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California Company and Stanford Researchers To Develop FIX Therapy
 

Pain Therapeutics, Inc., a biopharmaceutical company based in San Francisco, announced
that it will collaborate with Stanford University researchers to develop a therapy for
factor IX defiency.

In 2002, Michele P. Calos, PhD, associate professor of genetics at Stanford, developed
and patented a technique that delivers genetic material to chromosomes to effect a
positive therapeutic response. Specifically, a functioning copy of the gene responsible for
factor IX (FIX) is inserted into the cells of a person with hemophilia B, triggering regular
production of the missing protein. The primary advantage of such a therapy is that it
could possibly sustain normal clotting or at least reduce the number of needed factor
infusions for patients.

Unlike some other approaches to gene-based therapies, the new integration technology
does not use viral vectors for delivery, which can be unstable and somewhat ineffective.
Results of a pre-clinical study, “Site-specific Genomic Integration Produces Therapeutic
Factor IX Levels in Mice,” were published in the November 2002 edition of the journal
Nature Biotechnology. According to the company, the study showed “robust, persistent
and normal levels” of FIX in mice.

Pain Therapeutics, Inc., has entered into an agreement with Poetic Genetics, Inc., a
company co-founded by Calos in 2002, to commercialize the novel technology. Research
will continue at Stanford University, while Pain Therapeutics, Inc., initiates a clinical
program for drug development.      

Source: Pain Therapeutics, Inc. press release dated March 22, 2007