From Trials to Triumph: Patient Advocates Share the Path from Research to Approval

One standout session at ASGCT’s Empowering Patients Summit was the panel The Journey from Early-stage Trials to Approval, which brought together leaders from the front lines of patient advocacy. This live discussion was moderated by Dr. Kim Goodspeed, a clinician and researcher deeply committed to rare disease families, the panel featured Brendan Hayes (National Bleeding Disorder Foundation), Amanda Moore (Angelman Syndrome Foundation), Kelly Brazzo (CureLGMD2i Foundation), and Sarah Cortell Vandersypen (United MSD Foundation). 

Each panelist shared their organization’s unique journey to advance gene therapies—stories marked by perseverance, smart strategy, and deep emotional investment. Their collective message was clear: patient advocates are not just part of the CGT journey—they are catalysts for it. 

Starting with Community-Led Science 

A major theme of the discussion was how progress often begins with grassroots leadership. In areas where commercial interest is minimal or nonexistent, it’s usually patient foundations that fund the first studies, build the first registries, and push the science forward. Amanda Moore described how the Angelman Syndrome Foundation supported essential early-stage research that laid the groundwork for later clinical efforts. 

These initial investments don’t just enable science—they attract attention, signal readiness, and build the kind of momentum that gets therapies into development. 

Bridging the “Valley of Death” 

Many rare disease organizations face the infamous “valley of death”—the gap between promising lab findings and the launch of clinical trials. Sarah Cortell Vandersypen shared how the United MSD Foundation funded toxicology studies and regulatory consulting to close this gap and present a more complete, de-risked opportunity for potential partners. 

Brendan Hayes encouraged fellow advocates to think like venture capitalists—prioritizing projects that are scientifically sound and fundable, and staying laser-focused on long-term impact. 

Collaborating with Industry and Regulators 

The panel emphasized the importance of becoming true partners to industry and regulators. Kelly Brazzo spoke about CureLGMD2i’s work with the FDA to ensure trial endpoints reflected real patient needs. Amanda Moore echoed this, highlighting the power of advocates to shape clinical trial design—not just participate in it. 

Early and ongoing collaboration was identified as key to reducing friction, building trust, and ensuring that programs move forward efficiently and ethically. 

Leading with Heart 

While strategic thinking and scientific literacy are crucial, what ultimately drives this work is love—love for family, community, and future generations. “This work is not optional—it’s personal,” said Sarah Vandersypen. “We do it because we must.” 

Calls to Action for Patient Advocacy Groups 

For advocates who couldn’t attend the session, here are 5 key takeaways to guide your path forward: 

• Invest early and strategically. Even small grants can spark critical early data. Choose projects that move your community closer to clinical readiness.  

• Build infrastructure. Patient registries, biobanks, and natural history studies are invaluable assets that attract biotech partners and accelerate trial design. 

• Close the gaps. Fund the hard-to-finance steps—like toxicology or regulatory prep—that bridge lab research to clinical trials.  

• Be a proactive partner. Engage with regulators and companies early. Your voice helps shape trials that are meaningful and feasible for patients. 

• Lead with urgency—and sustainability. Emotional drive is essential, but long-term impact comes from building strong teams, clear plans, and collaborative networks. 

A Note to ASGCT Members 

For those in the field: the progress of gene therapies depends not only on innovation, but on the vision and commitment of patient advocates. Engage them early, listen closely, and build with them—not for them. 

In case you missed it, you can listen to the full panel discussion on demand and share these insights with anyone else who may benefit!  

Ali is ASGCT's Senior Patient Outreach Manager.

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