San Rocco Therapeutics

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 XIV century. 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.

San Rocco Goal

Bring affordable gene therapy to patients

-patg pact

 

Yearly checkups were mandatory in Catholic school. Ma loaded us on the bus and paid $10 to Dr. Farrell. That was the going rate for people like us and people like him.

My son was diagnosed in ‘92.’ I made a pact. It was pretty easy to do.

Many executives treat startups as personal smorgasbords, their compensation dwarves patient interest. As an American entrepreneur, I say that research comes before my salary.

San Rocco is revered for benevolence during the plague.

San Rocco Therapeutics, following his example.

Rare and Orphan Diseases

Collectively rare diseases affect more than 25 million Americans. The prevalence of more than 6,000 rare diseases and the absence of available treatments in the United States for these diseases received extensive publicity in 1980.

Sickle Cell DISEASE

Sickle cell disease is a group of inherited red blood cell disorders that affects hemoglobin, the protein that carries oxygen through the body. Normally, red blood cells are disc shaped and flexible to move easily through the blood vessels. If you have sickle cell disease, your red blood cells are crescent or “sickle” shaped. These cells do not bend and move easily and can block blood flow to the rest of your body.

Beta Thalassemia

Thalassemia is an inherited blood disorder that reduces the production of functional hemoglobin (the protein in red blood cells that carries oxygen). This causes a shortage of red blood cells and low levels of oxygen in the bloodstream, leading to a variety of health problems.[1] There are two main types of thalassemia, alpha thalassemia and beta thalassemia. Signs and symptoms vary but may include mild to severe anemia, paleness, fatigue, yellow discoloration of skin (jaundice), and bone problems.[2] Beta thalassemia is caused by changes (mutations) in the HBB gene while alpha thalassemia is caused by mutations in the HBA1 and/or HBA2 genes. Both are inherited in an autosomal recessive manner.[3][4] Treatment depends on the type and severity of the condition but may include blood transfusions and/or folic acid supplements.[5][2]

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+1 (312) 441-1800

Our Locations

Wells Fargo Center
100 S. Ashley Drive
Suite 600
Tampa, FL
33602

 

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