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 led by AMT-126, an S100A1 gene therapy targeting congestive heart failure.

Congestive Heart Failure

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 CHF by selectively restoring cardiac deficiency of the calcium-binding protein S100A1, a master regulator of myocardial function. 

Congestive heart failure (CHF) is the inability of the cardiac muscle to provide sufficient circulatory support both at rest and during exercise. CHF is a rapidly progressing disease affecting 26 million people worldwide, with patients suffering from severe heart failure facing a 5-year mortality rate of over 50%. According to the American Heart Association, the prevalence of CHF is expected to double or triple by 2030. Currently, there is no effective long-term or causative treatment for this disease.

uniQure is developing a novel gene therapy for the one-time treatment of CHF. This gene therapy candidate, AMT-126, is designed to selectively restore cardiac deficiency of the calcium-binding protein S100A1, a master regulator of myocardial function. It is intended to be a potential one-time therapeutic intervention in advanced CHF patients who often continue to decline despite standard of care treatment. S100A1 is downregulated in these patients, and administration of S100A1 has demonstrated potential beneficial effects in vivo on contractile force, growth control of heart muscle cells and rhythm stability of the heart. 

AMT-126 (AAV-S100A1) has shown long-term therapeutic efficacy, safety and reduced mortality in a human-relevant in vivo heart failure model compatible with clinical drug regimens. uniQure and Bristol-Myers Squibb continue to conduct IND-enabling studies for AMT-126. In addition to AMT-126, uniQure and Bristol-Myers Squibb have initiated work on three additional targets designated under the collaboration.