NORD’s 2025 Rare Diseases & Orphan Products Breakthrough Summit to Spotlight CEOs, Investors, and Regulatory Leaders Driving Innovation

WASHINGTON, Aug. 21, 2025 /PRNewswire/ — The future of rare disease care and innovation will take center stage in the nation’s capital this fall. The National Organization for Rare Disorders (NORD^®) will host its annual Rare Diseases & Orphan Products Breakthrough Summit^® from Oct. 19-21 at the Grand Hyatt, convening more than 900 patient advocates, researchers, regulators, and industry leaders to accelerate progress for the over 30 million Americans living with rare diseases.

This premier event is where science, policy, capital, and patient voices converge to shape the next generation of rare disease diagnosis, treatment, and care.                           

“The NORD Breakthrough Summit isn’t just a gathering — it’s a catalyst for solving one of America’s most urgent public health challenges,” said Pamela K. Gavin, NORD Chief Executive Officer. “One in 10 Americans lives with a rare disease, a crisis hiding in plain sight. The conversations here influence how treatments are developed, funded, and delivered. This is where policy meets science, innovation meets investment, and patient voices drive the change we need.”

This year’s theme, “From Voices to Breakthroughs,” underscores the power of lived experience in shaping scientific and policy progress. The three-day summit will feature:

• Community keynotes from youth and adult patients, caregivers, and advocates
• Cutting-edge scientific sessions and strategic policy dialogues
• Insights from top regulatory leaders at the U.S. Food and Drug Administration (FDA), National Institutes of Health (NIH), and Centers for Medicare and Medicaid Services (CMS)
• Capitol Hill updates from members of the Rare Disease Congressional Caucus

Shaping the future of rare disease innovation, the C-Suite Perspectives panel will bring together leaders who are redefining what is possible, featuring conversations with:

• Miguel Fernández Alcalde, President, EMD Serono
• Christophe Arbet-Engels, MD, PhD, Chief Medical Officer, X4 Pharmaceuticals
• Catherine Owen Adams, Chief Executive Officer, Acadia Pharmaceuticals
• Pamela Gavin, President and Chief Executive Officer, NORD

With research funding under pressure, a special closing plenary panel will explore how to sustain and scale investment in rare disease research that leads to breakthroughs. Participants include:

• David Scheer, President, Scheer & Co. (moderator)
• Martin Mackay, Ph.D., Co-Founder, Rallybio
• Vinod Mitta, Director, Seamless Care Models Group, CMS Innovation Center, U.S. Health and Human Services (HHS)
• Sean P. Nolan, Chief Executive Officer, Taysha Gene Therapies
• Maha Radhakrishnan, M.D., Executive Partner, Sofinnova Investments
• Philip Ross, Vice Chairman, Jefferies LLC
• Stephen Squinto, Ph.D., Chief Investment Officer, J.P. Morgan Life Science Private Capital
• David Tischler, Chief Executive Officer, Stealth Mode

“We are witnessing unprecedented scientific momentum, but innovation alone is not enough. Without bold investment and policy leadership, too many promising treatments will never reach the rare disease patients who need them most,” Gavin said. “We are deeply grateful to the investors who recognize this urgency and continue to fuel progress. Our mission is to ensure the pace of funding matches the pace of discovery. We will not stop pushing until it does.”

The Breakthrough Summit is open to everyone interested and committed to rare disease patient care, research, drug development, and advocacy. Register by Sept. 8 to secure early-bird pricing: https://nordsummit.org/. See the full agenda here:  https://nordsummit.org/agenda/.

Media Registration:
If you are a member of an accredited media organization, please contact media@rarediseases.org to register for the NORD Breakthrough Summit.

About the National Organization for Rare Disorders
Founded in 1983, the National Organization for Rare Disorders (NORD^®) is a leading independent, nonpartisan, nonprofit and patient advocacy organization dedicated to improving the health and lives of over 30 million Americans living with rare diseases. In partnership with more than 350 disease-specific member patient organizations, NORD drives progress in rare disease research, care, and policy. Learn more at https://rarediseases.org/

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SOURCE National Organization for Rare Disorders (NORD®)