Bristol-Myers Squibb, uniQure to Develop Gene Therapies

April 7, 2015

Bristol-Myers Squibb Company and uniQure N.V. announced an agreement that gives BMS exclusive access to uniQure’s gene therapy technology platform for multiple targets in cardiovascular diseases. According to a press release, 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. The agreement also includes the potential for target-exclusive collaboration in other disease areas. In total, the companies may collaborate on 10 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, the release said. Bristol-Myers Squibb will lead development and regulatory activities across all programs and be responsible for all research and development costs. Bristol-Myers Squibb will be solely responsible for commercialization of all products from the collaboration.

“Collaborating with uniQure, a clear leader in the field with an innovative and validated gene therapy platform, further strengthens our capability to bring forward transformational new therapeutics for difficult-to-treat diseases, including cardiovascular diseases such as heart failure,” said Carl Decicco, Ph.D., Head of Discovery, R&D, Bristol-Myers Squibb.

“This collaboration will accelerate the application of gene therapy for large patient populations suffering from heart diseases and will complement the further development of uniQure’s internal pipeline in two focus areas: liver diseases, including hemophilia, and CNS, including lysosomal storage diseases,” said Joern Aldag, CEO of uniQure.

The companies anticipate the collaboration to be effective during the second quarter of 2015. For the terms of the agreement, read the full release.