Sangamo Biosciences, Inc.

Bioverativ Therapeutics Inc.

Sangamo Therapeutics, Inc.

Biogen Ma Inc.

Siyuan Tan

Fyodor Urnov

Philip D Gregory

David Paschon

Lei Zhang

Edward J Rebar

Jeffrey C Miller

Robert T Peters

Tongyao Liu

Casey Christopher Case

Andrew Snowden

Albert Lai

Steve H Zhang

Joe Salas

Xiangdong Meng

Josee Laganiere

Elizabeth J Wolffe, Legal Representative

Elizabeth Wolffe

Carolyn Dent

The present invention provides codon optimized Factor IX sequences, vectors and host cells comprising codon optimized Factor IX sequences, polypeptides encoded by codon optimized Factor IX sequences, and methods of producing such polypeptides. The present invention also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor IX nucleic acid sequence or the polypeptide encoded thereby.

Optimized factor IX gene

The present invention provides codon optimized Factor VIII sequences, vectors and host cells comprising codon optimized Factor VIII sequences, polypeptides encoded by codon optimized Factor VIII sequences, and methods of producing such polypeptides. The present invention also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor VIII nucleic acid sequence or the polypeptide encoded thereby.

Optimized factor VIII gene

The present disclosure provides codon optimized Factor VIII sequences, vectors, and host cells comprising codon optimized Factor VIII sequences, polypeptides encoded by codon optimized Factor VIII sequences, and methods of producing such polypeptides. The present disclosure also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor VIII nucleic acid sequence or the polypeptide encoded thereby.

Optimized factor VIII genes

Disclosed herein are polypeptides, polynucleotides encoding, cells and organisms comprising novel DNA-binding domains, including TALE DNA-binding domains. Also disclosed are methods of using these novel DNA-binding domains for modulation of gene expression and/or genomic editing of endogenous cellular sequences.

DNA-binding proteins and uses thereof

The present invention provides codon optimized Factor VIII sequences, vectors and host cells comprising codon optimized Factor VIII sequences, polypeptides encoded by codon optimized Factor VIII sequences, and methods of producing such polypeptides.

The present invention provides antibodies and antigen-binding molecules thereof which specifically bind the &#x3b1; and/or &#x3b2; subunits of the non-active form of the GPIIb/IIIIa receptor. The antibodies and antigen-binding molecules can be genetically fused and/or conjugated to heterologous moieties and used, for example, as targeting moieties. The invention also includes methods for screening for these antibodies, as well as methods of making and methods of using chimeric molecules derived from the antibodies.

Anti-GPIIB/IIIA antibodies or uses thereof

Disclosed herein are enhanced polypeptides, polynucleotides encoding these polypeptides, cells and organisms comprising novel DNA-binding domains, including TALE DNA-binding domains. Also disclosed are methods of using these novel DNA-binding domains for modulation of gene expression and/or genomic editing of endogenous cellular sequences.

Modified DNA-binding proteins and uses thereof

Disclosed herein are methods and compositions for treating neuropathies by modulating endogenous NT-3 of GDNF gene expression.

Growing community of inventors

Lexington, MA, United States of America

Siyuan Tan