How Taxpayer-Funded Research Led to a $2 Million Gene Therapy: The Zolgensma Story
By
Robin Fields
Summary
This investigative piece traces the development of Zolgensma, a gene therapy for spinal muscular atrophy that became the world's most expensive drug at $2 million per dose. It reveals how taxpayer funding, charitable donations, and academic research at Nationwide Children's Hospital were instrumental in its creation — challenging the pharmaceutical industry's narrative that high drug prices reflect massive private R&D investments. The story follows the journey from the first patient trial in 2014 through AveXis's acquisition by Novartis, and the subsequent pricing decisions that made it inaccessible to many healthcare systems globally.
Source
bskyHow Taxpayer-Funded Research Led to a $2 Million Gene Therapy: The Zolgensma Storypropublica.orgKey quotes
· 3 pulledVincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma had ended and the forces that turned it into the world's most expensive drug had taken over.
At the time, children born with the most severe form of SMA typically died before age 2. There was no cure.
The story of Zolgensma upends the widely held conception that high prices reflect huge industry investments in innovation.
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