‘Family Man’ and What Happens If a Company Gets Drug Approval Using Someone Else’s Technology?

For years, TrialSite News reported in “Family Man,” the gene therapy saga of healthcare giants Agios Pharma, Bluebird Bio, Third Rock Ventures, 2Seventy Bio and Memorial Sloan Kettering lined up against the tiny but gritty San Rocco Therapeutics (formerly Errant Gene Therapeutics).

San Rocco the biotech company seeking to develop gene therapies at a substantially lower cost to patients claims that its goal is to bring accessible gene therapy to Sickle Cell Disease and Thalassemic patients. Both diseases are caused by a defect on the beta globin gene and both can be cured by the same therapy, replacing and or editing the beta globin gene. Girondi’s son was diagnosed with Thalassemia in October of 1992. Mr. Girondi left a successful trading career to cure his son. (Prior to his son’s diagnosis, Girondi was a two-time guest on the “Oprah Show.”)