What's next for osteoarthritis gene therapy?

Interest in using gene therapy to treat osteoarthritis (OA) is growing and a number of clinical trials have been initiated. This commentary identifies three intersecting areas that need to be addressed for the field to move forward expeditiously. The first relates to lowering the cost of manufacturing clinical grade viral vectors, addressing various aspects of their deployment, and overcoming immune barriers to dosing and re-dosing. The second area requires an improved understanding of the pathophysiology of OA, including its stratification by endotype and phenotype. Coupled to the development of reliable biomarkers, this will enable the creation of personalized gene therapies, facilitate patient selection, and aid the identification of additional molecular targets. Moreover, progress in the early diagnosis of OA will enable administration of gene therapeutics at a stage when they are most likely to be successful. Finally, important issues with regard to financing and regulation are discussed. Top line data from two pivotal Phase III clinical trials are expected to be released this year. The findings from these trials will exercise considerable influence on the future development of the field.