We are experiencing seriously increasing interest in gene therapies and related capabilities. Our business model is predicated on partnerships and licensing as a key source of funding. As a consequence we are actively pursuing various business opportunities – both out- and in-licensing.
Partners can license from our portfolio programs in Hemophilia, Porphyria, GDNF-related neuro-degenerative diseases (Parkinson’s, Multiple Systems Atrophy, Huntington’s and hearing loss), and Sanfilippo (Lysosomal Storage Disease). We are open for early-stage collaborations (risk-sharing, option-type deals).
We leverage the preclinical and clinical work and expertise of academic partners in many orphan diseases and combine this with our proprietary gene delivery and manufacturing platform and clinical development and regulatory capabilities to balance risks and reduce time-to-market significantly. For example, our agreements with CIMA/Digna Biotech in Spain and with Institut Pasteur in France, give uniQure access to cutting edge medical research while providing them an expertise which academic institutes normally do not possess.
A biotechnological company focused on developing new drugs from CIMA (Center of Applied Medical Research) of the University of Navarra.