The compounds disclosed herein compound of Formula (I), substructures thereof, and pharmaceutically acceptable salts thereof. The compounds 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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Described herein are compositions and techniques related to generation and therapeutic application of artificial synapses. Artificial synapses are engineered extracellular vesicles, including exosomes, which incorporate sticky binders on their surface to anchor signaling domains against biological targets, such as receptors. These engineered additives can be organized in genetic vector constructs, expressed in mammalian cells, wherein the sticky binders attach to extracellular vesicles such as exosomes, thereby presenting their joined signaling domains which are rapidly taken up by recipient cells. Artificial synapses adopt the hallmark biophysical and biochemical features of extracellular vesicles, allowing for rapid deployment and scale-up. Importantly, this strategy can allow for kinetically favorable signal generation and signal propagation. This includes, for example, increasing density of agonist presentation to support receptor clustering—an onerous barrier for traditional receptor targeting strategies.

Methods for inducing an immune response against tau protein in a subject suffering from a neurodegenerative disease, disorder or condition, such as Alzheimer's Disease, are described. The methods include administering a liposomal priming composition containing tau peptides, preferably phosphorylated tau peptides, and a conjugate boosting composition containing tau peptides, preferably phosphorylated tau peptides, conjugated to an immunogenic carrier.

The disclosure describes nanocarried and/or microcarried jasmonate compounds and their pharmaceutical compositions, as well as use thereof for treating or preventing angiogenesis-related or NF-κB-related disorders. Also disclosed are methods of making the nanocarried and/or microcarried compounds and their compositions.

The invention provides compositions including stabilized multilamellar lipid vesicles having crosslinked lipid bilayers (referred to herein as interbilayer-crosslinked multilamellar vesicles or ICMV) and including an ALK variant, pharmaceutical compositions containing vesicles (e.g., ICMV) including an ALK variant, and methods of treatment using such compositions. The invention provides compositions including stabilized multilamellar lipid vesicles with crosslinked lipid bilayers (e.g., an interbilayer-crosslinked multilamellar vesicle or ICMV) containing an Anaplastic lymphoma kinase (ALK) variant as an antigen that is associated with solid tumor cancers.

The present invention provides compositions and methods for treating or preventing an NLRP3 immunosome-related disorder. In one embodiment, the method includes administering a therapeutically effective amount of a composition comprising at least one NLRP3 inflammasome inhibitor to a subject in need thereof.

Targeting or binding peptides to neutrophil elastase for specific targeting of activated neutrophils includes amino acid sequences substantially similar to a reactive center loop portion of alpha-1 anti-trypsin (AAT).

The present disclosure provides, among other things, polynucleotide constructs, compositions, and methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need thereof a composition comprising a polynucleotide construct comprising a 5′ UTR, a codon optimized mRNA encoding an ornithine transcarbamylase, and a 3′ UTR.

Described herein is a nanoparticle system including a multivalent nanoparticle core having a plurality of β-hairpin peptides conjugated thereto. Also included are pharmaceutical compositions and methods of making the nanoparticle system. Further included are immunotherapy methods including administering the nanoparticle system to a subject in need thereof, such as a human cancer patient.

Described herein are ultra-deformable liposomes comprising a first lipid and second lipid. The first lipid comprises a first hydrophilic head linked to a first aliphatic tail, and the second lipid comprises a second hydrophilic head linked to a second aliphatic tail having at least two carbons less than the first aliphatic tail. The ultra-deformable liposomes described herein are useful, for example, as drug delivery vehicles. Accordingly, also described herein are compositions comprising an ultra-deformable liposome and a cargo, such as a drug, as well as methods for delivering a drug, such as an anti-cancer therapeutic, to a tumor.