The present invention is directed to a pharmaceutical composition including (e.g. for use as an adjuvant) a polymeric carrier cargo complex, comprising as a carrier a polymeric carrier formed by disulfide-crosslinked cationic components; and as a cargo at least one nucleic acid molecule, and at least one antigen that is selected from an antigen from a pathogen associated with infectious disease; an antigen associated with allergy or allergic disease; an antigen associated with autoimmune disease; or an antigen associated with a cancer or tumour disease, or in each case a fragment, variant and/or derivative of said antigen. The pharmaceutical composition allows for efficient induction of an adaptive immune response directed against said antigen. The present invention furthermore provides kits, as well as the use of the pharmaceutical composition or the kit as a vaccine, particularly in the treatment of infectious diseases, allergies, autoimmune diseases and tumour or cancer diseases.

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##

The present disclosure provides, in part, nucleic acid loaded flowable hydrogels and compositions, systems and methods related thereto, to effectively deliver nucleic acids to cells that contact the flowable hydrogels.

This disclosure relates to mRNA therapy for the treatment of Crigler-Najjar Syndrome Type 1 (CN-1). mRNAs for use in the invention, when administered in vivo, encode uridine diphosphate glycosyltransferase 1 family, polypeptide A1 (UGT1A1). mRNA therapies of the disclosure increase and/or restore deficient levels of UGT1A1 expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of bilirubin associated with deficient UGT1A1 activity in subjects.

The present disclosure provides virus-like particles (VLPs) comprising: i) a CRISPR-Cas effector polypeptide; ii) a recombinant lentivirus comprising a nucleotide sequence encoding a therapeutic polypeptide having a length of from about 250 amino acids to about 3,000 amino acids, where the VLP comprises a pseudotyping viral glycoprotein and/or a polypeptide that provides for binding to a target cell. The present disclosure provides systems for producing a VLP. The present disclosure provides methods of delivering a therapeutic protein, using a VLP of the present disclosure.

The present invention includes compositions and methods comprising viral vector systems for multiplexed activation of endogenous genes as immunotherapy and viral-based immune-gene therapy.

The present disclosure relates to AAV gene therapy vectors, AAV replicons, and pharmaceutical compositions for delivering a human HDAC8 gene to a subject for treating Cornelia de Lange Syndrome. In addition, methods of treatment and gene transfer are provided.

The present document describes compounds, or pharmaceutically acceptable salt thereof, of a core formula (I) Wherein R1 includes an amino group. These compounds are particularly useful in the formulation and in vivo and ex vivo delivery of nucleic acid and protein therapeutics for preparing and implementing T cell transfection, gene editing, cancer therapies, cancer prophylactics, and in the preparation of vaccines.

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The present disclosure belongs to the technical field of gene therapy, and specifically relates to a series of lipid compounds as well as lipid vectors, nucleic acid lipid nanoparticle compositions, and pharmaceutical preparations containing the same. The compound having the structure of formula (I) provided by the present disclosure may be used in combination with other lipid compounds to prepare a lipid vector, which exhibits pH responsiveness, has high encapsulation efficiency for nucleic acid drugs, and greatly enhances the in-vivo delivery efficiency of nucleic acid drugs. Moreover, it is possible to select lipid compounds with different structures as lipid vectors to adjust the enrichment of nucleic acid drugs in different organs, thereby having good market application prospect.

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The present invention relates to adeno-associated virus (AAV) vectors modified by the covalent coupling of at least one compound comprising a lactam moiety (e.g., β-lactam) to at least one amino group of an amino acid residue of the capsid of the AAV vectors. The AAV vectors are useful in transducing a cell, especially for gene therapy.