Gene therapy has the potential to transform today’s healthcare by offering treatments that actually cure severe diseases. CombiGene’s long-standing ambition has been to contribute to this development, and we are working intensively to find additional research assets that we can add value to through preclinical and clinical development. The COZY pain program is the latest example of how we are working to broaden our project portfolio, but there are several other areas that also are of interest, including diseases of the central nervous system, endocrine[1] diseases and genetic muscle diseases.
There are several perspectives from which one can view the development of new gene therapies and CombiGene’s role. The first perspective that makes gene therapy so interesting is, of course, the potential of curing severe diseases. It is this goal that drives researchers all over the world, and it is this that gives us at CombiGene the energy to keep moving our own development forward every day. At the same time, there is of course a commercial perspective to our work – by successfully developing our projects, we also want to create significant financial value for the company and our shareholders, and a broad portfolio of projects increases the chances of achieving just that.
CombiGene is currently working intensively to find interesting new projects to complement our current project portfolio. The evaluation of potential projects is a structured and thorough process based on a number of key criteria. The work includes a review and analysis of intellectual property issues, preclinical data, intended contract structure, size of patient population and medical need, competitive situation, and the project’s commercial conditions. In this work, it is also important that we understand that it is not only we who evaluate potential collaborations – our potential partners naturally evaluate CombiGene. It is therefore very satisfying to be able to demonstrate our strengths and our significant experience in the development of research assets and commercial capabilities.
Over the past five years, CombiGene has built up an infrastructure with considerable strength. Of our ten employees, all of our project managers, six employees, have PhDs in relevant subject areas and we have step by step established an impressive international network of leading companies. Today, we can offer potential partners top-class preclinical project management, world-leading networks of CDMO and CRO partners. We also have established relationships with regulatory advisors, which is extremely valuable when we design clinical studies. Through the agreement with Spark Therapeutics, we have also demonstrated a proven ability to enter into large agreements with leading Big Pharma companies.
[1)Endocrine diseases include diseases of the body’s hormone-producing glands, such as the gonads, pituitary gland, pancreas, and thyroid gland. Diseases in the area also include errors in metabolism – also known as metabolism.
