Healing begins with research: Promising development program on stem cells in rare diseases

Main image: The stem cells are isolated and multiplied using a complex, patented process in a GMP (Good Manufacturing Practice) certified clean room, which ensures that ABCB+ MSCs are produced under the strictest quality and hygiene conditions. This process delivers a high purity product consisting of more than 90% ABCB5+ cells.

RHEACELL has been researching ABCB5-positive stem cell therapies for patients with severe immune and inflammation-related diseases for which there are currently no adequate treatment options. Positive feedback from the regulatory authorities on the study program now gives reason to hope that those affected will be able to benefit from this new approach to regenerative medicine in the near future.

RHEACELL, recognised as a leading integrative biopharmaceutical stem cell company, has been at the cutting edge of stem cell research for more than two decades. The German company, founded in 2003 by Dr. Christoph Ganss, has focused on the development of ABCB5-positive mesenchymal stem cells (MSCs), a specific subset that have shown remarkable anti-inflammatory and regenerative properties.

With an extensive stem cell pipeline for use in rare, severe immune and inflammatory diseases the biotech company conducts numerous national and international multicentre clinical trials together with world-leading experts, some of them already as Phase 3 clinical trials. In close coordination with the respective regulatory authorities and under the strictest scientific biopharmaceutical and regulatory frameworks, their innovative stem cell therapies could soon be a new treatment option for diseases that have long been considered untreatable. Currently, only 5% of approximately 6,000 to 10,000 known rare diseases are treatable, and many companies are shying away from the financial outlaw in view of low patient numbers.

“I have always been interested in the connection between research, medicine and business. I worked as a doctor in a clinic for many years. Then the opportunity arose to start my own business.,” says Dr. Ganss. "Our ABCB5+ MSCs as a pure active ingredient can significantly improve the lives of many patients and have the potential to represent a real turning point for the regenerative medicine.” 

One research focus is on the rare disease Epidermolysis bullosa (EB), a devastating genetic skin disorder that affects approximately 500,000 people worldwide. Known as "butterfly disease" due to the fragility of the skin, EB causes painful blisters and wounds that severely impact the quality of life. In its most severe forms, the disease can lead to scarring of internal organs, malnutrition, and even early death.

Despite the severity of EB, there is currently no cure for the disease, and treatment options are limited to managing symptoms. The RHEACELL stem cells have demonstrated significant potential in promoting wound healing and reducing inflammation, offering a much-needed therapeutic option for EB patients.

In December 2023, the U.S. Food and Drug Administration (FDA) awarded RHEACELL a Rare Pediatric Disease Priority Review Voucher (RPDD) for their work on EB. This was followed by a Regenerative Medicine Advanced Therapy (RMAT) fast-track designation in February 2024. These designations are designed to accelerate the development and review of promising treatments for serious and life-threatening conditions.

"The FDA's positive assessment for accelerating our clinical development programme for butterfly disease is of great importance to those affected in view of their enormous suffering,” says Dr. Ganss, “and we will now continue to develop our stem cell candidate at full speed after a successful phase 2a study."

While the focus on EB is a critical part of RHEACELL's mission, the potential applications of ABCB5-positive MSCs extend beyond this rare disease. The stem cells have shown promise in treating a range of inflammatory and degenerative conditions, particularly those with high unmet medical needs. 

One of the company's significant achievements was the national marketing authorisation granted by the Paul-Ehrlich-Institute in Germany for the use of ABCB5-positive MSCs in treating chronic venous ulcers (CVU). This marked only the second somatic cell product to receive such approval in Germany, underscoring the groundbreaking nature of RHEACELL's work. The company’s pipeline is robust, with several ongoing clinical trials.  
 
“We will develop other drugs that we have in the pipeline clinically and bring them into approval,” Dr. Ganss explains. “Since we will have to massively increase our production capacities in the near future in order to be able to meet the emerging global demand for our cells, we will expand and also look for partners for global commercial marketing. The positive feedback from the regulatory authorities on our clinical development program is a great incentive for us. It is also an important confirmation of the therapeutic concept of regenerative, anti-inflammatory ABCB5+ MSCs as a new promising therapeutic option for various severe immune and inflammation-driven diseases. And we will now work flat out on clinical development and market access," says Dr. Ganss.