The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.

This invention provides for a compound of formula (I):

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or a pharmaceutically acceptable salt thereof, wherein R.sup.1-R.sup.4, L.sub.1, n and p are defined herein. The compounds of formula (X) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

This invention provides for a compound of formula (I):

##STR00001##

or a pharmaceutically acceptable salt thereof, wherein R.sup.1-R.sup.4, L.sub.1, n and p are defined herein. The compounds of formula (X) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

The disclosure provides ionizable amine lipids and salts thereof (e.g., pharmaceutically acceptable salts thereof) useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells. The ionizable amine lipids disclosed herein are useful as ionizable lipids in the formulation of lipid nanoparticle-based compositions.

The disclosure provides ionizable amine lipids and salts thereof (e.g., pharmaceutically acceptable salts thereof) useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells. The ionizable amine lipids disclosed herein are useful as ionizable lipids in the formulation of lipid nanoparticle-based compositions.

This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R.sup.1-R.sup.4, n and p are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues. ##STR00001##

This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R.sup.1-R.sup.4, n and p are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues. ##STR00001##

The present invention relates to long-chain dimethylaniline derivative compounds, their preparation methods, self-assembled texture, and uses thereof. Said compounds show ultralong acting anaesthetic effect, and belong to N-diethylaminoacetyl-2,6-dimethylaniline compounds, having a structure according to formula (I). Said compounds may self-assemble into micelle or gel in an aqueous solvent, and exert an in vivo elongated local anesthetic actions. Local anesthesia and/or analgesic activity can last more than 72 hours. The biomaterials can self-assemble into micelles or gels in water and have local anesthetic effect, as well as can further be used as the coating of medicinal bioactive molecules for treatment of pain, itching and the like and/or the drug carriers, together with as pharmaceutical adjuvants for delivery system. Thus, said materials have a favorable perspective. ##STR00001##

A composition and method of treatment of neuromuscular, neuromuscular degenerative, neurodegenerative, autoimmune, developmental, traumatic, hearing loss related, and/or metabolic diseases, including spinal muscular atrophy (SMA) syndrome (SMA1, SMA2, SMA3, and SMA4, also called Type I, II, III and IV), traumatic brain injury (TBI), concussion, keratoconjunctivitis sicca (Dry Eye Disease), glaucoma, Sjogren's syndrome, rheumatoid arthritis, post-LASIK surgery, anti-depressants use, Wolfram Syndrome, and Wolcott-Rallison syndrome. The composition is selected from the group consisting of 2,3-DNP, 2,4-DNP, 2,5-DNP, 2,6-DNP, 3,4-DNP, or 3,5-DNP, bipartite 2,3-dinitrophenol, 2,4-dinitrophenol, 2,5-dinitrophenol, 2,6-dinitrophenol, 3,4-dinitrophenol, or 3,5-dinitrophenol (2,3-DNP, 2,4-DNP, 2,5-DNP, 2,6-DNP, 3,4-DNP, or 3,5-DNP) prodrugs; Gemini prodrugs, bioprecursor molecules, and combinations thereof. A dose of the composition for treatment of neurodegenerative diseases may be from about 0.01 mg/kg of body weight to about 50 mg/kg of body weight of the patient in need of treatment. A dose of the composition for treatment of metabolic diseases may be from about 1 mg/70 kg of body weight to about 100 mg/70 kg of body weight of the patient in need of treatment, and a maximum dose per day is about 200 mg/70 kg of body weight of the patient in need of treatment.

The present invention relates to long-chain dimethylaniline derivative compounds, their preparation methods, self-assembled texture, and uses thereof. Said compounds show ultralong acting anaesthetic effect, and belong to N-diethylaminoacetyl-2,6-dimethylaniline compounds, having a structure according to formula (I). Said compounds may self-assemble into micelle or gel in an aqueous solvent, and exert an in vivo elongated local anesthetic actions. Local anesthesia and/or analgesic activity can last more than 72 hours. The biomaterials can self-assemble into micelles or gels in water and have local anesthetic effect, as well as can further be used as the coating of medicinal bioactive molecules for treatment of pain, itching and the like and/or the drug carriers, together with as pharmaceutical adjuvants for delivery system. Thus, said materials have a favourable perspective.

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