The present application relates to aqueous solution compositions of engineered dimeric proteins comprising monomers that comprise at least one human serpin polypeptide operably linked to a human immunoglobulin Fc polypeptide or a polypeptide that is derived from an immunoglobulin Fc polypeptide, at low buffer concentrations and low ionic strength and containing a neutral amino acid. The aqueous solution compositions increase the stability of the an Fc domain in aqueous solution compositions, and in particular increase the stability of an engineered dimeric protein.

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Fusion proteins comprising a protein expression enhancing polypeptide linked to a target protein binding domain and nucleic acid molecules encoding such fusion proteins are described for use in enhancing expression and/or location of a targeted protein of interest, for restoring lost functions in cells, and for treating disease. Additional fusion proteins comprising a target protein of interest modified with a fusion partner comprising a protein expression enhancing polypeptide are also disclosed.

The specification relates to a method of manufacturing a diabetic foot patient-specific skin regeneration sheet, and a diabetic foot patient-specific skin regeneration sheet.

Disclosed herein are compositions and methods useful for treating an alpha-1-antitrypsin deficiency.

The present invention relates to methods for reducing antennary fucosylation of complex N-glycans in recombinantly expressed glycoproteins, cell lines that can be used in said methods, respective recombinant glycoproteins, and methods for expressing the same in said cell lines.

The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

Embodiments herein report compositions of alpha-1 antitrypsin fusion polypeptides or peptide derivatives thereof. In certain embodiments, compositions and methods relate to generating a construct of use in pharmaceutically acceptable compositions to treat a subject in need of alpha-1 antitrypsin therapy or treatment. In other embodiments, compositions and methods disclosed herein concern linking alpha-1 antitrypsin or derivative thereof to an immune fragment.

The invention relates to a new, more potent, coagulation Factor IX (FIX) expression cassette for gene therapy of Haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein, and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.

Disclosed are products and methods for therapy using nucleic acid molecules, and in particular in relation to treatment of alpha-1-antitrypsin deficiency. Also disclosed are pharmaceutical compositions including the nucleic acids and/or delivery vehicles including the nucleic acids, and their use in manufacture of pharmaceutical compositions for use in therapy, such as treatment of alpha-1-antitrypsin deficiency.