Baxalta Gmbh

Takeda Pharmaceutical Company Limited

Johannes Lengler

Friedrich Scheiflinger

Hanspeter Rottensteiner

Franziska Horling

Falko-Günter Falkner

Falko-Gunter Falkner

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

Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression

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.

Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A

Codon-altered polynucleotides encoding Factor IX variants for expression in mammalian cells are provided, including sequences with high nucleotide sequence identity to the CS02, CS03, CS04, CS05, and CS06 sequences. Mammalian gene therapy vectors, such as adeno-associated viral vectors, and methods for treating hemophilia B using those vectors are also provided.

Growing community of inventors

Vienna, Austria

Johannes Lengler