The present invention provides compositions for the treatment of phenylketonuria in mammals, as well as methods of preparing said compositions. The present invention also provides methods of treating phenylketonuria using the compositions of the invention.

The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human β-globin protein or a human γ-globin protein, which is oriented from 5′ to 3′ relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.

The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human β-globin protein or a human γ-globin protein, which is oriented from 5′ to 3′ relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.

The invention discloses a hemostatic composition comprising: a) a biocompatible polymer in particulate form suitable for use in hemostasis, and b) one hydrophilic polymeric component comprising reactive groups.

The invention discloses a hemostatic composition comprising: a) a biocompatible polymer in particulate form suitable for use in hemostasis, and b) one hydrophilic polymeric component comprising reactive groups.

The present invention relates to a method of treating HER2/NEU overexpressing cancers. The inventors discovered that the heme-mediated formation of dimers and in general oligomers of Trastuzumab is associated with an improved complement-mediated cytotoxicity on breast cancer cells. The present data highlight that the sensitivity to heme of Trastuzumab, may have major repercussion on its therapeutic activity. Thus the invention relates to the combination of an HER2/neu antibody with a heme and/or of its oligomers and its therapeutic composition in the HER2/NEU characteristic cancer treatment.

The present invention relates to a method of treating HER2/NEU overexpressing cancers. The inventors discovered that the heme-mediated formation of dimers and in general oligomers of Trastuzumab is associated with an improved complement-mediated cytotoxicity on breast cancer cells. The present data highlight that the sensitivity to heme of Trastuzumab, may have major repercussion on its therapeutic activity. Thus the invention relates to the combination of an HER2/neu antibody with a heme and/or of its oligomers and its therapeutic composition in the HER2/NEU characteristic cancer treatment.

The present invention relates to a process for producing a modified nucleic acid, wherein the nucleic acid comprises a mutant haemoglobin B (HBB) gene encoding a mutant Hb-β polypeptide. The process comprises using a base editor, preferably with a gRNA, to edit the mutant HBB gene to change a first (mutant) codon in that gene into a second, non-wild-type codon, wherein the Hb-β polypeptide encoded by that edited HBB gene has a non-wild-type, yet phenotypically-viable, amino acid sequence. The invention also provides a population of isolated haematopoietic stem cells, the stem cells comprising edited HBB genes.

The present invention provides an enteric-coated hemoglobin multiparticulate comprising a core, a hemoglobin formulation coating, an inner or sub-coating, and an enteric coating. The present invention also provides a method of preparing said enteric-coated hemoglobin multiparticulate. The present invention further provides a method for treating various diseases caused by oxygen deficiency comprising administering to a subject said enteric-coated hemoglobin multiparticulate in order to orally deliver the encapsulated hemoglobin-based oxygen carriers to a specific target of said subject in a controlled release manner.

The present invention provides an enteric-coated hemoglobin multiparticulate comprising a core, a hemoglobin formulation coating, an inner or sub-coating, and an enteric coating. The present invention also provides a method of preparing said enteric-coated hemoglobin multiparticulate. The present invention further provides a method for treating various diseases caused by oxygen deficiency comprising administering to a subject said enteric-coated hemoglobin multiparticulate in order to orally deliver the encapsulated hemoglobin-based oxygen carriers to a specific target of said subject in a controlled release manner.