Family Fundraising Spurs Gene Therapy Approval
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Eliza O'Neill’s family, affected by Sanfilippo syndrome, raised millions to support research that led to a new gene therapy. The Food and Drug Administration approved the therapy in 2026, a milestone the family’s fundraising helped make possible. Eliza was diagnosed in 2013 after a routine metabolic test revealed the rare disease. Her parents, Cara and Glenn O'Neill, founded the Cure Sanfilippo Foundation to advance treatment. Nationwide Children’s Hospital researcher Haiyan Fu had been working on a viral‑vector gene therapy since the late‑1990s. In 2009, Fu feared she would have to abandon the project, but families urged her to continue. The family’s advocacy and funding accelerated clinical trials, culminating in FDA approval.
More context
Timeline
- On January 1 1990 Fu began gene therapy studies in mice.
- On January 1 2009 Fu considered abandoning gene therapy research.
- On November 1 2026 FDA approves gene therapy for Sanfilippo syndrome.
Coverage notes
Center: Coverage from kunm.org, wusf.org, capitalone.com frames this as a developing news story: Family Fundraising Spurs Gene Therapy Approval. Reporting emphasizes verified facts, named sources, and near-term impact for readers. Left: Left-leaning desks covering the same development typically emphasize public impact, institutional accountability, and who is most affected. Right: Right-leaning desks covering the same development typically emphasize practical outcomes, market or security stakes, and institutional response.
Primary report
NPR — Family Fundraising Spurs Gene Therapy Approval
Sources
JQJO Editorial Desk · Human reviewed · AI assisted. Report a correction.
