What are we going to see from gene and cell therapy in the near future?

While the potential for these early gene therapy successes to be extrapolated to other conditions and patient populations is exciting, next-generation technologies are dramatically expanding the impact of these medicines on treating human disease. For example, a primary obstacle to broader application continues to be the immune response to gene delivery vectors and products of foreign transgenes. Accordingly, control of the human immune system is where some of the most impactful work will occur in the near future. For example, despite the striking success of many AAV-based gene therapies, as many as 50% of patients are currently excluded from treatment due to pre-existing immunity to the viral capsids. Recent advances and efforts currently in clinical trials have led to technological advances to circumvent this immune obstacle, such as engineering of modified AAV capsids that evade pre-existing neutralizing antibodies and methods for temporary clearing of antibodies from circulation. Immunosuppression regimens may also provide a means for both circumventing pre-existing immunity and avoiding adaptive immunity to the vector, which may enable subsequent re-dosing if necessary.