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Global Genes’ RARE-X issues open call to researchers to participate in the Inaugural Xcelerate RARE Challenge 

Aliso Viejo, Calif.—May 31, 2023—Global Genes today announced an open call to researchers interested in participating in its inaugural Xcelerate RARE Challenge, a rare disease open science challenge focused on rare pediatric neurodevelopmental diseases. 

The Xcelerate RARE Challenge brings together academic and biopharmaceutical industry researchers and data scientists to use patient reported data to address unanswered research questions about rare diseases. It is designed to generate new insights into these conditions and allow participants to test hypotheses to fuel therapeutic development by leveraging the growing amount of patient data on Global Genes’ RARE-X platform and federated data sets. 

For academic researchers, the event is an opportunity to work on important problems facing rare disease families and compete for the grant prizes. For industry scientists, it’s an opportunity to get access to rare disease patient data in a pre-competitive, open environment. This data can help their team better understand the underlying biological mechanisms of neurodevelopmental diseases to develop insights into potential therapeutic approaches and advance their research pipeline. 

“With the Xcelerate RARE Challenge, we’re bringing together researchers and data scientists, many of whom don’t normally work on these rare diseases,” said Karmen Trzupek, Senior Director of Scientific Programs for Global Genes. “By creating specific challenge questions, teams will have the opportunity to collaborate and vet analytical approaches and therapeutic strategies.” 

Researchers will have the opportunity to address three challenge questions. The questions include one aimed at expanding known phenotypes with previously unrecognized symptoms, another focused on creating machine learning algorithms to predict disease diagnoses based on the diagnostic journey that is documented by these families providing data, and a third one aimed at using data to validate a potential therapeutic approach. The RTW Charitable Foundation, one of the Xcelerate RARE Challenge sponsors, has set aside grant funding for up to three submissions that provide a compelling therapeutic hypothesis to test in a lab. Global Genes worked with Dream Challenge through the nonprofit Sage Bionetworks to craft the challenge questions and create a dedicated workspace. 

More than 26 rare disease communities represented by more than 35 patient advocacy groups are participating in the challenge and patients and their families affected by these conditions, who entered their health data to the RARE-X data platform to enable researchers to make discoveries. 

“We know that including our information into Global Genes’ RARE-X data platform will help researchers learn more about CACNA1A related disorders,” said Pangkong Fox, Science Engagement Director for the CACNA1A Foundation. “We’re hoping that this initiative brings in many different scientists to take on

these rare diseases and help find treatments to improve the quality of life for our kids and our loved ones.” 

The Challenge begins May 31 and will run through July 31, 2023. Challenge winners will be named September 19 at the Global Genes Week in RARE and 2023 RARE Patient Advocacy Summit. The Xcelerate RARE Challenge will also be the subject of a panel discussion. 

“We believe that patient-reported data is powerful and can be used to fuel and test important hypotheses,” said Charlene Son Rigby, CEO of Global Genes. “With this Xcelerate RARE Challenge, our goal is to advance rare disease understanding and meaningful therapeutic research for rare diseases through open sharing of robust data, and collaboration.” 

Global Genes and the Challenge founding partner and co-organizer, Roche, would like to extend gratitude to the Challenge’s sponsors – Datavant, Horizon, and RTW Charitable Foundation. Data partners, including Ciitizen, Sanford Health, and NetraMark, have also been instrumental in making the Challenge an impactful experience for all involved. Without the generosity and unwavering support of the sponsors and partners, the Xcelerate RARE Challenge would not have been possible.

Researchers can register to participate in the Xcelerate RARE Challenge on the Dream Challenges website

About Global Genes 

Global Genes is a 501(c)(3) nonprofit rare disease patient advocacy organization dedicated to providing patients and advocates with a continuum of services to accelerate their path from early support and awareness through research readiness. Using a collaborative approach that involves biopharma, researchers and advocates with data as a central core, Global Genes also enables research and data collection through the RARE-X research program. Through this effort, Global Genes is building the largest collaborative patient-driven, open-data access initiative for rare diseases globally. 

Media Contact: 

Lauren Alford 

Senior Director, Marketing and Communications 

Global Genes

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