Gene Therapy:


uniQure and its partner, Bristol-Myers Squibb, have taken the leadership position in developing gene therapies in cardiovascular diseases.

With our collaboration we are building a portfolio of gene therapies focused on cardiovascular disease.

In April 2015, uniQure signed a strategic partnership with Bristol-Myers Squibb (view press release) to discover and develop novel gene therapies to treat a range of cardiovascular diseases, initially focused on the one-time treatment of congestive heart failure with the gene therapy candidate, AMT-126.

uniQure and our collaboration partner, Bristol-Myers Squibb, completed a heart function proof-of-concept study of the first target, AMT-126 in a pre-clinical animal model of heart failure. The study demonstrated DNA delivery and expression of S100A1 in the myocardium, thereby validating uniQure’s vector delivery platform in the animal model. The data did not show a benefit on heart function at six months, and consequently, the Joint Steering Committee has chosen to discontinue work on S100A1. Bristol-Myers Squibb intends to replace the S100A1 collaboration target with another cardiovascular target. Bristol-Myers Squibb and uniQure will continue working on the other collaboration targets under the collaboration.

Gene therapy cardiovascular sidebar