The subject of the present invention is a lipidoid of general formula (I), wherein X, Y, Z and R are as defined in the claims. This lipidoid is useful as a transfection agent. The invention further describes transfection agents, transfection particles containing this lipidoid, and their use.

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The present invention relates to an A1CF inhibitor for use in treatment of an HBV infection, in particular a chronic HBV infection. The invention in particular relates to the use of A1CF inhibitors for destabilizing cccDNA, such as HBV cccDNA. The invention also relates to nucleic acid molecules which are complementary to A1CF and capable of reducing the level of an A1CF mRNA. Also comprised in the present invention is a pharmaceutical composition and its use in the treatment of a HBV infection.

Provided herein are compositions of lipid-based nanoparticles, such as exosomes, that comprise a therapeutic anti-aging agent. Also provided are methods of using such compositions to treat a patient having an age-associated disorder. In particular, exosomes comprising a telomerase-encoding RNA are provided along with methods of their use in treating age-associated disorders.

Provided herein are, in various embodiments, methods and compositions of inducing M2-like macrophage morphology. In certain embodiments, a composition comprising a polynucleotide encoding a ring finger protein 41 (RNF41) is contemplated. The disclosure also provides a method of preventing, treating, managing, and/or ameliorating tissue damage in a subject in need thereof. In some embodiments, the subject has chronic liver disease, chronic liver inflammation, chronic hepatic fibrosis, cirrhosis, or a combination thereof. In still further embodiments, the subject has undergone liver resection or liver transplantation.

Improved formulations of lipid nanoparticles are provided. Use of the lipid nanoparticles for delivery of a therapeutic agent and methods for their preparation are also provided.

Disclosed herein are an mRNA targeting molecule comprising an N-acetylgalactosamine binding polypeptide and a preparation method therefor. A plasmid vector containing a DNA fragment formed by sequentially connecting a promoter, a target gene, a specific protease cleavage sequence, and a polynucleotide sequence encoding a GBD capable of binding to N-acetylgalactosamine, is transcribed to obtain an mRNA, which is connected to a DNA-puromycin linker under the action of a T4 ligase. The resulting connection product is subjected to protein translation, followed by cleavage using a specific protease to obtain an mRNA-puromycin-GBD complex, which then binds to a GBD protein sequence under the action of an N-acetylgalactosamine transferase to form an mRNA-puromycin-GBD-GalNAc complex, thereby modifying the mRNA with GalNAc, thus achieving the purpose of precise administration in a process of mRNA drug delivery and increasing the efficacy of the mRNA drug molecule.

Provided herein are polypeptides comprising one or more non-native cysteine residues that form a disulfide bridge between non-native cysteines within the protein or between non-native cysteines of two monomers of the protein. Such modified human polypeptides are useful in treatment of genetic diseases via enzyme replacement therapy and/or gene therapy.

Nucleotides encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein are provided herein. Also describe are mRNA constructs that can be used to express CFTR protein in vitro or in vivo. The mRNA constructs can be formulated in a lipid formulation and administered via inhalation to treat cystic fibrosis.

An environmentally sensitive membrane binding polypeptide, pH (low)-sensitive membrane peptide (pHLIP) has improved insertion kinetics balanced with solubility to selectively target acidic tissues.

The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.