Proprietary genetic platform for OA disease modification
The goal of our proprietary osteoarthritis (OA) gene therapy platform is to offer the prospect of long-term treatments restoring the physiological expression of dysregulated proteins after a single intra-articular administration.
Most common OA therapies result in short-term mitigation of symptoms and are associated with increased risk of medical complications. Intra-articular viral-based gene therapy is an attractive strategy for OA because it allows for long-term and site-specific action directly to the diseased joint, thus enhancing its effectiveness while greatly lowering the risk of adverse events and reducing costs.
Adeno-associated viruses (AAVs) can transduce chondrocytes in diseased joints more effectively than other viral vectors, most likely thanks to their smaller capsid size and their maintenance as stable episomes. Gene therapy with AAV has an attractive vector safety profile and the ability of long-term expression in tissues with low mitotic rate.
Despite promising AAV gene therapies under development in OA, the AAV vectors still suffer from many limitations. The most important are the host immune response and the efficacy and specificity of transgene expression in the joint tissue. Therefore, there are still some technological hurdles that need to be overcome to improve transduction efficiency in the joint, to increase the stability of transgene expression and off-target expression and to escape neutralizing immune responses.
At Rottapharm Biotech we are working to increase the natural tropism of AAV towards joint tissues and on the optimization of the transgene sequences to improve the clinical translation of AAV gene therapy for OA. In addition, we are prioritizing effective human targets for the development of innovative cures for selected OA phenotypes.