Gene therapy is in crisis. For nine hours, the field’s leading minds looked for a solution

WASHINGTON — 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. 

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.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Individual plans

Group plans

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $399/year

$30 for 3 months Get Started

Then $399/year

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

To read the rest of this story subscribe to STAT+.

Subscribe

Log In