UniQure welcomes possible adcomm for Huntington’s asset as regulatory ‘jitters’ spread through biopharma
UniQure welcomes possible adcomm for Huntington’s asset as regulatory ‘jitters’ spread through biopharma
uniQure
uniQure N.V. is a gene therapy company developing a single administration of AAV-based therapies to treat serious genetic diseases, with a portfolio spanning hemophilia, Huntington's disease, ALS, Fabry disease, and refractory epilepsy. The company developed the first FDA-approved hemophilia B gene therapy (Hemgenix, partnered with CSL Behring) and is advancing AMT-130, a one-time gene therapy for Huntington's disease, as its lead wholly-owned program.
Company Info
Sector:Biotechnology / Gene Therapy
Headquarters:Amsterdam, Netherlands
Founded:2012
Website:www.uniqure.com/
Upcoming Milestones & Key Dates
Q2 2026
Earnings
Q1 2026 Earnings — AMT-130 Cohort 3 dosing update and Hemgenix royalties
Late 2026
Clinical Trial
Interim AMT-130 efficacy and biomarker data in early-stage Huntington's patients
Clinical Development Pipeline
Hemgenix (etranacogene dezaparvovec)
Target: Factor IX (FIX) Padua variant via AAV5AMT-130
Target: HTT-lowering via AAV5-delivered microRNA (one-time CNS administration)AMT-260
Target: Calmodulin-regulated adenylyl cyclase 1 (ADCY1) suppression via AAVRecent News
UniQure's chronic epilepsy gene therapy 'should be on investors' radar'
UniQure's chronic epilepsy gene therapy drew renewed analyst attention, with BioSpace flagging it as a story investors should track amid a competitive gene therapy landscape.
New hope in treating Huntington's disease and a report card on RFK Jr.'s promises
The Readout LOUD podcast covers new hope for Huntington's disease treatment and a report card on RFK Jr.'s promises.
Huntington's Disease Gene Therapy AMT-130 Is on Track for FDA Approval After uniQure Reaches Regulatory Agreement
Huntington's Disease Gene Therapy AMT-130 Is on Track for FDA Approval After uniQure Reaches Regulatory Agreement.
Leadership Team
Careers & Job Openings