Gene Therapy:


uniQure has built an exciting portfolio of clinical and preclinical programs focused on hemophilia.

uniQure has built an exciting portfolio of clinical and pre-clinical programs focused on hemophilia. This overall effort is well supported by uniQure’s proprietary position with our AAV5 viral vector, which has shown particular affinity for reaching the liver. AAV5 has the benefit of a low prevalence of pre-existing anti-AAV5 antibodies in patients screened to date in uniQure’s clinical trials and as indicated in medical literature. uniQure also has gained extensive experience in the development and regulatory approval of the first gene therapy approved in the Western world: Glybera.

Hemophilia B

uniQure is developing a gene therapy for hemophilia B, a severe orphan blood clotting disorder. The program’s gene therapy product candidate consists of an AAV5 vector carrying a therapeutic human Factor IX, or FIX, gene cassette that uniQure has exclusively licensed from St. Jude Children’s Research Hospital. uniQure is currently conducting a Phase I/II study of AMT-060 in patients with severe hemophilia B and advanced joint disease.

In January 2017, AMT-060 received Breakthrough Therapy designation from the U.S. Food and Drug Administration in January 2017 based on results from this ongoing, dose-ranging study (view press release).  In April 2017, AMT-060 also received PRIME designation by the European Medicines Agency (EMA) on the strength of the same data.  

In July 2017, the Company presented updated clinical data from our Phase I/II trial (view press release).   

The data from our Phase I/II study demonstrate that our AAV5-based gene therapy has been safe, effective and durable, with no loss of efficacy at up to 18 months of observation and no cellular immune responses in any patient.  AMT-060 is delivering sustained and significantly improved clinical benefits to patients suffering from severe hemophilia B by enabling them to discontinue bi-weekly infusions of FIX replacement therapy and essentially eliminating the risk of spontaneous bleeding. We aim to provide a therapeutic benefit from AMT-060 that is superior to previous prophylactic FIX  replacement therapy, even in patients with advanced joint disease who still experienced many bleeds despite prophylaxis with FIX.

The data from this ongoing study demonstrate clinically significant and sustained increases in FIX activity, substantial reductions in FIX replacement usage and a near cessation of spontaneous bleeding episodes.

AMT-060 has demonstrated a very low screening failure rate, and we have presented clinical data demonstrating that the presence of pre-existing anti-AAV5 neutralizing antibodies (NABs) does not predict the potential efficacy of AAV5-mediated gene transfer in patients with hemophilia B (view press release).  This development potentially expands the applicability of AAV5 gene therapies to nearly all hemophilia B patients. We believe these factors contribute to making AAV5 a potential best-in-class vector for delivering gene therapies more effectively and safely to a greater portion of patients in need of treatment.

The proprietary elements of AMT-060, including our fully-humanized FIX gene cassette and AAV5 vector, are the only gene therapy components clinically demonstrated in hemophilia B to be safe, effective, and durable for up to seven years.  These factors, along with our commercial-scale manufacturing capabilities, differentiate AMT-060 from other hemophilia gene therapies in development, and we look forward to advancing our program into a late-stage clinical study.


Hemophilia A

The Company is in pre-clinical evaluation for a gene therapy to treat hemophilia A.  We expect to provide an update on this program in 2017.