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Accelerating Impact: EB Research Partnership is Empowering Rare Disease Patients to be in The Driver’s Seat

In a world where 95% of rare diseases lack FDA-approved treatments, organizations like EB Research Partnership (EBRP) are making a significant impact. With their groundbreaking work, EBRP not only provides hope to countless individuals and families affected by this devastating disease, but is also paving the way for progress across the wider rare disease health care space. Last year, EB Research won the 2022 Horizon Prize powered by MIT Solve and Horizon Therapeutics, which has been instrumental in propelling their mission to transform the lives of those living with Epidermolysis Bullosa (EB) and inspiring others to join the cause.

Michael Hund (center), CEO of EBRP, accepting the 2022 Horizon Prize on stage at the annual Concordia Summit in New York City. Pictured with Holly Copeland (right) from Horizon Therapeutics, and Rachael Drew (left) from MIT Solve.

With over 400 million people worldwide living with one of the 7,000 known rare diseases, the lack of FDA-approved treatments is a significant hurdle. EB, a life-threatening, painful genetic skin disorder affecting children from birth, is no exception. The scarcity of centralized EB datasets and reliable resources blocks progress toward effective therapies and improved patient experiences. 

That’s where EBRP comes in. Their platform aims to bridge these gaps by collecting comprehensive patient data and making it accessible to all stakeholders. By allowing patients to contribute their health information directly, the platform accelerates therapy development, creating a roadmap toward a personalized patient journey and treatments. The vision is to create a world where rare disease data is securely shared among researchers, doctors collaborate seamlessly, and patients are empowered to navigate their journey towards a cure.

Impactful projects and industry advancements for rare disease 

EBRP has accomplished significant projects to-date, leading to transformative advancements in the rare disease health care fields. Since its establishment in 2010, EBRP has funded over 120 EB research projects, generating promising outcomes. Notably, their support for Krystal Biotech enabled the development of VYJUVEK™, the first-ever FDA-approved topical gene therapy for EB. Beyond the boundaries of EB, their efforts have far-reaching implications. With more than 40 EB clinical trials underway, including three Phase III trials, the potential to treat or cure EB is within reach—which will also pave the way for thousands of other rare diseases. 

“The funding we received from the Horizon Prize in 2022 provided us with a great foundation to further advance a centralized platform for patients with EB and their caregivers,” said Michael Hund, Chief Executive Officer, EBRP. “Creating a comprehensive digital platform for patients living with EB has been an important step to improving quality of life and enabling more real-time collaboration between patients, families, and the health care providers who can help manage their treatment.”

Cure Curator is a first-of-its-kind data platform uniting EB patients and investigators to accelerate the path to treatments and cures, aspiring to expand to all rare diseases. After their Horizon Prize win, EBRP was able to rapidly expedite Curator’s development and are currently preparing to launch an MVP to a diverse patient advisory panel. They have built an interface with via their API to ingest, analyze, and organize EB clinical trials in real time, allowing patients and caregivers to search clinical trials in a user friendly way based on location, academic medical center, disease subtype, age, and trial status. 

6-year-old Elodie suffers from the life-threatening disease EB. Like all “Butterfly Children,” Elodie’s skin is extremely fragile—as thin as the wings of a butterfly—leading to severe pain and wounds.

Positioning for the future 

Looking ahead, EBRP has ambitious long-term goals. Having already analyzed and shared data on over 500 patients, they are on the cusp of launching a beta testing group where patients and caregivers can directly provide and access health care data, including at-home genetic sequencing kits, giving them agency over their care and participation in finding cures for their disease. The aim is not only to accelerate a cure for EB but also to scale the platform to encompass the 7,000 rare diseases affecting millions of people globally.

As a company advancing research and development in the rare disease space, EBRP hopes to inspire other organizations to follow in their footsteps. Their work and the positive impact of winning the 2022 Horizon Prize serve as a testament to the potential for collaboration, innovation, and patient-driven initiatives. By sharing their experiences, knowledge, and best practices, EBRP seeks to encourage a collective effort to tackle the challenges faced by rare disease communities worldwide.

EB Research Partnership's journey is a remarkable example of how dedication, innovation, and the support of initiatives like the Horizon Prize can drive transformative change in the rare disease landscape. By harnessing the power of data, collaboration, and patient empowerment, EBRP is paving the way for improved treatments, enhanced care, and ultimately, a brighter future for individuals and families impacted by rare diseases like Epidermolysis Bullosa.

If you are interested in applying to the 2023 Horizon Prize, learn about this year’s challenge, focused on decreasing the environmental impacts of the rare disease health care space. $150,000 will be awarded to the prize winner, who will be announced live on stage at the Concordia Annual Summit this September. The deadline for all applications is June 23, 2023. 

Cover photo: Salim, pictured above with his mom, lives with EB. They battle this rare genetic skin disorder every day and have hope that one day, thanks to passionate researchers and advocates, EB will be history.

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