# AMT-130

Type: Product

Source: Biotech Intelligence Brief — https://getbiobrief.com/entity/amt-130
Canonical HTML page: https://getbiobrief.com/entity/amt-130

## Timeline

- **2026-09**: Four-Year Follow-Up Data from AMT-130 Pivotal Study — Durability of gene therapy effect expected to be reported, a key factor for regulatory decisions.
- **2026-09**: Regulatory Submissions for AMT-130 in Huntington's Disease (Planned) — uniQure aims to file for U.S. and U.K. approval in Q3 2026, targeting accelerated pathways.
- **2026-06-17**: FDA accepts AMT-130 Phase 1/2 data for BLA — The FDA reversed its earlier requirement for additional data, agreeing existing clinical data supports a near-term BLA submission for Huntington’s gene therapy.
- **2026-06**: AMT-260 Preliminary Data and AMT-191 Further Analyses — Six-month follow-up from the Phase I/IIa AMT-260 trial in epilepsy and new Fabry data presented.
- **2026-Q2**: FDA Type B Meeting — Planned follow-up meeting to define the Phase III pathway for AMT-130 in Huntington's disease.
- **2026-03-02**: Earnings Release — Company announces 2025 results and provides update on FDA Type A meeting.
- **2026-02**: Updated AMT-191 Phase I/II Data in Fabry Disease — Interim data demonstrate sustained enzyme activity and safety in Fabry disease patients.
- **2026**: AMT-260 Data — Clinical data expected from the Phase I/IIa study in refractory mesial temporal lobe epilepsy.
- **2026**: Planned BLA submission in Q3 2026 — UniQure intends to submit a Biologics License Application for AMT-130 in the third quarter, pending alignment meeting on confirmatory study design.
- **2025-12-31**: Fiscal Year End — uniQure closes 2025 with $622.5M in cash and equivalents.
- **2025-09**: Positive Topline Data for AMT-130 in Huntington's Disease — uniQure reports positive pivotal Phase I/II data, showing statistically significant improvements in disease markers.

## Recent coverage (4 stories)

### uniQure Eyes Q3 2026 Filing for HD Gene Therapy; 4-Year Data in Sept
2026-08-05 15:43:02 · Sentiment: Neutral · Impact: 5/10 · Sources: 2

uniQure's AMT-130 for Huntington's disease is on track for U.S. and U.K. regulatory filings in Q3 2026, following positive pivotal data. Pipeline updates for AMT-191 and AMT-260 in Fabry disease and epilepsy show a broadening gene therapy platform.
Full story: https://getbiobrief.com/story/uniqur-hd-gene-therapy-q3-filing

### UniQure Jumps 78% as FDA Accepts Phase 1/2 Data for AMT-130 BLA
2026-07-12 05:40:50 · Sentiment: Very Bullish · Impact: 8/10 · Sources: 2

FDA’s acceptance of existing clinical data for uniQure’s AMT-130 gene therapy signals a major regulatory win, with the company on track for a BLA submission in Q3 2026 and a potential first-in-class approval for Huntington’s disease.
Full story: https://getbiobrief.com/story/uniqure-amt130-fda-phase1-2-data-bla-bio

### AMT-130, NEO212 Progress: 4 Biotech Stocks Jump on Gene Therapy, M&A News
2026-06-19 00:09:11 · Sentiment: Bullish · Impact: 6/10 · Sources: 2

uniQure’s AMT-130 update validates its gene therapy platform, while NTHI’s NEO212 advancement and an acquisition deal spotlight a busy week for biotech pipelines and dealmaking.
Full story: https://getbiobrief.com/story/amt130-neo212-biotech-surge

### uniQure Eyes 2029 Runway as FDA Talks for Huntington's Gene Therapy Continue
2026-03-03 00:54:14 · Sentiment: Neutral · Impact: 6/10 · Sources: 2

uniQure reported a robust $622.5 million cash position, extending its operational runway into late 2029. While clinical data for its Huntington’s disease candidate AMT-130 remains strong, the company is still seeking regulatory alignment with the FDA on a definitive Phase III pathway.
Full story: https://getbiobrief.com/story/uniqure-2025-earnings-huntingtons-gene-therapy-update

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