BMS, uniQure collaborate to develop gene therapies for CVDs

BMS will lead development and regulatory activities across all programs and be responsible for all research and development costs.


Mr Joern Aldag, CEO, uniQure (Photo Courtesy:

The companies anticipate the collaboration to be effective during the second quarter of 2015.

The collaboration includes uniQure's proprietary gene therapy program for congestive heart failure that is intended to restore the heart's ability to synthesize S100A1, a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction.

Beyond cardiovascular diseases, the agreement also includes the potential for target-exclusive collaboration in other disease areas.

In total, the companies may collaborate on ten targets, including S100A1.

uniQure will lead discovery efforts and be responsible for manufacturing of clinical and commercial supplies using its vector technologies and its industrial, proprietary insect-cell based manufacturing platform.


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