uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients
with severe disorders.
We believe our multiple partnerships and the regulatory approval of our lead product Glybera in the European Union for a subset of patients with LPLD provides validation for our approach.
Positive Topline Results Announced from Phase I/II Trial in Sanfilippo B Syndrome Patients Using uniQure’s Novel AAV5-Based Gene Therapy
Dr. Charles W. Richard Joins uniQure as Senior Vice President to Lead Development of CNS-focused Gene Therapy Pipeline