A61K47/549

Short chain ceramide-based lipids and uses thereof

Provided herein, in some aspects, are delivery vehicles comprising a glycosphingolipid and an agent to be delivered attached to the glycosphingolipid. In some embodiments, the glycosphingolipid comprises an oligosaccharide and a short chain (e.g., C0-C3) ceramide. In some embodiments, the agent to be delivered is a therapeutic agent. The glycosphingolipid is able to deliver the agent to a cell or to a cellular compartment, as well as across the musical barrier. In some embodiments, agents delivered using the glycosphingolipid described herein exhibit longer half-life, compared to agents delivered alone. Methods of delivering a therapeutic agent to a subject for treating a disease using the glycosphingolipid delivery vehicle are also provided.

Hydrogel and method for preparing the same
11559476 · 2023-01-24 · ·

The present application provides a method for preparing a hydrogel and the obtained hydrogel. The method including: forming a first part by mixing a first single-stranded nucleotide with a first liposome, and forming a second part by mixing a second single-stranded nucleotide with a second liposome, wherein the first single-stranded nucleotide and the second single-stranded nucleotide have complementary sticky ends; forming a hydrogel by mixing the first part and the second part.

SiRNAs with vinylphosphonate at the 5′ end of the antisense strand

The present invention relates to nucleic acids for inhibiting expression of a target gene in a cell, comprising at least one duplex region that comprises at least a portion of a first strand and at least a portion of a second strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene to be inhibited. The first strand of the nucleic acid has a terminal 5′ (E)-vinylphosphonate nucleotide that is linked to the second nucleotide in the first strand by a phosphodiester linkage.

ENZYMATICALLY SYNTHESIZED OMEGA-3 STRUCTURED PHOSPHOLIPIDS

The invention provides a process of incorporation of omega-3 fatty acids such as EPA/DHA into polar lipid molecules present in lecithin, which consists of: (a) an enzymatic exchange reaction between the fatty acids present in the polar lipids of lecithin and the omega-3 fatty acids present in concentrated fish oil, to obtain an oil with a high content of polar lipids and omega-3 fatty acids and (b) a stage of concentration of the polar lipid content of the oil obtained in stage a, by supercritical fractionation or molecular distillation. The composition of the invention promotes at least a 25% peak incremental concentration in plasma of the EPA/DHA when compared to a krill oil derived composition.

COMPOSITIONS AND METHODS FOR DELIVERY OF NUCLEIC ACIDS TO CELLS
20230227583 · 2023-07-20 · ·

Compositions and methods of use thereof for delivering nucleic acid cargo into cells are provided. The compositions typically include (a) a 3E10 monoclonal antibody or an antigen binding, cell-penetrating fragment thereof; a monovalent, divalent, or multivalent single chain variable fragment (scFv); or a diabody; or humanized form or variant thereof, and (b) a nucleic acid cargo including, for example, a nucleic acid encoding a polypeptide, a functional nucleic acid, a nucleic acid encoding a functional nucleic acid, or a combination thereof. Elements (a) and (b) are typically non-covalently linked to form a complex.

RNA and nucleic acid carrier including the same
11701378 · 2023-07-18 · ·

A nucleic acid carrier according to an embodiment of the present disclosure includes CpG-ODN-RNA conjugate and a porous silica particle carrying the conjugate inside pores thereof. In this regard, the nucleic acid carrier of the present invention can stably deliver loaded nucleic acid molecules to a body and release the same to a target, thereby increasing Type 1 interferon and exhibiting RNA-inherent functions.

PEPTIDE PHARMACEUTICALS FOR TREATMENT OF NASH AND OTHER DISORDERS
20230218564 · 2023-07-13 · ·

The disclosure provides peptide products comprising a peptide covalently attached to a surfactant moiety which have improved properties, including increased duration of action and bioavailability. The peptide products are useful for treating insulin resistance, diabetes, obesity, metabolic syndrome and cardiovascular diseases, and conditions associated therewith, such as NASH and PCOS.

ANTIFUNGAL PRODRUGS

The invention relates to an antifungal prodrug which comprises an antifungal moiety which is linked to a trigger moiety by means of a self-immolative spacer. The trigger moiety is selected from glycosyl residues and oligosaccharides, stabilizes the self-immolative spacer and is cleavable by a pathogen hydrolytic enzyme which is preferably an extracellular glycosidase (EC 3.2.1). When the trigger moiety is cleaved by the pathogen hydrolytic enzyme, the self-immolative spacer undergoes a spontaneous degradation so as to release the antifungal moiety. The invention also relates to pharmaceutical compositions comprising said prodrug and to its use in the treatment of infectious diseases.

SUGAR COMPOUND HAVING POLYETHYLENE GLYCOL CHAIN, AND PRECURSOR OF ANTIBODY-DRUG COMPLEX
20230218766 · 2023-07-13 ·

The object of the present invention is to provide a synthetic method for ADC synthesis with controllable regioselectivity and number of drugs introduced, as well as synthetic intermediates and synthetic raw materials for such methods. The object can be solved by a sugar compound having a polyethylene glycol chain, of the following general formula (1).

##STR00001##

wherein each X is independently a single bond, an oxygen atom, —NH—, —COHN—, —COO—, or a group of formula (5) or (6), (wherein R.sup.1 is trivalent branched hydrocarbon group having 1 to 6 carbon atoms, R.sup.2 and R.sup.3 are each independently an alkylene group having 1 to 3 carbon atoms), any one or more of Y1 to Y3 are present and are independently a PEG chain, a substituted PEG chain, or a PEG chain containing an oxygen atom, —NH—, —COHN—, or —COO— in the main chain, a structure of the PEG chain is linear or branched, and the number of branches is 2 to 10 in the case of branched structure, Z is independently a hydroxy group, a methoxy group, an azido group, a tetrazine group which may be optionally substituted, a norbornene group, a trans-cyclooctene group, a dibenzylcyclooctyl group, or a bicyclo[6.1.0]nona-4-yn-9-ylmethyl group, or a cyanobenzothiazole group which may be optionally substituted, wherein at least one of Z is not a hydroxy group or methoxy group, when any of Y1 to Y3 is not present, Z is hydrogen and X is oxygen, when X is the group of formula (5) or (6), Y1 to Y3 are bonded to each of R.sup.2 and R.sup.3 of the branched chains thereof, when the PEG chain is branched structure, Z is bonded to each of branched chains.

BIVALENT COMPOUNDS, CONJUGATES AND USES THEREOF

There are provided bivalent compounds and conjugates thereof, the conjugates comprising a bivalent compound, a targeting moiety, and a bioactive agent, as well as pharmaceutical compositions and methods of use of the conjugate for the treatment, inhibition, or prevention of diseases and disorders which are therapeutic targets of the bioactive agent.

##STR00001##