RHEACELL and AOP Health: Strategic Partnership to Deliver Breakthrough Therapies for the “Butterfly Children’s Disease” and Chronic Venous Wounds

RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. This partnership marks a crucial step toward making breakthrough therapies available to patients with high unmet medical needs – particularly those affected by Epidermolysis bullosa (EB or “butterfly children’s disease”) and chronic venous wounds. Both conditions currently have limited treatment options, severely impacting the lives of patients and their families.Two novel therapies from RHEACELL’s pipeline are currently in Phase 3 clinical development. Combining RHEACELL’s cutting-edge R&D capabilities with AOP Health’s proven expertise in bringing rare disease therapies to patients in Europe, this partnership underscores both companies’ strong commitment to addressing high unmet medical needs.

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With 30 years of experience and a growing presence in more than 50 countries, AOP Health complements this ambition by providing the expertise needed to make RHEACELL’s cell therapies available not only in Europe, but also in the Middle East, North Africa, Turkey, and Israel.

Promising cell therapy

Cell-based regenerative therapies are gaining increasing importance, particularly for diseases with currently limited therapeutic options. For over 20 years, RHEACELL has focused on the research and development of so-called stromal cells - stabilizing cells obtained from donor skin. RHEACELL's cell therapy is only the second stromal cell product to receive national marketing authorization from the Paul Ehrlich Institute in Germany for the treatment of therapy-resistant, chronic venous wounds ("leg ulcer") on the basis of positive phase 2 clinical trial data.^{1; 2}

An innovative mechanism of action

RHEACELL’s cell therapy is based on a specific type of cells, so-called ABCB5+ mesenchymal stromal cells, with anti-inflammatory and immunomodulatory properties. These cells represent a promising new therapeutic approach for EB and chronic venous wounds. In patients with EB, this therapy is administered systemically and can thus stimulate both internal and external wound healing by restoring the normal physiological function of affected tissues – an innovative mechanism of action. This can lead to a reduction in the number of existing wounds and help prevent the formation of new ones.³

Building on positive Phase 2 results and positive feedback from regulatory authorities, two Phase 3 clinical trials are currently underway, and a first EMA submission is already expected in 2026.

Background:

Voices on the Partnership

"This partnership marks a significant milestone for RHEACELL and the further development of our stem cell medication programs. Collaborating with an established partner like AOP Health to commercialize our stromal cell medications will allow us to bring our innovative therapies to the European patients faster and more efficiently."
Dr. med. Christoph Ganss, CEO RHEACELL

"Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovation. This partnership with stem cell pioneer RHEACELL enables us to jointly bring new treatment options to patients living with chronic venous wounds or Epidermolysis bullosa, who currently have limited or no treatment options. Our teams are fully committed to making these groundbreaking treatments available to all patients affected."
Dr. Martin Steinhart, CEO AOP Health

"In treating Epidermolysis bullosa, we have had few therapeutic options to date. This lack of treatment options adds an additional burden on those affected. From a medical perspective, the concept of regenerative, anti-inflammatory cell therapy is a promising approach. That’s why our study center is participating in the next milestone—Phase 3 trials."
Univ.-Prof. Dr. Johann Bauer, Head of EB Research at the EB-Haus Austria, the world’s first specialized clinic for ‘butterfly children’.

"RHEACELL's approach of administering stromal cells systemically to stimulate internal and external wound healing distinguishes it from other approaches that focus primarily on local applications on the skin. We are thrilled to follow the ongoing clinical development."
Dr. Rainer Riedl, founder and chairman of DEBRA Austria

About Epidermolysis bullosa (EB)

EB is a congenital and currently incurable condition that can affect not only the skin but also internal organs. With approximately 500,000 people affected worldwide⁴, EB is a rare disease that severely impacts patients’ quality of life. In this genetic disorder, the skin is as fragile as a butterfly’s wing. Even minor mechanical stress or friction can cause blistering and painful chronic wounds.

The patient organization DEBRA describes EB as “the worst disease you’ve never heard of.” DEBRA was founded in 1995 as a patient organization by patients, families, and physicians with the goal of facilitating exchange and providing support to people living with EB. One of DEBRA Austria’s key initiatives is the EB-Haus Austria, located at the Salzburg University Hospital campus. It serves as a center of excellence for Epidermolysis bullosa and is also the world’s first specialized clinic for “butterfly children.”

About Chronic Venous Wounds (leg ulcer)

Venous ulcers are chronic wounds that result from prolonged venous insufficiency, primarily affecting older adults. These ulcers develop due to poor blood flow in the veins, leading to tissue breakdown, especially in the lower extremities. They are the most common type of leg ulcers, accounting for 60–80% of cases.

About AOP Health

AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions. (aop-health.com)

About RHEACELL

With more than 20 years of experience, we are a leading, integrated biopharmaceutical stem cell company based in Heidelberg, Germany, currently conducting two approval trials (EU, US). We focus on innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases who experience high levels of distress and for whom no adequate treatments currently exist. Our goal is to offer these patients a new and innovative treatment option. Our ABCB5+ mesenchymal stromal cells, as a purified active ingredient, can improve the lives of these patients—for example, in cases of Epidermolysis bullosa — and have the potential to transform treatment paradigms for these diseases. Our targeted approach to fighting inflammation through our proprietary stem cell therapy enables the restoration of normal physiological function in affected individuals.

¹https://www.rheacell.com/pdf/Gebrauchs-und-Fachinformation_AMESANAR.pdf
² https://www.pei.de/SiteGlobals/Forms/Suche/Arzneimittelsuche_Formular.html
³Kiritsi et al. Clinical trial of ABCB5-positive mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight 2021;6:e151922
⁴Rashidghamat, E. et al (2017). Persistent and rare diseases research. 2017; 6(1):6-20. DOI: 10.5582/irdr.2017.01005

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