Promoting Patient Access to Cell and Gene Therapies: Highlights from the IGT and ARM Capitol Hill Briefing

On June 17, the Institute for Gene Therapies (IGT), in partnership with the Alliance for Regenerative Medicine (ARM), convened a panel discussion on Capitol Hill to spotlight the promise and challenges of cell and gene therapies. The event, “Promoting Innovation and Access to Cell and Gene Therapies,” brought together leading scientists, patient advocates, and policy experts to discuss how these groundbreaking treatments are transforming lives – and what must be done to ensure access for all who need them.

The event featured:

• Erik Paulsen, Chairman of IGT (Moderator)

• Erica Cischke, Vice President of Government Affairs, ARM

• Nate Plassman, Parent Advocate whose son received gene therapy for Duchenne Muscular Dystrophy

• Dr. Kiran Musunuru, Professor of Cardiovascular Medicine & Genetics, University of Pennsylvania

• Annie Virginia Eggiman, Chief Regulatory Officer, Tessera; ARM Board Member

• Maria Kefalas, Co-founder, Calliope Joy Foundation, Cure MLD

Dr. Musunuru, who worked with the first patient to receive a personalized CRISPR gene editing therapy, opened with a powerful overview of these treatments, explaining how they differ from traditional pharmaceuticals. He emphasized their unique potential:

Gene therapies, he explained, offer the hope of “one and done” solutions – potentially curing diseases with a single treatment, rather than requiring lifelong management.

Panelists like Nate Plassman and Maria Kefalas shared personal stories underscoring the real-world impact of gene therapies on patients and families. Their experiences brought home the urgency of expanding access to these life-changing innovations, and the long-term cost implications of doing so.

Annie-Virginia Eggiman and Erica Cischke discussed the regulatory landscape and the importance of equipping agencies like the FDA to keep pace with scientific advances. They also highlighted ongoing efforts in Congress, including the re-introduced Accelerating Kids’ Access to Care Act to support innovation while ensuring patient access.

While scientific breakthroughs are accelerating, policymakers, regulators, and advocates must work together to remove barriers to access. As gene therapies move from the lab to the clinic, ensuring affordability and availability for all patients is critical.

As Dr. Musunuru reiterated, we cannot stop here:

Watch the full discussion here.