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.
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
In another reversal, FDA gives uniQure accelerated approval path for Huntington's gene therapy
FDA reverses course again and grants uniQure an accelerated approval pathway for its Huntington's disease gene therapy, with the company planning a Q3 submission.
Leadership Team
Careers & Job Openings