AAV gene therapy is finally hitting its stride. However, disruptive advancements for the next generation of these gene therapies, and how we price them, will not come until we innovate the payload, the capsid, patient pre-treatment, manufacturing, and develop creative policies. I will discuss our work in these areas and that of the broader field, as well as highlight opportunities for innovation to address key unresolved areas in the field of AAV gene therapy.
Challenges and Opportunities in AAV Gene Therapy
Assistant Adjunct Professor, Biochemistry and Biophysics, School of Medicine
University of California, San Francisco