American Thought Leaders

How I Discovered a Possible Cure for My Son’s Incurable Disease | Patrick Girondi

Nov 12, 2025
Patrick Girondi is an author, musician, and the founder of San Rocco Therapeutics, established after his son Rocco was diagnosed with thalassemia. In this engaging conversation, he shares his journey from high school dropout to successful entrepreneur, driven by a father's love. Girondi discusses developing gene therapies, navigating challenges in the pharmaceutical world, and the importance of accessible treatments for rare diseases. He reveals exciting advancements with patients, including his son, and outlines a vision for universal access to innovative cures.
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ANECDOTE

Father Teaches Himself Medicine To Save Son

  • Patrick Girondi taught himself hematology and moved to Italy to treat his son when U.S. care was unaffordable.
  • He bought intellectual property in 2005 and produced the world's first multi-dose gene therapy batch in 2010.
INSIGHT

How Gene Replacement Works

  • Gene replacement inserts a corrected beta-globin gene into patient stem cells using a disabled lentiviral vector.
  • After reinfusion and marrow recovery the body produces correct adult hemoglobin permanently.
ANECDOTE

Early Trials Show Partial Success

  • San Rocco treated patients in 2012 and achieved a 43% transfusion reduction in some patients after cautious conditioning.
  • The team concluded full myeloablation would be required for cures and later updated the product to improve efficacy.
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