Abstract: This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA or gene editing therapeutics for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects. Nucleic acid therapeutics (e.g., mRNAs) of the disclosure further decrease abnormal accumulation of ammonia associated with deficient CFTR activity in subjects.

Abstract: The present disclosure provides LNPs comprising payload molecules, e.g., mRNA therapeutics, for the treatment of diseases or disorders, which would benefit from delivery of payload molecules to airway cells.

Abstract: The present invention generally relates to particles, including nanocapsules or other nanoentities, comprising a polymer such as polysialic acid. The particles are able to access the interior of the cells, and/or to procure the intracellular release of the associated drugs. In 5 one aspect, the present invention is directed to nanocapsules or other entities having an exterior or surface comprising a polymer such as polysialic acid. In some cases, targeting moieties such as Lyp-1 or tLyp-1 peptide are bonded to the polymer, e.g., using aminoalkyl (C1-C4) succinimide or other linkers. These may be created, for example, by reacting a carboxylate moiety on a polymer with an aminoalkyl maleimide (C1-C4) or an aminoalkyl 10 (C1-C4) methacrylamide, and reacting the resulting aminoalkyl (C1-C4) maleimide or the aminoalkyl (C1-C4) methacrylamide to a cysteine or other sulfur group.

Abstract: The present invention generally relates to particles, including nanocapsules or other nanoentities, comprising a polymer such as polysialic acid. The particles are able to access the interior of the cells, and/or to procure the intracellular release of the associated drugs. In 5 one aspect, the present invention is directed to nanocapsules or other entities having an exterior or surface comprising a polymer such as polysialic acid. In some cases, targeting moieties such as Lyp-1 or tLyp-1 peptide are bonded to the polymer, e.g., using aminoalkyl (C1-C4) succinimide or other linkers. These may be created, for example, by reacting a carboxylate moiety on a polymer with an aminoalkyl maleimide (C1-C4) or an aminoalkyl 10 (C1-C4) methacrylamide, and reacting the resulting aminoalkyl (C1-C4) maleimide or the aminoalkyl (C1-C4) methacrylamide to a cysteine or other sulfur group.