uniQure N.V., a biopharmaceutical company, develops adeno-associated virus (AAV) based gene therapies through its technology platform for multiple therapeutic areas. The company offers Glybera for the treatment of patients with lipoprotein lipase deficiency. It also develops AMT-060, a gene therapy that is in Phase I/II clinical trial for the treatment of hemophilia B; S100A1, a preclinical therapeutic for congestive heart failure; AMT-021, a gene therapy, which has completed Phase I clinical trial for acute intermittent porphyria; AMT-110, a gene therapy that is in Phase I/II clinical trial for Sanfilippo B syndrome; and AAV2/glial cell line-derived neurotrophic factor for Parkinson's disease. The company has collaborations with Chiesi Farmaceutici S.p.A., Digna Biotech, Institut Pasteur, University of California San Francisco, Bristol-Myers Squibb, and 4D Molecular Therapeutics. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
January 30, 2015
Spark Therapeutics (ONCE) Gene-therapy company shares double in debut
December 24, 2013
Merry Christmas
November 16, 2013
Debt is a drag
November 16, 2013
The Tell
35 companies, 1 interpreter!
Insight, foresight and recommendation
My motto … never leave an investor uninformed! I say today what others won’t, so you can do what others can’t.
My name is Henry McCusker; I endorse these opinions under my own name and with a subscription based membership.
Do you have an opinion you would like to share with us? We would like to hear from you.
Please e-mail us at hwm@regmedinvestors.com.
Editor and Publisher ... Henry McCusker enters his tenth (10th) year at RegMed Investors
© 2026 RegMed Investors (regmedinvestors.com), all rights are reserved.
RegMed Investors (RMi) or Regmedinvestors.com content is the intellectual property of Henry McCusker. Any copying, republication or redistribution of RMI’s content, including by framing or similar means, is expressly prohibited without the prior written consent of Scimitar; who also shall not be liable for any errors or delays in content, or for any actions taken in reliance thereon. For additional information on other Scimitar media services or reposting please email hwm@regmedinvestors.com.
The information included above as well as individual companies and/or securities mentioned should be construed as guidance in RMi reviews.
The facts stated and the opinions given in this publication are based on data and information considered to be reliable and have been carefully worked into my qualitative analyses and prognoses. However, no guarantee can be given as to their fairness, accuracy or completeness.
Henry McCusker, the editor and publisher of RegMed Investors does not hold or have positions in securities referred to in this publication.