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8th annual RARE Drug Development Symposium – May 1-3, 2023

Global Genes and Orphan Disease Center at the University of Pennsylvania to hold 8th annual RARE Drug Development

ALISO VIEJO, Calif.— The rare disease patient advocacy organization Global Genes and the Orphan Disease Center at the University of Pennsylvania will host the 8th Annual RARE Drug Development Symposium to equip patient advocates with the knowledge, skills, and connections they need to support the advancements in therapy development for their communities

This year’s symposium will focus on collaboration as the foundation for success in rare disease research. Attendees will meet individuals, advocacy leaders, industry executives, and research experts who have successfully navigated the therapy development process, and have formed successful research collaborations, during the two days of panel discussions, hands-on workshops, and one-on-one meetings with drug development experts.

Using case studies and real-world examples, advocates will discover how they can leverage the power of data to drive research, accelerate and expand research opportunities using new technologies, and collaborate to advance research strategies and fill critical gaps. A full agenda of the two-day event can be found here. The program concludes with a walk through the framework of the RARE-X Research Readiness Program, an initiative that helps identify needs in a community’s clinical trial readiness, and – with input from clinical experts and drug developers – prioritize the gaps for investment. Attendees will come away with strategies they can apply to their own endeavors, whether they have just started developing their research strategy or are well into their journey.

“Rare disease advocates know that they can’t sit and wait until a drug company decides to develop a therapy for their disease. Patient advocates are playing a growing role in not only funding foundational research, but also in driving translational research and carrying drug development forward to a point where pharmaceutical and biotech companies are willing to in-license programs,” said Charlene Son Rigby, CEO of Global Genes. “We are working to demystify the therapy development process for advocates, foster connections, and help them redefine what is possible.”

The event will be held Monday, May 1, 2023, through Wednesday, May 3, 2023, at the Sheraton Philadelphia Downtown hotel at 201 N. 17th Street, Philadelphia, Pa. A pre-conference webinar on how to support your community before, during, and after advanced therapy trials will be held in April. There will also be a post-conference webinar on key considerations when building a global registry.

“We are in an unprecedented time of drug development with advances in genetic medicine enabling potential cures for many rare diseases,” said Monique Molloy, Executive Director, Orphan Disease Center at the University of Pennsylvania. “The RARE Drug Development Symposium offers a unique opportunity to bring together patient advocates, clinicians, researchers, and drug developers to learn from each other, lay the groundwork for collaboration, and work together to accelerate the development of much needed therapies.”

The RARE Drug Development Symposium is sponsored by Horizon Therapeutics, Pfizer, Travere Therapeutics, Genentech, Pharming, Ultragenyx, Ovid Therapeutics, Spark Therapeutics, Amicus, BridgeBio and REGENXBIO. For more information, visit

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of that mission, Global Genes connects, empowers, and inspires the rare disease community to stand up, stand out, and become more effective on their own behalf⁠—helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. Global Genes serves more than 400 million people around the globe, and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE or visit the Resource Hub.

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