How an Ohio Kids’ Hospital Quietly Became Ground Zero for Gene Therapy
Summary
“It looked like it had no future, people were really frightened,” says Jerry Mendell, the longtime director of Nationwide’s gene therapy center and a pioneer in the field of neuromuscular disease research. In clinical tests, the spinal muscular atrophy treatment Zolgensma, developed by Mendell’s team and already the centerpiece of an $8.7 billion buyout, has given infants with a probable death sentence a chance to live, grow, and thrive. (Yes, they insist on the “The.”) Between 1950 and 2000 as suburbs sprouted up around Columbus, it annexed more than 150 miles of nearby land and grew considerably, building a diverse economy that went beyond its industrial roots. “They saw that the way to take the hospital to the next level was to invest in research and academic medicine,” says Johnson, now the chief scientific officer of a stealthy Philadelphia gene therapy startup called Limelight Bio . Johnson and his first hire Reed Clark, who had earned his PhD in molecular genetics from Ohio State, began working on ways to produce AAVs more efficiently and in greater batches.