In the rapidly evolving field of adeno-associated virus (AAV) gene therapy, patient advocacy groups (PAGs) have emerged as critical partners driving scientific, operational, and regulatory progress. Their influence extends far beyond awareness and fundraising—they help shape trial design, regulatory engagement, and real-world access.
This white paper examines how PAGs have accelerated the path to approval for seven AAV therapies, illustrating the power of coordinated, patient-centered collaboration to bridge scientific discovery and patient impact.
What’s Inside
- How advocacy groups drive early research, data generation, and translational insights.
- Real examples of PAG contributions across seven approved AAV therapies.
- Key opportunities for sponsors to strengthen regulatory, clinical, and access strategies through structured advocacy collaboration.
- A practical checklist for integrating PAG engagement throughout development.
Download the Whitepaper
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