A61K9/5123

AAV-mediated delivery of therapeutic antibodies to the inner ear

Provided herein are methods that include introducing into an inner ear of a mammal a therapeutically effective amount of an adeno-associated virus (AAV) vector that includes a nucleotide sequence encoding (a) a polypeptide including an antibody heavy chain variable domain operably linked to a signal peptide and a polypeptide including an antibody light chain variable domain operably linked to a signal peptide; (b) a polypeptide including an antigen-binding antibody fragment operably linked to a signal peptide; or (c) a soluble vascular endothelial growth factor receptor operably linked to a signal peptide.

PARTICLE CONTAINING LIPID NANOPARTICLES AND METHOD FOR PRODUCING SAME

A particle includes at least one or more kinds of substrate and lipid nanoparticles. The lipid nanoparticles are dispersed in the substrate and contain a physiologically active substance. The lipid nanoparticles are one or more kinds selected from liposomes, lipid emulsions, and solid lipid nanoparticles. A corresponding powder inhalant contains the particle. A production method for the particle includes granulating and drying, in which a suspension containing the substrate and the lipid nanoparticles are granulated and dried in a gas medium.

Nanoparticle vaccine adjuvant and methods of use thereof

Non-liposome, non-micelle particles formed of a lipid, an additional adjuvant such as a TLR4 agonist, a sterol, and a saponin are provided. The particles are porous, cage-like nanoparticles, also referred to as nanocages, and are typically between about 30 nm and about 60 nm. In some embodiments, the nanocages include or are administered in combination with an antigen. The particles can increase immune responses and are particularly useful as adjuvants in vaccine applications and related methods of treatment. Preferred lipids, additional adjuvants including TLR4 agonists, sterols, and saponins, methods of making the nanocages, and method of using them are also provided.

Delivery of target specific nucleases

Described herein are lipid nanoparticles comprising cationic lipids and other lipids and also comprising engineered nucleases facilitate transfer of nucleic acids to cells.

Tumor pH-shiftable coating and the nucleus-directed nanoparticles facilitate the targeted chemotherapy and gene therapy against multiple cancers and use thereof
11690803 · 2023-07-04 · ·

At present, there is a great need for the development of new tumor pH-shiftable nanoparticles that are effective to reduce side effects, enhance active tumor focusing, improve the cellular uptake, and nuclear/cytoplasmic targeting of chemotherapy and gene therapeutic. Hence, we designed novel solid lipid nanoparticles (SLN) and liposomes (Lip) to deliver microRNA and antineoplastic agent, respectively. The designed SLN and liposomes incorporating microRNA and anticancer drugs in the core, which is surrounded by lipids modified with peptide T (a ligand plus a cell-penetrating peptide) and a nucleus-targeted sequence of peptide R as the inner shell. Moreover, coating a pH-responsive polymer (PGA-PEG) on the outer layer of Lip-TR (PGA-Lip-TR) and SLN-T (PGA-SLN-T) can protect the peptide T and R from degradation by peptidases during systemic circulation and enhance directing to the acidic tumor sites. Collectively, these pH-shiftable nanoparticles may provide a novel and potential strategy for anticancer therapy.

Lipid compound as well as lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing same

The present invention belongs to the technical field of gene therapy, and particularly relates to a series of lipid compounds as well as a lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing the same. A compound having a structure of a formula (I) provided by the present invention can be used for preparing a lipid carrier together with other lipid compounds, and exhibits pH response, and the entrapment efficiency to a nucleic acid drug is high, which greatly improves in-vivo delivery efficiency of the nucleic acid drug; and furthermore, a lipid compound with a specific structure can be chosen as a lipid carrier based on an organ in which the nucleic acid drug needs to be enriched, having a good market application prospect. ##STR00001##

OPIOID INDEPENDENT SURGICAL ANESTHETIC

An opioid independent surgical anesthetic composition includes an injectable dosage form of a hydrogel having a plurality of solid lipid matrix particles entrapped therein. The solid lipid matrix particles include a lipophilic local anesthetic drug and a lipid glyceride (e.g., saturated triglyceride or lipid blend of various lipid glycerides). Methods for creating a long-acting local anesthetic product can include creating a bulk solid of a lipid matrix product by heating a lipid solvent above its melting point, dissolving a lipophilic local anesthetic drug therein, reducing a temperature of the resultant drug-lipid solution to below the melting point of the lipid solvent, and heat annealing the lipid matrix to remove or reduce presence of any unstable polymorphs in the lipid matrix. The methods can further include crushing the bulk solid of the lipid matrix product to form solid lipid matrix particles and entrapping the solid lipid matrix particles within a hydrogel.

LIPID NANOPARTICLE FORMULATIONS AND COMPOSITIONS
20250228966 · 2025-07-17 ·

Disclosed are compositions of lipid nanoparticles (LNP) comprising an ionizable cationic lipid, a phospholipid, a sterol, and a PEG-lipid (non-functionalized and optionally functionalized). The functionalized PEG-lipid can be conjugated with a binding moiety to create a targeted LNP (tLNP). The disclosed tLNP preferentially deliver a nucleic acid molecule or other negatively charged payload to cells expressing a cell surface antigen recognized by the binding moiety of the tLNP, and are better tolerated, as compared to LNPs and tLNPs comprising ionizable cationic lipids found in marketed pharmaceuticals comprising LNPs.

Pharmaceutical composition comprising nanoperforator for preventing or treating viral infectious diseases

The present invention relates to a nanoperforator having a lipid-bilayer nanodisc and a membrane-structured protein surrounding the nanodisc and to a pharmaceutical composition having the nanoperforator as an active ingredient for preventing or treating viral infectious diseases. The use of the lipid-bilayer nanoperforator provided in the present invention can lead to the safe prevention or treatment of a disease caused by viral infection regardless of whether the virus is a variant or not, and thus the present invention can be widely used for the safe and effective treatment of viral infectious diseases.

Lipids for use in lipid nanoparticle formulations
11542225 · 2023-01-03 · ·

Compounds are provided having the following structure (I) or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein G.sup.1, G.sup.1′, G.sup.2, G.sup.2′, G.sup.3, L.sup.1, L.sup.1′, L.sup.2, L.sup.2′, X, X′, Y and Y′ are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided. ##STR00001##