

Why Gene Therapy is So Expensive
Jun 15, 2025
Dive into the world of gene therapy and uncover why it's the priciest treatment available today. Explore the labyrinthine production process of viral vectors, which poses unique manufacturing challenges. Discover the historical milestones that have shaped the industry and the regulatory hurdles that complicate development. This fascinating topic reveals the intricate balance between innovation and cost in biopharmaceuticals.
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Gene Therapy's Cost Drivers
- Gene therapies are the most expensive drugs due to the complex production of viral vectors for in vivo gene therapy.
- Producing these vectors is arguably the hardest scaled process in biopharmaceuticals.
Early SCID Gene Therapy Trial
- In 1990, two girls with severe immune disorder SCID received retrovirus-based gene therapy.
- The trial showed some recovery but unclear if gene therapy or enzyme therapy caused it.
Jesse Gelsinger Tragedy
- Jesse Gelsinger died after receiving an adenovirus-based gene therapy in a 1999 trial.
- The high dose triggered a fatal immune response, halting adenovirus use in US gene therapies.