Pain Therapeutics Licenses Biotechnology to Treat Hemophilia

Pain Therapeutics, Inc. (San Francisco, CA, USA) has licensed new technology to treat hemophilia, a rare blood disorder. The technology, originally developed by researchers at Stanford University School of Medicine (Stanford, CA, USA) is expected to enter the clinic in 2008. Pain Therapeutics continues to expect its net cash requirements for 2007 to be approximately US$10 million, inclusive of program expenses related to hemophilia.

Hemophilia is a rare, inherited blood clotting disorder. It affects approximately 400,000 people worldwide. People with hemophilia are deficient in the key proteins--factor VIII (hemophilia A) or factor IX (hemophilia B)--that are vital to prevent uncontrolled bleeding. Both forms of hemophilia are characterized by spontaneous or prolonged bleeding, typically into patients' joints and soft tissue. Patients with hemophilia A or B are dependent on protein replacement therapy with Factor VIII or Factor IX, respectively, for life. Outside Western countries, many patients with hemophilia lack access to protein replacement therapy; left untreated, they have a short life expectancy.

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.

This licensing is part of our strategy to develop novel drug candidates in hematology/oncology, while maintaining a business model that requires a modest outlay of cash, said Remi Barbier, president and CEO of Pain Therapeutics.
Over the next 12 months, we plan to develop a novel drug candidate that specifically targets a key mechanism underlying hemophilia, added Nadav Friedmann, Ph.D., M.D., chief operating, and medical officer of Pain Therapeutics. Basic research will continue to be conducted at Stanford, while we focus solely on the initiation of a clinical program.

In a pre-clinical experiment, 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.


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Stanford University School of Medicine
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