TheTechno Optimist.

AMT-130uniQure—uniQure holds Type B meeting with FDA; BLA submission pathway for AMT-130 confirmeduniQure—uniQure raises $242.7 million in follow-on public offering; cash reserves reach $811.9 million

uniQure

Gene therapy, Huntington's disease, hemophilia, Amsterdam

NL$650M market cap~220 staffest. 1998uniqure.com (opens uniqure.com)Checked 10 Oct

The brief

Analyst view
  • uniQure is the first company to bring a gene therapy to market in Europe (Glybera for hemophilia B).
  • Q2 2026 revenue was $5.8 million; cash position $811.9 million after a June 2026 public offering of $242.7 million.
  • AMT-130 for Huntington's disease achieved 75% slowing of disease progression at 36 months in Phase 1/2; FDA and MHRA submissions Q3 2026.

Technical approach

As reported
Gene delivery

AAV vectors across the blood-brain barrier

AAV vectors deliver therapeutic genes to CNS cells. They are small, naturally derived, and achieve durable transgene expression.

Mechanism

Huntingtin silencing via microRNA

Synthetic microRNA targets and degrades mRNA from the mutated huntingtin gene, reducing toxic protein production.

Durability

One-time CNS treatment

A single AMT-130 infusion achieves sustained huntingtin reduction in the CNS, aiming for one-time cure, not chronic dosing.

Primer