Post by Benjamin McLeod

A gene therapy for Huntington’s was left for dead by the FDA in November. What happened this week might be the biggest CGT story of the year so far. Background: - Huntington’s is a genetic death sentence. - One inherited gene leads to neuron decay over years.  - Movement, memory, and personality slip away. 30,000 to 41,000 Americans live with it today. Zero approved therapies slow it down. That is the world uniQure walked into with AMT-130: - A one-time gene therapy.  - Delivered through MRI-guided brain surgery.  - Built to lower the mutant protein at its source. THE CLIMB The data was incredibly promsing: → High-dose cohort hit its primary endpoint at 3 years → 75% slowing of progression on cUHDRS By June 2025, uniQure had FDA alignment and a runway to file. THE FALL In November 2025, the FDA reversed.  The Phase I/II data was suddenly not enough.  The agency wanted another trial, a demand many called impractical, even unethical, for a disease this severe. The filing timeline went dark. Stock dropped dramatically. THE CLIMB Thankfully, uniQure refused to quit. They re-engaged with the FDA.  The agency’s leadership turned over. New flexibility on rare disease started to emerge. Then, on June 17, the reversal: → The 3-year data got accepted as the primary basis for accelerated approval → BLA filing set for Q3 2026 → Potential approval in 2027 (!!) QURE shares jumped nearly 80% in a day. That being said, the story isn’t finished yet. But for the first time in many months, it has a sizeable shot at a good ending. Congrats to the entire uniQure team - we're rooting for you! Anything else you’d add? Drop it in the comments.