A non-profit brought an abandoned rare disease gene therapy to market. Can the model scale?

The approval of a gene therapy for Wiskott-Aldrich syndrome that was brought to the finish line by a non-profit organization is evidence that paths to regulatory approval exist for rare disease gene therapies abandoned by industry. The challenge is finding ways to scale the approach.

This month, FDA announced the approval of Waskyra etuvetidigene autotemcel, a lentiviral stem cell-based gene therapy to treat the rare, X-linked genetic blood disorder that causes immunodeficiency and low platelet count, resulting from mutations in the WAS gene. The approval was remarkable because it was the first time a non-profit served as the applicant and because it’s one of the only rare disease gene therapies to gain FDA approval this year...