US-amerikanische Firma will Gen-Therapie für Hämophile entwickeln

Pain Therapeutics targets hemophilia

Pain Therapeutics, best known for developing abuse-resistant pain killers, is stepping into gene therapy to pursue a cure for hemophilia.

The South San Francisco-based company has licensed technology developed at Stanford University School of Medicine that allows for the integration of a gene into a patient's chromosomes. Pain Therapeutics licensed the technology from Poetic Genetics Inc., a company co-founded by Michele Calos, an associate professor of genetics at Stanford.

The deal is part of an effort by Pain Therapeutics to use its strong cash position and its drug development expertise to expand its pipeline beyond pain killers. The company already is developing a monoclonal antibody to treat metastatic melanoma. And even though gene therapy might seem like a radical departure for the company, CEO Remi Barbier said he views it as nothing more than drug development.

"Our company has a very narrow charter -- to develop new drugs -- drugs that save lives," he said. "Within that narrow charter, there's room to maneuver."

Hemophilia is a rare, inherited blood clotting disorder that affects about 400,000 people worldwide. People with hemophilia lack the ability to produce a protein that is that is critical in clotting. Hemophiliacs can suffer spontaneous or prolonged bleeding and must take regular injections of the missing protein for life to control the disease. The cost of such therapy can exceed $100,000 a year.

Using the technology that Calos developed, the missing gene can be pumped into the liver along with a gene for an enzyme that will integrate the missing gene into the patient's chromosomes.

Once there, the patient's own cells will continue to replicate the gene as cells regenerate, raising hopes that patients could be cured with a single treatment.

Under the terms of the license agreement, Pain Therapeutics paid Poetic Genetics an undisclosed up-front fee. Poetic is eligible to receive milestone payments totaling $4 million and a 4 percent royalty on net sales - 6 percent if the first U.S. sale occurs before 2011