Genetically Targeted Technology: Patient Perspectives and Future Promise Briefing
This Capitol Hill briefing explored the life-changing potential of Genetically Targeted Technology (GTT) through the eyes of the patients it serves. Building on our March 2025 program, this session moved beyond clinical data to highlight the lived experiences of families at the forefront of medical innovation.
GTT represents a transformative class of treatments—including RNA-based approaches and RNAi—that address the root causes of disease rather than merely managing symptoms. While these technologies are designed to target the genetic drivers of disease, their success is measured in human milestones. On June 23rd, we heard directly from patients and advocates about the future of genetic medicine and learn how these therapies are transforming care for both rare and common conditions. To learn more, check out our infographic from the briefing.
Moderator: Michael Beard, Vice President of Federal and Global Public Policy, NORD
Speakers:
- Adrienne Weinstein, Patient Advocate, CMT Research Foundation
- Giselle Benmoin, Patient Advocate, Partnership to Advance Cardiovascular Health (PACH)
- Nichole Goble, Director of Community Initiatives, Caregiver Action Network
- Jeff Coller, PhD, Bloomberg Distinguished Professor of RNA Biology and Therapeutics; Johns Hopkins University
When: Tuesday, June 23, 2026 from 8:30 to 10am
Where: 2060 Rayburn House Office Building – 45 Independence Ave SW, Washington, DC 20515
For questions, contact us at [email protected]
Research!America’s “Genetically Targeted Technology: Patient Perspectives and Future Promise” briefing was supported in part by Alnylam Pharmaceuticals, Novartis, and Ultragenyx.