Post by Patrick Page

CASE STUDY / INDUSTRY LESSON uniQure: 20 Years of Breakthroughs, Setbacks, and the Slow Reality of Gene Therapy From Glybera, the first-ever approved gene therapy in Europe, to Hemgenix, the world’s most expensive treatment, uniQure has spent two decades at the cutting edge of science — yet commercial success has remained elusive. Why? Because gene therapy isn’t just about science. It’s about timing, patient populations, delivery complexity, and an economic model still incompatible with multi-million-dollar one-shot treatments. In my latest Decode Pharma Insights article, I retrace uniQure’s journey: From early triumphs and massive hype to modest adoption. Strategic funding moves raising over €1 billion ($1.1 B) while surviving the slow uptake of revolutionary therapies. Lessons from Hemgenix and AMT 130: scientific success doesn’t equal commercial scale. What the slow reality of gene therapy teaches us about timing, strategy, and the future of biotech. If you want a realistic, unsentimental view of gene therapy’s progress and pitfalls — and what it means for investors, innovators, and the next generation of rare disease therapies — this one’s for you. Read the full article in my LinkedIn weekly newsletter below #GeneTherapy #Biotech #UniQure #Hemgenix #Glybera #RareDiseases #PharmaInsights #Biopharma #Innovation #HealthcareStrategy #DecodePharma