Developing Therapies to Treat Life-Threatening Rare Diseases

Statue of San Rocco outside of the former Centro Medico San Rocco, Altamura, Italy (SRT’s logo is derived from this sculpture)

Photo Credit: Luigi Porzia

About San Rocco Therapeutics

Our goal of treating life-threatening rare diseases started in 1992 when Patrick Girondi’s son, SRT Founder, was diagnosed with Thalassemia.

Wanting to avoid blood transfusions at a time when AIDS and Hepatitis C was present in donated blood, Patrick Girondi’s son began taking Dr. Susan Perrine’s experimental medicine, Arginine Butyrate, in 1993. Arginine Butyrate, one of the first gene therapies, stimulated expression of the gamma globin gene (otherwise known as fetal hemoglobin gene, which is normally silenced when a child is 18 months old). Beta globin is the adult hemoglobin gene.

Perrine’s father was a doctor in Saudi Arabia. Culture prevented him from visiting patient homes in the absence of male heads of household. Susan assisted with house calls and noticed asymptomatic Sickle Cell Disease patients. She discovered that patients naturally had genetic expression of the gamma globin gene, stimulated  through high amounts of butyric acid. 

In 1993, Patrick Girondi and Sandy and Walter Hayhurst founded Emerging Pharmaceutical Technologies (EPT) around Perrine’s Arginine Butyrate. 

EPT became Beacon laboratories through a partnership with John Walton (Sam Walton’s son), in 1995. 

In 1995, Girondi also founded Centro Medico San Rocco in the city of Altamura, Italy, to treat Thalassemia patients. In 1998, Girondi sponsored and conducted a 38-patient Clinical Trial with Arginine Butyrate and the oral version, Isobutyramide, with universities of Milan, Boston, Bari, and medical centers in Oakland, Toronto and Taranto, Italy. 

Girondi met Michel Sadelain of Memorial Sloan Kettering in 2000 and began supporting Sadelain’s gene therapy project for Sickle Cell Disease and Thalassemia. 

In 2004, Beacon became Errant Gene Therapeutics (EGT).

In 2005, EGT became the World Wide Licensor of Thalagen for Sickle Cell Disease and Thalassemia, together the most common hematological rare diseases in the world. EGT built a world wide team with dozens of researchers doctors and collaborators including and not limited; Saint Jude, University of Milan, University of Minnesota, University of NY, Thessaloniki, Greece Medical Center, Taranto Hospital, University of Bari, Boston Children’s Hospital, Beckman Research Institute, Children Hospital of Oakland Research institute, Sick Children’s Hospital of Toronto, Canada and others.

From 2005, EGT dedicated all of its resources to the Thalagen project. 

In 2020, after an almost 6 year court battle, EGT is again pushing ahead with the Sadelain vector. See Article

EGT became San Rocco Therapeutics in 2022. 

San Rocco is the patron saint of hopeless disease. He became known for his brave and humane deeds caring for people with the plague in the 1500’s. San Rocco contracted the plague himself and went off to die in the wilderness. He was befriended by a dog who brought him food, companionship, and licked his wounds. Statues of San Rocco often depict him with a dog, bread in mouth.