery of CRISPR/Cas-mediated gene editing systems using lipid nanoparticles (LNP) to trabecular meshwork cells. Methods, compositions and systems for treating glaucoma are also provided herein, which involve reducing the expression of myocilin (MYOC) gene in the trabecular meshwork cells of patients' eyes.

The present disclosure provides a lipid nanoparticle comprising: a nucleic acid cargo molecule; sterol or a derivative thereof present at elevated content; neutral lipid; an ionizable lipid; and a hydrophilic polymer-lipid conjugate present at a content between 0.5 and 3 mol %, wherein each mol % content is relative to total lipid present in the lipid nanoparticle.

Methods for targeted delivery of therapeutic agents to a target cell or tissue with lipid nanoparticles comprising ApoE3. In embodiments, the invention specifically relates to targeted delivery of anticancer drugs, antibiotics, antifungal drugs, and diagnostic contrast agents, and associated treatment and diagnostic methods. In embodiments, diseases/conditions treated include those associated with over-expression of LDL receptors.

Disclosed is a nanocarrier-containing immunosuppressive agent that is targeted to C3 breakdown products, integrin, or a combination thereof, to reduce the deleterious systemic effects of the immunosuppressive agent. Also disclosed is a method for suppressing an allo-immune response in a subject, such as one that can occur after an allograft transplantation.

The present invention provides compositions comprising nucleic acid molecules, such as mRNA molecules, encapsulated within lipid particles. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease.

The present invention relates to sterile injectable compositions comprising drug-phospholipid micelles. The present invention also relates to processes for the preparation of the sterile injectable compositions.

The present invention relates to reverse-micellar systems comprising at least an active agent, an acylglycerol, a sterol, lecithin, ethanol and water, for use in chelation and/or sequestering of a radionuclide and/or a metal in a patient. The invention also relates to the reverse-micellar systems and to pharmaceutical compositions comprising said reverse-micellar systems.

A micelle, comprises a first phospholipid, a second phospholipid, a targeting agent, conjugated to the first phospholipid, a perfluorocarbon, and a therapeutically active compound. The first phospholipid and the second phospholipid form a shell enclosing the perfluorocarbon and the therapeutically active compound. The targeting agent comprises an anti-nucleolin agent, and the therapeutically active compound comprises a chemotherapeutic agent and/or a cytotoxic agent. An emulsion may be formed, comprising a plurality of the micelles, and continuous aqueous phase. A pharmaceutical composition for treating cancer may be prepared, comprising the emulsion, and a pharmaceutically acceptable carrier. A method of treating cancer includes administering an effective amount of the pharmaceutical composition to a patient in need thereof.

The invention creates engineered surface protein expression on vesicles for specific targeting and delivery of agents to autophagic and apoptotic cells. Moreover, the vesicles of the invention can achieve a synergistic effect on the targeting and drug delivery to autophagic and apoptotic cells and autophagic and apoptotic cells-containing tissues.

The present disclosure provides a lipid nanoparticle comprising: a nucleic acid cargo molecule; sterol or a derivative thereof present at elevated content; neutral lipid; an ionizable lipid; and a hydrophilic polymer-lipid conjugate present at a content between 0.5 and 3 mol %, wherein each mol % content is relative to total lipid present in the lipid nanoparticle.