HEMOPHILIA

In 2007 we announced we in-licensed new technology to treat hemophilia, a rare blood disorder.

As an alternative to replacement therapy, Michele P. Calos, PhD, associate professor of genetics at Stanford University School of Medicine, has developed a novel gene integration technology. Her patented technology proposes to replace daily or weekly protein replacement therapy with a single, lasting procedure that promotes normal blood coagulation. The key is to insert a healthy copy of the human gene for Factor IX, a protein responsible for blood clotting, into the cells of a hemophilia patient. If this method promotes robust, persistent and normal levels of Factor IX, patients may be cured of hemophilia. Importantly, this method of gene integration does not rely on viral vectors. Instead, Dr. Calos has demonstrated that a bacteriophage integration system provides a precise method for directing and inserting a gene of interest into chromosomes.

In a pre-clinical experiment published in Nature Biotechnology, this method of gene integration was shown to be highly site-specific. It also resulted in robust, persistent and normal levels of Factor IX in mice. Furthermore, protein expression levels remained stable throughout the eight months of the experiment. Full details of the experiment are available in a publication titled, “Site-specific Genomic Integration Produces Therapeutic Factor IX Levels in Mice” (Nature Biotechnology 20, 1124-1128 (2002)).

In 2002, Dr. Calos co-founded Poetic Genetics, Inc. to broadly commercialize her technology. Under the terms of a license agreement between Pain Therapeutics and Poetic Genetics, Poetic was paid an undisclosed upfront fee and is eligible to receive milestone payments totaling $4 million in the aggregate, based on clinical and regulatory progress, and a 4% royalty on net sales (or 6% if the first U.S. sale of a licensed product occurs before January 1, 2011). In exchange, Pain Therapeutics was granted exclusive, worldwide commercial rights to the technology in all indications in hemophilia and pain management.