Galapagos NV, of Mechelen, Belgium, announced that its service division, BioFocus, has entered into a 3-year target discovery alliance with Arthrogen BV, of Amsterdam, The Netherlands, to develop innovative gene therapy targets for the treatment of patients with rheumatic diseases. The alliance focuses on identifying novel targets for gene therapy applications in rheumatoid arthritis (RA).

The BioFocus target discovery platform is based on adenoviruses that efficiently introduce human gene sequences into a wide variety of human cells to knock down specific proteins. High-throughput assays that model a selected human disease are then used to functionally select for those proteins that have a causative effect in the disease models. Arthrogen is working on the development of innovative local gene therapy for patients with RA. The stable transfer of a therapeutic gene to a joint, using adeno-associated virus (AAV) as the vector, allows intra-articular synthesis of the protein at the site of inflammation for a sustained period of time, without significant systemic side effects. Synoviocytes are easily reached via the intra-articular space and are relatively long-lived, thus representing an ideal target for gene therapy strategies.

Galapagos will receive an upfront payment as well as R&D funding and will be eligible for development milestones, with the total revenue potentially exceeding € 7.5 million ($9 million) per validated target. Additionally, Galapagos is entitled to receive royalties on any marketed products that may arise from the alliance.

— A. Techman