Global Genes and the Orphan Disease Center of the University of Pennsylvania Host 7th Annual RARE Drug Development Symposium


Symposium Convenes Leading Advocates, Researchers and Clinicians To Tackle Critical Issues Facing Innovators in Rare Disease

ALISO VIEJO, Calif.–(BUSINESS WIRE)–Global Genes, a leading rare disease patient advocacy organization, today announced its 7th annual RARE Drug Development Symposium (RDDS), in partnership with the Orphan Disease Center at the University of Pennsylvania. The two-day event will bring together rare disease advocacy leaders and researchers to identify barriers to research, create solutions, and accelerate progress for the rare disease community.

Caribbean News Global GG_Logo Global Genes and the Orphan Disease Center of the University of Pennsylvania Host 7th Annual RARE Drug Development Symposium

From June 8-10, leaders will meet in-person to engage in peer-to-peer, interactive small group workshops and discussions covering topics such as accelerated clinical trials, the use of AI-driven screening platforms, emerging models and partnerships, and fostering successful connections between rare disease stakeholders.

Alongside the workshops, the symposium will feature speakers from throughout the rare disease community and provide networking opportunities designed to create meaningful connections and deepen relationships between advocates, researchers, and leaders.

Each morning, industry leaders will host fireside chats about their experiences in their areas of specific expertise.

Thursday Fireside Chat: What are the Keys to Accelerating Rare Disease Research?

The speakers will include:

  • Paul Howard, PhD – Senior Director of Public Policy, Amicus Therapeutics
  • David Fajgenbaum, MD, MBA, MSc, Assistant Professor, Perelman School of Medicine at the University of Pennsylvania
  • Carla Rodriguez Watson, PhD, MPH – Director of Research, Reagan-Udall Foundation

Friday Fireside Chat: What Can We Do Together?

The speakers will include:

  • Eric Marsh, MD, PhD – Clinical Director, ODC, University of Pennsylvania
  • Nicole Boice – Founder, Rare-X
  • Sarita Edwards – Founder & CEO, E.WE Foundation

“We know that for many rare disease patients the burden to fund, research and advocate for rare disease drug development often falls to caregivers and families, most of whom have no experience in drug development,” said Craig Martin, CEO of Global Genes. “It’s incumbent on us to work together to share knowledge in order to ensure that viable therapeutic approaches have the best possible chance of reaching patients.”

Following the event, Global Genes and the Orphan Disease Center at the University of Pennsylvania will publish a comprehensive report summarizing key takeaways, proposed solutions, and tactical steps forward.

“As we co-host our 7th RDDS we are emboldened by the leadership within the rare disease community and are grateful for those who are participating in this important event,” said Jim Wilson, MD, PhD, Director, Gene Therapy Program; Rose H. Weiss professor and director, Orphan Disease Center; and professor in the Departments of Medicine and Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. “By combining efforts, we can seek to create therapeutic resolutions that may lead to treatment for rare disease states of all kinds.”

In addition to the in-person RDDS event, Global Genes will host and share a series of webinars, educational videos and resources throughout the remainder of the year.

Sponsors for the 7th Annual RARE Drug Development Symposium include:

Gold sponsor: Horizon Therapeutics

Silver sponsors: Editas Medicine, Janssen, Sanofi, and Travere Therapeutics;

Bronze sponsors: Alexion, Pfizer, Regeneron, and UCB

Partner sponsors: Avidity Biosciences, Beam Therapeutics, BioCryst, Daiichi-Sankyo, and Ovid Therapeutics; and

Industry session sponsor: Sangamo Therapeutics

Registration for the symposium is now open. For more information on the event, view a video invitation from Craig Martin and Jim Wilson.

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 and families globally. In pursuit of our mission, we connect, empower, and inspire 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. We serve the 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 our Resource Hub.


Global Genes:

Laura Vinci

Finn Partners