UniQure and 4D Sign Broad Collaboration

Amsterdam, the Netherlands, and Berkeley, CA, January 19, 2014 — uniQure B.V., a leader in human gene therapy, and 4D Molecular Therapeutics LLC, a pioneer in next-generation gene delivery technologies, today announced a broad collaboration and license agreement under which uniQure will gain exclusive access to 4D’s adeno-associated viral (AAV) vector discovery and optimization technology for gene delivery to the central nervous system and liver. No financial details were disclosed.

“In order to treat diseases where high gene expression levels may be required for therapeutic benefit, more potent gene delivery vehicles or vectors than are available today may be needed. As such we have entered into a collaboration and license agreement with 4D Molecular Therapeutics who use directed evolution techniques to produce next generation vectors.  Directed evolution involves an iterative selection process where libraries of mutant AAV variants are screened for optimal properties with the potential to produce vectors with improved potency as compared to vectors based today on naturally occurring serotypes of AAV,” said Harald Petry, CSO of uniQure.

“uniQure’s strategic goal is to provide disease modifying treatments for many severe and chronic diseases through single gene therapy interventions. We believe 4D’s capabilities are world leading and will provide a powerful additional tool in our modular toolkit to efficiently develop new gene therapies for a wider variety of indications,” said Jörn Aldag, CEO of uniQure. “We look forward to a close collaboration with the 4D team under the leadership of David Schaffer to expand our pipeline of gene therapy product candidates.”

“We established 4D with the objective of applying our proprietary Directed Evolution AAV variants platform to design and develop transformative gene therapies. As the global leader in AAV vector discovery, our proprietary processes and vector libraries are designed to transform the efficacy, safety and dosing of genetic therapy products,” said Dr. David Schaffer, co-founder of 4D Molecular Therapeutics and Professor of Chemical and Biomolecular Engineering, Bioengineering, and the Helen Wills Neuroscience Institute at the University of California, Berkeley.  “We believe that bringing our expertise together with uniQure’s leadership in the development, manufacturing and commercialization of gene therapy products will be a powerful and fully-integrated combination,” said Dr. David Kirn, co-founder of 4D Molecular Therapeutics.

About uniQure

uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product Glybera. www.uniqure.com

About 4D Molecular Therapeutics

Our vision is to design, develop, and commercialize transformative gene therapeutic products from our proprietary “directed evolution” AAV variants platform for serious unmet medical conditions in partnership with complimentary biopharmaceutical companies.www.4dmoleculartherapeutics.com