Founded in 2010 by a dedicated group of parents along with Jill and Eddie Vedder of Pearl Jam, EB Research Partnership (EBRP) is the largest global nonprofit dedicated to funding research to accelerate treatments and find a cure for Epidermolysis Bullosa (EB), a group of devastating and life-threatening skin disorders that affect children from birth. Children with EB are often called "Butterfly Children" because their skin is as fragile as the wings of a butterfly. For the 500,000 people around the world living with EB, everyday activities like eating, sleeping, walking and playing can become monumental tasks that often require modification.
Over the last decade, EBRP has made undeniable progress in its mission, including: raising over $80M to fund 180+ EB projects, contributing to a 25x growth in the EB clinical trial landscape, and directly funding two FDA-approved EB treatments while helping to accelerate a third. EBRP will continue to build off this momentum and is dedicated to delivering more treatments and ultimately cures for the EB community while paving a path for rare disease at large.
To achieve this bold mission, we're accelerating innovative research right in your backyard!
Over the last decade, EBRP has made undeniable progress in its mission, including: raising over $80M to fund 180+ EB projects, contributing to a 25x growth in the EB clinical trial landscape, and directly funding two FDA-approved EB treatments while helping to accelerate a third. EBRP will continue to build off this momentum and is dedicated to delivering more treatments and ultimately cures for the EB community while paving a path for rare disease at large.
To achieve this bold mission, we're accelerating innovative research right in your backyard!
|
Squamous cell carcinoma (SCC) metastasizes quickly and is the most common cause of death in young adults with RDEB, yet current treatment options are non-existent. The South Lab has identified a lead compound, rigosertib, that exhibited significant specificity for RDEB cancer. Two "first in EB" investigator led clinical trials have been initiated in the USA and Austria to assess tolerability and tumor targeting of rigosertib in patients with late stage, metastatic and/ or unresectable SCC. A recent publication detailing results from this trial shows that rigosertib has great potential for treating RDEB SCC, since four of the five patients treated showed a response, 50% of which showed a complete and durable response over the 12 months of treatment. Now, the team is readying rigosertib for clinical development and approaching clinical trials with the overall goal of developing this treatment for the high unmet need in the RDEB community.
|
Dr. Andrew South is the Oros Family Professor in Rare Skin Diseases in the Department of Dermatology at the University of Wisconsin-Madison. His lab has studied epidermolysis bullosa since 1999 when he began his post-doctoral studies with Professor John McGrath in London. Dr. South’s laboratory focuses on mechanisms of squamous cell carcinoma (SCC) initiation and progression in EB patients with a view to developing preventative or curative therapies. He studies EB disease mechanisms that lead to the development of cancer and has worked with a number of companies developing therapies for patients with EB such as Krystal Biotech’s Vyjuvek.
|
|
While supporting local pioneers like Dr. South and his critical work to treat RDEB SCC, EBRP continues to fund additional work in the SCC space to help improve diagnosis rates and quality of life. For example, EBRP has funded an ongoing project at Northwestern to develop an AI, deep learning model that can distinguish SCC from non-SCC lesions on photographs of RDEB skin. The model will be integrated into an app with the long-term goal of a tool for patients and physicians for interpretation of RDEB skin photography to assist in finding SCCs as early as possible, even from the comfort of their homes!
|
|
|
Microphysiological Systems (MPS) are advanced in vitro platforms that allow researchers to mimic complex biological structures such as the architecture of human skin. The team at UW is using this MPS technology to generate a human-derived skin construct, or skin organoid, to evaluate new genome editing therapies for EB Simplex. Excitingly, their system supports co culture of keratinocytes, dermal fibroblasts, and microvascular endothelial cells in a 3D architecture, enabling real time, non destructive assessment of keratinocyte stratification, barrier integrity, and mechanical resilience. This provides a quantitative, high throughput system that can evaluate genome editing efficacy, safety, and functional rescue. The resulting data from this project will inform genome editor selection, dosing strategies, and translational readiness for future preclinical and IND enabling studies to bring treatments closer to those living with EB Simplex.
