CEO Patrick Girondi Says San Rocco’s Gene Therapy Research into Thalassemia Treatments is a Labor of Love

Patrick Girondi said he was inspired to found his San Rocco Therapeutics company to find a cure for his son’s thalassemia. Globally, it is estimated that there are 270 million carriers with abnormal hemoglobins and thalassemias, of which 80 million are carriers of ?thalassemia. Recent surveys suggest that between 300,000 and 400,000 babies are born with a serious hemoglobin disorder each year.

Thalassemia is an inherited blood disorder that causes your body to have less hemoglobin than normal and can cause anemia and fatigue. In severe cases, such as Girondi’s son’s case, patients require repeated blood transfusions. A researcher who has been associated with Girondi’s company, first known as Errant Gene Therapeutics, recently published a pre-print article called “Globin Vector Regulatory Elements Are Active in Early Hematopoietic Progenitor Cells.” The paper proposes a safer way to deliver gene therapies to thalassemiasufferers using lentivirally encoded locus control region (LCR) elements, in particular HS1 and HS2, that can be activated in early hematopoietic cells, including hematopoietic stem cells and myeloid progenitors.

This activity is position-dependent and results in the transcriptional activation of a nearby reporter gene in these progenitor cell populations. We further show that flanking a globin vector with an insulator can effectively restrain this non-erythroid activity without impairing therapeutic globin expression and help mitigate the activation of cancer-related genes. After a series of legal disputes over their technology, Girondi said San Rocco is poised to complete clinical trials within three years. TrialSite: Can you talk about the new paper you have shared with TrialSite? Patrizio Girondi, CEO of San Rocco: In December, we recorded three patients on our protocol with yelosuppression using eight milligrams per kilogram. We had fantastic results where after nine years, they had a reduction of 43 percent of transfusions, which leads us to believe that a product made today is going to be anywhere from 25 to 70 percent more effective because, in 2009, we made our original product and in 13 years, you know, it’s only gotten better. So we’re very excited about that article that came out in December. And the article that came out now is incredibly important.

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