Biotechnology Merger to Spur Development of a Gene Therapy Approach for Treatment of Congestive Heart Failure

It is a rapidly progressing disease affecting some 26 million people worldwide, with patients suffering from severe heart failure facing a five-year mortality rate of over 50%.

Currently there is no cure for CHF, but this may change following the purchase of InoCard GmbH (Heidelberg, Germany) by uniQure N.V. (Amsterdam, The Netherlands). InoCard GmbH, a privately held early-stage biotechnology company, has developed an innovative AAV (adeno-associated virus)-based gene therapy approach for the long-term treatment of CHF, targeting the calcium-binding protein S100A1. The Dutch company uniQure has developed a modular platform to rapidly bring new disease-modifying therapies to patients with severe disorders.

The target of the new partnership is to develop a commercially viable gene therapy approach to restore S100A1 levels in CHF patients. Research has shown that administration of S100A1 to CHF patients has 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. In a porcine heart failure model, treatment with InoCard's gene therapy AAV-S100A1 demonstrated a 12-month survival rate of 90%.

"The acquisition of InoCard is a further demonstration of uniQure's strategy to access the best early-stage programs in our industry and accelerate their development by the application of our proven modular platform," said Jörn Aldag, CEO of uniQure. There is strong scientific rationale that addressing calcium dysregulation leads to an astounding effect in congestive heart failure. We believe that together we will deliver the best-in-class treatment for congestive heart failure."

"Despite the continuously growing prevalence of CHF, there have been no therapeutic innovations in decades. InoCard has successfully laid the basis for the development of a long-term, causative treatment of this devastating disease," said Dr. Patrick Most, founder of InoCard. "We believe that combining our promising S100A1 therapy with uniQure's capabilities in innovating safe and effective gene therapies has the potential to transform the treatment of cardiovascular diseases."

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