STAT+: In a first, a nonprofit wins FDA approval for rare disease gene therapy

The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug company, but an Italian charity. 

The authorization of Waskyra on Tuesday for Wiskott-Aldrich syndrome reflects the challenges buffeting gene therapies, and how academics and nonprofits are trying to overcome them.

Companies have broadly fled the genetic medicine field, finding that the complex treatments are too difficult to make steady profits off of, especially given that many are designed to treat extremely rare conditions. Even with price tags in the millions of dollars, these one-time therapies have, with few exceptions, not become reliable moneymakers.

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