Rare disease pioneer Rogerio Vivaldi, M.D., was the first to bring enzyme replacement therapies to Latin America as a senior vice president and head of the rare disease unit at Genzyme. Later, as chief commercial officer of Spark Therapeutics, he paved the way for the commercialization of Luxturna, the first gene therapy approved in the U.S. He also served as EVP and chief global therapeutic operations officer at Bioverativ. Now, as chief executive officer of Sigilon Therapeutics, he’s advancing a pipeline of novel cell therapies for chronic diseases, including hemophilia and diabetes. They are designed to be implanted in patients and function for years as biofactories, churning out the protein or factor they need to stabilize their health. In this address, Dr. Vivaldi will share lessons learned from his career on how to:
- Lay the groundwork with regulators
- Think about ex-U.S. opportunities
- Prepare the market – payers, providers and patients – for the arrival of a new modality
