#USA #Boston #Rare Disease #Global Genes #RARE Symposium

10th Annual RARE Drug Development Symposium

The RARE Drug Development Symposium (RDDS) held by Global Genes, in collaboration with The Rosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center (RSZ TNC) at Boston Children's Hospital and the Termeer Institute, is set to celebrate its 10th anniversary from September 9 to 11, 2026, in Boston. This milestone event is a gathering designed to foster innovative cross-sector collaboration focused on rare disease research and the development of effective treatments.

Event Highlights

This year's symposium carries the theme "Accelerating Rare Disease Progress: Aligning Advocates, Science & Industry". To commemorate a decade of impactful work, the RDDS introduces Pitch Sessions dedicated to giving rare disease advocacy leaders the opportunity to present their research initiatives and data to leading biotech companies, clinical researchers, and philanthropic investors. This new platform is expected to mitigate risks associated with drug development and to channel investments into under-supported diseases.

A Decade of Achievements

For ten years, the RDDS has significantly contributed to the rare disease landscape, providing a venue where advocates, researchers, and pharmaceutical companies can interact meaningfully. “The symposium has been a cornerstone for the patient community, academia, and industry to collaborate on research that addresses real-world needs,” remarked Christophe Hotermans, Senior Vice President of Medical Affairs at Alexion, AstraZeneca Rare Disease, who is this year’s Visionary Sponsor.

Charlene Son Rigby, CEO of Global Genes, highlighted the evolution of the symposium, stating, "What started as an experiment in collaboration between advocates and industry has now evolved into a vital launchpad for patient-driven research. The commitment to ushering rare disease advancements is stronger than ever.”

Engaging and Educational Sessions

Building on previous successes, where over 90% of attendees reported increased knowledge and confidence in research, the agenda for the 2026 symposium is designed to provide attendees with actionable insights.

• - Pitch Sessions: A main-stage event where advocates present their research portfolios and development plans to expert judges, gaining valuable feedback and potential funding.
• - Expert Office Hours: Unique opportunities for one-on-one consultations with leading experts in drug development, data strategy, and regulatory practices.
• - Deep Dives into Data and Development: Sessions dedicated to developing regulatory-compliant datasets and case studies of successful rare disease interventions, aimed at empowering advocacy organizations.
• - Trial Readiness Frameworks: Workshops that help patient organizations influence trial design and endpoint development for improved research outcomes.

Acknowledgment of Sponsors

The success of the 2026 RDDS relies heavily on the contributions of sponsors, including major industry leaders like Alexion-AstraZeneca Rare Disease, BridgeBio, and Travere Therapeutics. These partnerships illustrate the importance of collaboration in driving rare disease research forward.

As we look ahead to the upcoming symposium, there's great anticipation about what innovations and connections it may yield for the future of rare disease therapies. For further details on registration and agenda, visit the Global Genes official website.

About Global Genes

Global Genes is a non-profit organization focused on providing support to the rare disease community. With nearly two decades of experience, they have equipped patients and advocates with essential resources for research collaboration and advocacy. Their mission is to impact the lives of over 400 million individuals affected by rare diseases globally, making strides in research and community empowerment. To learn more, please visit www.globalgenes.org.