Consortium Mission
Through collaborative work and partnership of patients, industry, regulators, academia, payers and other stakeholders, construct an ideal pathway to ensure that all born with serious genetic conditions can find their way to effective gene therapies.
Pathway Development
Moving AAV Gene Therapies Forward
Advocate
Partner with patients to lead patient engagement in AAV gene therapy for rare diseases.
Collaborate
Work with the rare disease community and its partners to improve diagnosis rates and increase the availability of innovative AAV gene therapies.
Navigate
Advance the field of AAV gene therapy by identifying solutions to barriers and work toward defining a clear regulatory roadmap.
Integrate
Build a model AAV gene therapy pathway for patients and families that is replicable and scalable.
