Disclosed are cationic lipids which are compounds of Formula (A). Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

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Disclosed are the use of one or more lipid compounds in the delivery of nucleic acids, and a lipid-nucleic acid mixture, a pharmaceutical composition or a kit comprising the lipid compound and nucleic acids. The lipid compounds provided in the present invention can promote the absorption of nucleic acids, particularly via oral absorption, and can promote the entry of the nucleic acids at target sites in a subject in need thereof.

The present invention relates to methods and compositions for the treatment of hemangioma, and particularly, but not exclusively, methods and compositions for the treatment of infantile hemangioma. In certain aspects, the methods comprise locally administering an ACE inhibitor or an ATIIR2 antagonist to a subject. In other aspects, the methods comprise systemically administering two or more of an ACE inhibitor, a beta-blocker and an ATIIR2 antagonist. The present invention also relates to compositions that are suitable for local administration and comprise: an ACEi and a beta-blocker; an ACEi and an ATIIR2 antagonist; a beta-blocker and an AT11R2 antagonist; or, an ACEi, a beta-blocker, and an AT11R2 antagonist.

The present invention provides an mRNA molecule encoding at least one KRAS variant peptide. Further, the invention provides a pharmaceutical composition and kit comprising the mRNA molecule. The mRNA molecule, pharmaceutical composition and kit are useful for treating cancer.

Liposomes which comprise sphingomyelin in the lipid bilayer. The liposomes are configured to cross the blood-brain barrier for the treatment of neuro-degenerative diseases and spinal cord injuries. The liposomes are essentially free of ganglioside. A method of producing liposomes is also disclosed along with use of liposome as a medicament.

A topical composition comprising an aqueous-based composition comprising cannabinoids, in particular cannabidiol, in niosomes having size lower than 500 nm, and at least one topically acceptable excipient, characterized in that said niosomes comprise (i) at least one linear or branched polyglycerol, esterified with saturated or monounsaturated linear fatty acids, (ii) at least one polysaccharide, and optionally (iii) at least one glycol having 4 to 16 carbon atoms.

Pharmaceutical compositions and stable nano-suspensions comprising mifepristone and at least one pharmaceutically acceptable excipient, which exhibit enhanced bioavailability compared to the currently marketed or commercially available formulations. Manufacturing process and methods of use are also provided. The pharmaceutical compositions are used for prevention, treatment or prophylaxis of disorders in human patients in need thereof. Oral pharmaceutical compositions of mifepristone, methods for their administration, processes for thei r production, and use of these compositions are described for the treatment of diseases for which mifepristone is indicated.

A novel transgene for use to produce a coronavirus vaccine is provided. The transgene encodes: i) an RNA polymerase promoter; ii) a 5′ UTR; iii) a secretory sequence; iv) a coronavirus spike protein component, wherein the spike protein component incorporates a variant sequence at amino acid position 614 of a native spike protein; and v) a 3′ UTR and poly A sequence. A vaccine is also provided comprising the transgene or an mRNA transcript thereof.

The present disclosure provides catanionic surfactant vesicles (SVs). The vesicles may be functionalized on their outer leaflet such that they may be biologically active. The vesicles may encapsulate (at least partially in the lumen and/or at least partially in the leaflet) one or more small molecules, one or more RNAs, one or more DNAs, and/or one or more proteins/peptides. Also provided are compositions comprising the vesicles (e.g., vaccine compositions comprising the vesicles) and methods of making and using the same.

The disclosure features lipid nanoparticle (LNP) compositions comprising metabolic reprogramming molecules and uses thereof. The LNP compositions of the present disclosure comprise mRNA therapeutics encoding metabolic reprogramming polypeptides, e.g., IDO, TDO, AMPK, AhR, ALDH1A2, HMOX1, CD73 or CD39. The LNP compositions of the present disclosure can reprogram myeloid and/or dendritic cells, suppress T cells and/or induce immune tolerance in vivo.