Historic achievement: FDA approval of Abeona Therapeutics' ZEVASKYN accelerates momentum and expands treatment options for the EB community.

EB Research Partnership ("EBRP") invested in the work that led to ZEVASKYN at Stanford University under their pioneering Venture Philanthropy Model. This approval marks a historic moment for the EB community as the third FDA approved treatment and the first autologous, cell-based gene therapy for RDEB (Recessive Dystrophic Epidermolysis Bullosa). 

NEW YORK, April 29, 2025 /PRNewswire/ -- In a significant advancement for the Recessive Dystrophic Epidermolysis Bullosa (RDEB) community, the U.S. Food and Drug Administration (FDA) has approved a third treatment for this rare condition. The milestone approval of Abeona Therapeutics' ZEVASKYN not only represents a continued commitment to innovation in RDEB care, but reinforces the growing momentum toward improved quality of life and expanded treatment choices for individuals and families affected by RDEB. ZEVASKYN is the first autologous, cell-based gene therapy for RDEB, and the first RDEB treatment designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 gene.

A BREAKTHROUGH IS NOW A BLUEPRINT

EB Research Partnership (EBRP) is the world's largest nonprofit funding life-changing research to accelerate treatments and find a cure for EB, while pioneering a path for rare disease at large. EB is one of over 10,000 rare diseases, 95% of which have no approved treatments or cures. Through investments in cutting-edge approaches like gene therapies, stem cell therapies, antibody treatments, and more, EBRP is driving scientific breakthroughs that have the potential to impact the lives of 400 million people around the world living with a rare disease.

"For over 20 years, I've watched my son face the relentless challenges of EB with strength no child should ever have to summon," says Faye Dilgen, EBRP Board Member and mother of John Hudson Dilgen, who lives with RDEB. "This new treatment doesn't just offer new relief, hope, and expanded options, it brings us closer to a future where children with EB won't have to endure the same struggles. We are incredibly grateful to the scientists, advocates, and especially to Abeona and EB Research Partnership for their tireless dedication in making this day possible for families like ours and future generations."

The game-changing approval of ZEVASKYN underscores how progress in EB can serve as a model for advancements across the rare disease space. "Abeona's development and advancement of ZEVASKYN delivers a landmark moment for the global EB community, and their leadership in gene therapy holds so much promise to innovate the therapeutic landscape for not only EB, but many other rare diseases and conditions," says Michael Hund, CEO of EBRP.

A MODEL TO SCALE SOLUTIONS

With this FDA approval, EBRP gains continued momentum in its mission to discover treatments and a cure for EB. This milestone reinforces the validity of their scalable, sustainable Venture Philanthropy model, and, most importantly, provides treatment options and ignites hope for families around the world affected by EB.

EBRP invested in the ground-breaking work that led to ZEVASKYN at Stanford University under their Venture Philanthropy Model. The funding followed the highly competitive application and screening process overseen by EBRP's Scientific Advisory Board (SAB), which is composed of leading scientists and physicians. EBRP was able to realize a significant return on its investment and reinvest that capital back into additional promising EB research projects.

EBRP's Venture Philanthropy business model has been highlighted for its leadership by Harvard Business School, Stanford Social Innovation Review, Yale University, the Milken Institute, and MIT.

"EB Research Partnership is proud to be an early investor via our Venture Philanthropy Model in the science leading to ZEVASKYN at Stanford University. The collaboration between EBRP and Stanford exemplifies the mission of EBRP to advance commercially sustainable research aimed at treating and ultimately curing epidermolysis bullosa. We are honored to partner with the entire Abeona team and commend their leadership, determination, and passion to deliver much needed innovative solutions for individuals and their families living with EB. They continue to share our values and commitment to accelerate treatments to the EB community as quickly as possible," says Michael Hund, CEO of EBRP.

A COURAGEOUS COMMUNITY DRIVING CHANGE

EB is 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. With skin this fragile, those with EB face severe pain, open external and internal wounds, and grueling bandaging processes. 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 to find a cure for this community, including:

• Raising over $70M
• Funding 160+ EB projects
• Contributing to a 25x growth in the EB clinical trial landscape
• Directly funding two FDA-approved EB treatments

Today's FDA approval of the first autologous, cell-based gene therapy for RDEB is an exciting proof point of this progress. EBRP will continue to build off this momentum and is dedicated to delivering a cure.

A PERIOD OF UNPRECEDENTED AWARENESS

ZEVASKYN's approval arrives during an unprecedented wave of awareness for EB, fueled by recent high-impact initiatives led by EBRP and their Co-Founders Eddie and Jill Vedder.

Launched in 2025, the Vedder Cup is an annual series between the San Diego Padres and Seattle Mariners that carries a meaningful purpose beyond the game. Through their partnership with EBRP, both teams are actively driving increased awareness and visibility for Epidermolysis Bullosa. This collaboration is not only shining a national spotlight on EB but also advancing efforts to accelerate the development of treatments and a cure.

The upcoming premiere of "Matter of Time", a compelling documentary capturing the electrifying energy of Eddie Vedder's October 2023 solo concerts in Seattle, is bringing new levels of EB visibility on a global scale. Premiering in June 2025 at the Tribeca Festival, "Matter of Time" weaves together powerful music and the moving stories of individuals impacted by EB - including families, researchers, and thought leaders - and highlights how innovation and determination are driving progress toward a cure. One such researcher chronicled throughout the film is Dr. Jean Tang of Stanford University, who was lead principal investigator on the clinical of this third FDA approved treatment. The documentary is a testament to the power of music, community, and unwavering determination to drive change and inspire hope.

Public awareness of EB has never been stronger—and this most recent FDA approval underscores the momentum building around research, advocacy, and support.

About EB Research Partnership

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.

When making a grant to a research project, EBRP utilizes a Venture Philanthropy model. They retain the added upside of generating a recurring revenue stream if the therapy or product is commercially successful, then use the return on investment to fund additional EB research until a cure is found. While EB is a rare disease, there are 10,000 rare diseases that affect 1 in 10 people in the world, 95% of which have no treatments, that can benefit from the research they fund and this innovative model.

To learn more about EBRP, visit www.ebresearch.org
For more information on ZEVASKYN, visit www.zevaskyn.com
For more information on Matter of Time, visit www.ebresearch.org/matter-of-time-film

SOURCE EB Research Partnership