Let me tell you a story about Evangelina Padilla-Vaccaro. Evangelina was diagnosed after birth with severe combined immunodeficiency; also known as “bubble baby” disease. Most children with this condition die within the first few years of life. Evangelina was cured as a part of a clinical trial run by Dr. Don Kohn and his team at UCLA, working with a UK-based company called Orchard Therapeutics. The treatment she underwent was called OTL-101, which involves taking the patient’s own blood stem cells, genetically re-engineer them, and returned them to her body. These re-engineered stem cells created a new blood and immune system. Now this might sound like science fiction, but this is a true story. Evangelina is now 6 years old and she is living