While funding Dr. Ayuso's ground-breaking work, EBRP continues to support additional organoid projects to expand the access of these revolutionary platforms to additional subtypes. The team at Harvard Medical School have built and validated their own human skin organoid that accurately reproduces the key features of RDEB at a structural and molecular level, and plan to expand to JEB as well. Because these platforms can be used to test a wide range of emerging therapies across multiple EB subtypes, they essentially create a shared preclinical testing pipeline that other researchers developing gene therapies for EB can plug directly into, accelerating the path from laboratory discovery to human trials.
|
Dr. Jose Ayuso is an Assistant Professor of Dermatology Research Laboratories at the University of Wisconsin, as well as an affiliated member of the department of biomedical engineering. His lab's research focuses on studying human disease using advanced in vitro platforms that mimic the in vivo architecture and tissue microphysiology. Dr. Ayuso and his team use these novel technologies to study multiple diseases, including cancer, genetic diseases, and human-pathogen interactions.
|
At EBRP, we know that in order to do things that haven’t been done you need to be willing to break the mold.
With this innovative business model we’re pioneering a new path forward that will not only revolutionize the way nonprofits operate, but also show the world how cures are found. We don’t just write checks and hope for the best. Instead, we operate like a venture capitalist with a focus on a different kind of ROI, return on impact. We create venture agreements with each project we fund. When those projects succeed, the returns are reinvested back into other promising research and development projects.
Did we mention the science we fund is also scalable across thousands of other rare diseases? That's right. The impact of every dollar invested at EBRP is multiplied many times over.
With this innovative business model we’re pioneering a new path forward that will not only revolutionize the way nonprofits operate, but also show the world how cures are found. We don’t just write checks and hope for the best. Instead, we operate like a venture capitalist with a focus on a different kind of ROI, return on impact. We create venture agreements with each project we fund. When those projects succeed, the returns are reinvested back into other promising research and development projects.
Did we mention the science we fund is also scalable across thousands of other rare diseases? That's right. The impact of every dollar invested at EBRP is multiplied many times over.
|
Our Venture Philanthropy Model has been highlighted for its leadership by Harvard, Yale, MIT, the Milken Institute, Stanford Social Innovation Review, and more.
|
With this business model we are actively transforming the EB landscape while creating scalable impact. These projects, this work, and the way we operate will create a butterfly effect for the 400M+ globally impacted by a rare disease.
Download our Impact Booklet to explore the Harvard Business School and Yale School of Management case studies on EBRP's innovative model and transformative impact.
Download our Impact Booklet to explore the Harvard Business School and Yale School of Management case studies on EBRP's innovative model and transformative impact.
|
|
Children with EB are often called “Butterfly Children” because their skin is as fragile as the wings of a butterfly. With skin this fragile, everyday activities that many take for granted like eating, sleeping, walking and playing can become monumental tasks.
The harsh reality is that rare diseases affect more people than HIV and Cancer combined. EB is one of over 10,000 rare diseases, 95% of which have no approved treatments or cures. We are focused on finding a cure for EB, but for us that’s just the beginning. |
|
|
Founded in 2010 by a group of dedicated parents, Jill Vedder, and Eddie Vedder of Pearl Jam, EB Research Partnership (EBRP) is the largest nonprofit funding research aimed at finding a cure for Epidermolysis Bullosa (EB).
Over the last decade, we have made remarkable strides, including raising +$70M for life-saving research, contributing to a 25x growth in EB clinical trials, and directly funding 2 FDA approved treatments and helping to accelerate the third. The progress we’ve made in EB is undeniable, however, we are only scratching the surface. Our Venture Philanthropy Model is actively transforming the EB landscape while creating scalable impact. These projects, this work, and the way we operate will create a butterfly effect for the 400M+ globally impacted by a rare disease. |
