uniQure submitted a Marketing Authorisation Application (MAA) for AMT-130 to the U.K.'s MHRA.
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Today we announced the submission of a BLA to the U.S. FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the…
Company
uniQure
Transforming the lives of patients through gene therapy
- Industry
- Biotechnology Research
- Location
- Amsterdam, NL
- Company size
- 201–500 employees
About uniQure
uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases.
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Latest activity from uniQure
5 signals
Presence & Recognition
uniQure presented safety data related to the Fabry genetherapy trial at SSIEM 2026 in Helsinki, Finland
Presence & Recognition
uniQure hosted its first-ever Visiting Professors Lecture at the Lexington office, featuring Dr. Kelly Andrzejewski and Dr. Jonathan Riley.
Legal & Regulatory
uniQure submitted a BLA to the U.S. FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130) for the treatment of Huntington's Disease.
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The Pharma Letter
The Pharma Letter reports that a Marketing Authorization Application has gone to the UK Medicines and Healthcare products Regulatory Agency (MHRA) in parallel with a Biologics License Application submitted by uniQure to the US Food and Drug Administration (FDA).
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Ultragenyx
Ultragenyx received FDA approval for its first gene therapy drug substance and manufacturing facility for GSDIa.
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European Huntington's Disease Network (EHDN)
European Huntington's Disease Network (EHDN) announced that uniQure submitted a Biologics License Application (BLA) to the Food and Drug Administration in the US for the accelerated approval of AMT-130, an investigational gene therapy for the treatment of Huntington's Disease.
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Thermo Fisher Scientific
Thermo Fisher Scientific submitted AMT-130, its investigational gene therapy for Huntington’s disease, for approval in the US and UK.
Legal & Regulatory
PackGene Biotech, Inc.