Baxalta Gmbh

Takeda Pharmaceutical Company Limited

Franziska Horling

Friedrich Scheiflinger

Hanspeter Rottensteiner

Johannes Lengler

Falko-Günter Falkner

Michael Dockal

Ernst Böhm

Meinhard Hasslacher

Christian Fiedler

Falko-Gunter Falkner

Thomas Gatternig

Jadranka Koehn

Ernst Bohm

Emst Bohm

Ernst BÖHM

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.

Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B

Methods and systems for activating Factor X are disclosed.

Factor X activation

Disclosed herein are methods for production of fully-processed mature Factor X in an expression system producing a controlled amount of furin between 50 U/mL and 300 U/mL of culture supernatant. Also disclosed are transformed cells, expression systems, and expression vectors for the expression of furin and Factor X.

Production of fully processed and functional factor X in a furin-secreting mammalian expression system

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Gaenserndorf, Austria

Franziska Horling