Takeda Pharmaceutical Company Limited

Werner Hoellriegl

Hanspeter Rottensteiner

Barbara Plaimauer

Eva-Maria Muchitsch

Alexandra Nathalie Kopic

The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.

Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression

This invention relates to methods of subcutaneous administration of ADAMTS13 formulations to a treat a disease or condition associated with ADAMTS13 and VWF dysfunction. Furthermore, evidence of the unexpectedly high bioavailability of ADAMTS13 formulations administered subcutaneously is provided herein.

Growing community of inventors

Altenmarkt/Triesting, Austria

Werner Hoellriegl