We are continuously advancing our broad pipeline of innovative gene therapies to bring new treatments to patients with severe genetic diseases.
Congestive heart failure (CHF) is the disability 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.
Based on research and development at our German subsidiary uniQure GmbH, formerly the cardiology gene therapy company InoCard, uniQure is developing a novel gene therapy for the one-time treatment of CHF. This gene therapy candidate, AAV-S100A1 is a product designed to selectively restore cardiac deficiency of the calcium-binding protein S100A1, a master regulator of myocardial function. It is a 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 in vivo beneficial effects on contractile force, growth control of heart muscle cells and rhythm stability of the heart and is also able to adapt the heart’s energy supply to increased cardiac output. AAV-S100A1 has proven long-term therapeutic efficacy, safety and reduced mortality in a human-relevant in vivo heart failure model compatible with clinical drug regimens (view publication).
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