Accelerating AAV Gene Therapy Development
Working with multiple stakeholders, including patient groups, industry, and regulators, we are seeking to develop and propose a framework that can be applied to AAV gene therapies to facilitate the use of the FDA accelerated approval pathway in order to expedite patient access to such treatments. We are working to identify different categories of AAV gene therapies that target the underlying monogenetic changes that cause disease and to propose generalized approaches that clarify the evidence needed to support an approval.
Feedback to the proposed framework, drafted by REGENXBIO and Solid Biosciences, can be sent to info@pathwaydevelopmentconsortium.org.