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Gene therapy is in crisis. Its top minds searched for a solution

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W ASHINGTON — Jennifer Puck has successfully treated 10 children with a gene therapy for a fatal disorder that decimates their immune system. But she has no idea how to get her drug approved and frankly is running out of ideas. “I wish I had a clue about where to go from here,” said Puck, an immunologist at University of California, San Francisco, from a plush chair above Union Station. advertisement The problem is simple: Size. Puck’s therapy is for a disease, Artemis-SCID, that affects just two to three new U.S. patients every year — far too few for a company to generate a profit, or to even run the kind of studies regulators usually demand before approving drugs. She is far from alone. Puck’s case is emblematic of a crisis that drew a few dozen gene therapy researchers and a couple of key advocates and federal officials to an invite-only workshop in Washington last month, a quagmire born of the field’s own success. After a half-century of fits-and-starts, the tools of molecular biol

W ASHINGTON — Jennifer Puck has successfully treated 10 children with a gene therapy for a fatal disorder that decimates their immune system. But she has no idea how to get her drug approved and frankly is running out of ideas. “I wish I had a clue about where to go from here,” said Puck, an immunologist at University of California, San Francisco, from a plush chair above Union Station. Advertisement The problem is simple: Size. Puck’s therapy is for a disease, Artemis-SCID , that affects just two to three new U.S. patients every year — far too few for a company to generate a profit, or to eve

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