Press release RHEACELL
RHEACELL and AOP Health: Strategic Alliance for Groundbreaking Therapies for Epidermolysis Bullosa and Chronic Venous Ulcers
Partnership aims to provide patients with high unmet medical needs access to innovative stromal cell therapies
Heidelberg, Vienna [27 May 2025] – German biotech company RHEACELL, specialized in the development of stem cell therapies, and AOP Health, a European pioneer in integrated therapeutic approaches for rare diseases, are joining forces. Their shared goal: to make two promising therapies based on ABCB5-positive mesenchymal stromal cells accessible to patients — AMESANAR® and EBESANAR®. Both therapies are classified as Advanced Therapy Medicinal Products (ATMPs), are currently in Phase 3 clinical development, and target epidermolysis bullosa (a rare and currently barely treatable disease) and chronic venous ulcers (CVUs). This alliance combines RHEACELL’s research excellence with AOP Health’s proven experience in launching therapies for rare diseases in Europe, underscoring both companies’ commitment to closing critical gaps in medical care.
With 30 years of experience and a growing presence in over 50 countries, AOP Health brings the know-how needed to make RHEACELL’s innovative stem cell therapies available not only in Europe but also in the Middle East, North Africa, Turkey, and Israel.
Promising Stromal Cell Therapy
Cell-based regenerative approaches are gaining increasing relevance, especially for conditions with limited treatment options. These therapies belong to the class of Advanced Therapy Medicinal Products (ATMPs) and offer new avenues for care. Although stromal cells have been studied worldwide for years, very few products have reached clinical application. For over two decades, RHEACELL has been researching innovative stem cell therapies for severe immune- and inflammation-related diseases.
EBESANAR® is being developed for the ultra-rare disease epidermolysis bullosa (EB), also known as “butterfly children disease.” Affecting approximately 500,000 people globally, EB significantly impairs patients' quality of life. Even minimal mechanical stress or friction can cause blistering and painful chronic wounds on skin and mucosa.
AMESANAR®, indicated for therapy-resistant chronic venous ulcers (CVUs), is only the second stromal cell product to receive national market authorization in Germany from the Paul-Ehrlich-Institut based on positive Phase 2 clinical data. It is already available in Germany for treating CVUs.
An Innovative Mechanism of Action
AMESANAR® and EBESANAR® are novel first-in-class ATMPs that support immune modulation and wound healing. These somatic cell therapies are based on ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs), which are allogeneic and derived from human skin tissue. ABCB5+ MSCs have unique immune-modulating properties. Notably, they can release interleukin-1 receptor antagonist (IL-1RA), a molecule that binds to the IL-1 receptor and inhibits the release of IL-1β. This mechanism helps reduce excessive immune responses and shifts macrophages from a pro-inflammatory M1 phenotype to a wound-healing M2 phenotype — thus supporting the body’s natural healing processes.
Depending on the condition, ABCB5+ MSCs can either be applied topically to external wounds (as in CVUs) or administered intravenously for systemic impact (as in multi-systemic EB). In both cases, clinical data suggest a reduction in existing wounds and potential to prevent new lesions.
Info box:
Voices on the Collaboration
“So far, we have had very few options for treating epidermolysis bullosa, which intensifies patient suffering. From a medical standpoint, the regenerative, anti-inflammatory stromal cell therapy concept is a highly promising approach. That’s why our study center is participating in the upcoming Phase 3 trial.”
— Univ. Prof. Dr. Johann Bauer, Head of EB Research at EB-Haus Austria, the world’s first specialized clinic for “butterfly children”
“RHEACELL’s approach of administering stromal cells systemically to promote both internal and external wound healing sets them apart from other therapies that mainly focus on local skin applications. We are watching the continued clinical development with great interest.”
— Dr. Rainer Riedl, Founder and Chairman of DEBRA Austria
“This partnership marks a major milestone for our company and the advancement of our stem cell programs. Collaborating with an established partner like AOP Health enables us to bring our innovative stromal cell medicines to patients across Europe more quickly and efficiently.”
— Dr. Christoph Ganss, CEO of RHEACELL
“Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovations. Partnering with stem cell pioneer RHEACELL allows us to jointly offer new treatment options for people with epidermolysis bullosa or chronic venous ulcers — conditions for which there have been few or no treatments to date. Our teams are fully committed to making these breakthrough therapies accessible to those in need.”
— Dr. Martin Steinhart, CEO of AOP Health
“Together, we can set new standards in the treatment of rare diseases and improve patient care. Additionally, we’re excited that this collaboration strengthens Europe’s standing as a life sciences hub.”
— Dr. Michael Lange, Area Director DACH Region at AOP Health
About DEBRA
Founded in 1995 as a self-help group by individuals with EB, their families, and physicians, DEBRA Austria organizes experience sharing and support for those affected. One of DEBRA’s initiatives is the EB-Haus Austria, located at the Salzburg University Hospital campus. It serves as both a center of excellence for epidermolysis bullosa and a specialized clinic for “butterfly children.”
PRESS CONTACTS
RHEACELL
Daniela Weis
Im Neuenheimer Feld 517
69120 Heidelberg
T +49 6221 71833-0
E media@rheacell.com
AOP HEALTH
Mag. Nina Roth, MAS
Director of Corporate Communication
T +43 676 3131509
E nina.roth@aop-health.com
About RHEACELL
With over 20 years of experience, RHEACELL is a leading integrated biopharmaceutical stem cell company based in Heidelberg, Germany. We are currently conducting two approval studies (EU, US) and focus on innovative stem cell therapies for patients with severe immune and inflammatory diseases for which there are currently no adequate treatments. Our ABCB5+ mesenchymal stromal cells, as pure active substances, have the potential to significantly improve patients' lives, such as in epidermolysis bullosa, and represent a turning point in treatment concepts for these conditions. Through targeted anti-inflammatory effects, our proprietary stem cell therapy supports the restoration of normal physiological function.
About AOP Health
AOP Health is a global group headquartered in Austria, where AOP Orphan Pharmaceuticals GmbH is based. Since 1996, AOP Health has been dedicated to developing innovative solutions for patients with unmet medical needs, particularly in rare diseases and critical care. The group is internationally recognized as a pioneer in integrated therapy solutions, operating through subsidiaries, local offices, and a robust network of global partners. With its motto “Needs. Science. Trust.”, AOP Health emphasizes its commitment to research, development, and strong relationships with physicians and patient advocacy groups to meet stakeholders’ needs in every aspect of its work. (aop-health.com)
References:
[1] Rashidghamat, E. et al (2017). Persistent and rare diseases research. 2017; 6(1):6-20. DOI: 10.5582/irdr.2017.01005
[2] https://www.rheacell.com/pdf/Gebrauchs-und-Fachinformation_AMESANAR.pdf
[3] https://www.pei.de/SiteGlobals/Forms/Suche/Arzneimittelsuche_Formular.html
[4] Kiritsi et al. Clinical trial of ABCB5-positive mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight 2021;6:e151922
