AMT-130 by uniQure: status and timeline

Milestones

Next · Four-year topline data release

First patient dosed in Phase 1/2 study2021Complete.
6-month interim data shows disease slowing2024Complete.
36-month data: 75% slowing of disease progression (high-dose)Sep 2025Complete.
FDA Type B meeting aligns on BLA pathwayJun 2026Complete.
Four-year topline data releaseTarget Sep 2026Current milestone.
FDA and MHRA regulatory submissionsTarget Q3 2026Not yet reached.

Most important updates

uniQure holds Type B meeting with FDA; BLA submission pathway for AMT-130 confirmedJun 2026
uniQure announces 36-month Phase 1/2 data for AMT-130: 75% slowing of Huntington's disease progression in high-dose cohortSep 2025

Upcoming

Q3 2026FDA and MHRA regulatory submissions (next)
Sep 2026Four-year topline data release
2027FDA accelerated approval decision

Current obstacles

Transduction variabilityPre-existing AAV antibodies and individual CNS anatomy may reduce huntingtin reduction across patients.
Immune activationImmune responses to the vector could reduce efficacy or cause CNS inflammation.

Physics limits

AAV cargo capacity constraintsAAVs carry max ~4.7 kb payload. This limits gene size and sequences, restricting options to small effector genes like microRNAs.
Blood-brain barrier transitAAVs must cross cellular barriers and reach enough neurons in affected brain regions (striatum, cortex, subthalamic nucleus) for clinical benefit.
Multifactorial neurodegenerationHuntington's involves mutant huntingtin plus mitochondrial dysfunction, neuroinflammation, and synaptic loss. Lowering huntingtin may not fully arrest progression.

How it works

4 parts

Intrathecal infusion

Delivery to cerebrospinal fluid

AMT-130 is injected into the spinal canal, delivering AAV directly to cerebrospinal fluid and CNS.

AAV transduction

AAV-mediated cell entry

The AAV vector crosses the blood-brain barrier and enters neurons and glial cells, delivering the therapeutic microRNA gene.

MicroRNA silencing

Huntingtin mRNA degradation

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

Durability

Sustained expression

AAV-mediated expression is durable, providing sustained huntingtin reduction from one treatment, no re-administration.

Spec sheet

Spec	AMT-130
Disease progression slowing (36 mo)	75%^R (reported)
Functional capacity slowing	60%^R (reported)
High-dose cohort size	12 patients^R (reported)
Total Phase 1/2 enrollment	29 patients (17 high-dose, 12 low-dose)^R (reported)

^R reported by the company

Update log

2 updates

Jun 2026

Major: uniQure holds Type B meeting with FDA; BLA submission pathway for AMT-130 confirmedPressRegulatory

Sep 2025

Major: uniQure announces 36-month Phase 1/2 data for AMT-130: 75% slowing of Huntington's disease progression in high-dose cohortPressTest

About uniQure

The team behind AMT-130

uniQure

Gene therapy, Huntington's disease, hemophilia, Amsterdam

Gene therapy company founded in 1998 in Amsterdam. Developed Glybera, the first approved in Europe. Focuses on hemophilia and Huntington's. AMT-130 showed 75% disease slowing; submissions Q3 2026.

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.

uniqure.com (opens uniqure.com)Company profile →

Founded: 199828 yrs
Headquarters: Netherlands
Status: Public
Listed: QURE (opens google.com)NASDAQ
Valuation: $650Mmarket capJan 2026
Staff: ~220Mar 2026
Raised: $243M1 round
Last round: Follow-on public offering · $243MJun 2026
Works in: BiotechGene editing & therapy
Coverage: 1 program · 3 updateslatest 1 Jun 2026checked 10 Oct
People: Matthew Kapusta (opens uniqure.com)Chief Executive Officer

Spec

AMT-130

Disease progression slowing (36 mo)

75%^R (reported)

Functional capacity slowing

60%^R (reported)

High-dose cohort size

12 patients^R (reported)

Total Phase 1/2 enrollment

29 patients (17 high-dose, 12 low-dose)^R (reported)