our progress
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building momentum
Since our founding, we have funded over 180 research projects laser-focused on bringing life-saving therapies and cures to the EB community. Our work has transformed the clinical trial landscape from just 2 clinical trials in EB to over 50 today, an over 25x increase.
Charting the Course to Cures
Over the last decade, EBRP has made remarkable strides, including helping to accelerate the first three FDA-approved EB treatments in the past two years alone. Two of these treatments were directly funded by EBRP under our Venture Philanthropy model.
PHASE 2
Proof of Concept
PHASE 3
Pivotal Studies
FDA REVIEW
APPROVAL
For Patients
WOUND
HEALING
TOPICALS
HEALING
TOPICALS
PHASE 2 — Proof of Concept
Stanford University:
A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus for EB
Stanford University:
Using Topical Sirolimus 2% for Patients With EBS
Biomendics:
Topical Tolasure Targeting Aggregated Mutant Keratin in EBS (TAMES)
PHASE 3 — Pivotal Studies
TWi Biotechnology:
Diacerein 1% Ointment for Generalized EB Simplex (EB Shield Study)
Paradigm Therapeutics:
Confirmatory Study (SD-007) of the Efficacy and Safety of SD-101 Cream
APPROVED
Chiesi's
FILSUVEZ
PROTEIN
THERAPY
THERAPY
PHASE 2 — Proof of Concept
Phoenix Tissue Repair:
A Study of PTR-01 in REB
Phoenicis Therapeutics:
Study of PTW-002 in Patients With DEB or RDEB
GENE &
STEM CELL
THERAPY
STEM CELL
THERAPY
University of Southern California:
Gentamicin for JEB
Stanford University:
Gene Transfer for RDEB using Engineered Autologous Epidermal Sheets
University of Minnesota:
Stem Cell Transplant for Severe EB
Wings Therapeutics:
QR-313 for Treatment of DEB Due to Mutation(s) in Exon 73
Anterogen:
ALLO-ASC-SHEET with mesenchymal stem cells (ASCs)
Castle Creek Biosciences:
A Study of FCX-007 for RDEB (D-Fi)
Rheacell:
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells
Krystal Biotech:
KB803 for the Treatment and Prevention of Corneal Abrasions in DEB (IOLITE)
Krystal Biotech's
VYJUVEK
Abeona Therapeutics'
ZEVASKYN
SCC
TREATMENTS
TREATMENTS
Thomas Jefferson University:
Rigosertib for RDEB-SCC
Active studies showing promise and progress.
Pivotal studies to confirm safety and effectiveness.
Under FDA review for evaluation.
Approved therapies available for patients.
RESEARCH SPOTLIGHTS
Stanford University
Peter Marinkovich, MD
A Gene Therapy for One of EB’s Most Severe Forms
Stanford University
Anthony Oro, MD, PhD
Spray-On Skin: A CRISPR Gene Therapy for RDEB
Northwestern University
Amy Paller, MD
Relieving Chronic Pain and Itch in Dystrophic EB
Eliksa Therapeutics
Armen Karamanian, MD, PhD
Eye Drops to Treat EB’s Most Painful Eye Complications
Northwestern University
Amy Paller, MD
Testing an Existing Drug to Treat EB Simplex
Children’s Hospital Corporation
Karl Koehler, PhD
Building a Faster Lab Model for Testing EB Therapies
Instituto Nacional de Pediatría
Maria Teresa Garcia Romero, MD
How EB Affects the Immune System in Children
Explore all funded projects by year
Search the full EBRP Research Database to explore every project EBRP supporters have funded. Find the science that matters most to you.
EBRP Research Database ›
MAKE A DONATION
Thanks to EBRP's pioneering Venture Philanthropy model, every donation has the potential to grow to multiples of its original value.
Donate today to fund the science that will find the cure.
Donate today to fund the science that will find the cure.
