RARE Drug Development Symposium Marks 10th Anniversary with Dedicated Pitch Sessions and The Theme and Call to Action to Align Advocates, Science and Industry

BOSTON, July 28, 2026 /PRNewswire/ -- Global Genes, in partnership with The Rosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center (RSZ TNC) at Boston Children's Hospital and the Termeer Institute, is proud to announce the 10th Annual RARE Drug Development Symposium (RDDS) taking place September 9 – 11, 2026 in Boston. This landmark decennial event brings together the rare disease ecosystem to foster hands-on, collaborative learning designed to translate patient-lived experiences into research readiness and strategic therapeutic development.

This year's milestone symposium centers on the theme:

"Accelerating Rare Disease Progress: Aligning Advocates, Science & Industry"

To mark its decade of impact, the 2026 symposium is expanding its programming to include a dedicated day for Pitch Sessions. This new addition provides a critical platform for rare disease advocacy leaders to pitch their research portfolios, natural history data, and clinical trial readiness directly to biotech companies, clinical researchers, and venture philanthropists, de-risking pipelines and driving investment to underserved disease areas.

A Decade of Impact and Collaboration

For ten years, RDDS has served as a vital catalyst for rare disease advocates who are ready to lead, build vibrant ecosystems, partner with industry, and advance drug development pipelines.

"For a decade, the Rare Drug Development Symposia has brought together the patient community, academia and industry to advance advocate-partnered research, and this reflects the collaboration that underpins meaningful breakthroughs in rare disease," said Christophe Hotermans, SVP, Medical Affairs, Alexion, AstraZeneca Rare Disease, the Visionary Sponsor for this year's symposium. "We are committed to partnering with the rare disease community to advance research and enable innovation that reflects patient needs and real-world impact."

"Ten years ago, RDDS started as an experiment in bringing advocates and industry into the same room. Today it's a proven launchpad for patient-led and patient-partnered research," said Charlene Son Rigby, CEO of Global Genes. "Rare disease progress depends on advocates who know how to navigate research, build the right partnerships, and drive development forward. Ten years in, that's the commitment RDDS continues to deliver on."

Symposium Highlights & Interactive Programming

Building on a legacy where over 90% of past attendees reported significant increases in research knowledge and organizational confidence, the 2026 agenda is structured to deliver immediate, actionable value. Attendees will participate in:

• Pitch Sessions: A new main-stage and track-based feature where advocates pitch their research programs and data portfolios directly to expert judges and industry representatives to secure vital collaborations and investment.
• Expert Office Hours: Highly sought after, private 1-on-1 consultations giving attendees personalized, direct access to top minds in drug development, regulatory affairs, data strategy, and market access.
• Data & Drug Development Process Deep Dives: Practical case studies and interactive breakouts focused on building regulatory-grade datasets (natural history, biomarker, and Patient Reported Outcomes) and analyzing real-world rare disease drug approvals.
• Next-Level Trial Readiness: Advanced frameworks detailing how patient organizations can actively shape trial design, influence endpoint development, and sustain multi-stakeholder momentum across the research lifecycle.

This year's RDDS is made possible with the generous support of our sponsors: Alexion-AstraZeneca Rare Disease, BridgeBio, Travere Therapeutics, Astellas Gene Therapy, BioCryst, Catalyst Pharmaceuticals, Bial, Dyno Therapeutics, RTW Foundation, AlphaRose Therapeutics, Viralgen, and our Media Partner, RARE Revolution Magazine.

For more information regarding registration and the agenda for the 2026 RARE Drug Development Symposium, please visit the Global Genes website.

About Global Genes

Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients, their families and disease communities globally. For nearly two decades, we've equipped rare disease patients and advocates with tools, training and support – to connect patients with needed resources, activate communities and advance research. Global Genes serves the more than 400 million people around the globe, and the nearly one in 10 Americans affected by rare diseases. With over 800 patient advocacy group members in our Global Advocacy Alliance, we work with patient advocates, industry partners and academia to build vital ecosystems to progress critical work in rare disease. Learn more at www.globalgenes.org.

SOURCE RARE-X