RHEACELL

A late-stage cell therapy company focused on severe inflammation-driven diseases

RHEACELL is developing cell therapies based on its proprietary ABCB5+ platform to deliver highly differentiated, allogeneic (off-the-shelf) mesenchymal stromal cell therapies to patients with significant inflammation driven diseases.

RHEACELL's lead program, allo-APZ2-OTS, is a first-in-class intravenous ABCB5+ MSC therapy in late-stage clinical development for Epidermolysis bullosa, a group of rare, inherited disorders characterized by mechanical fragility of the skin and mucosal tissues.

RHEACELL has in-house GMP manufacturing and robust CMC capabilities spanning late-stage clinical development and commercial-scale readiness.

Investment Highlights

A focused late-stage investment case

Lead Clinical Program

allo-APZ2-OTS - A systemic approach to Epidermolysis Bullosa (EB)

allo-APZ2-OTS is RHEACELL’s lead clinical development program for Epidermolysis bullosa (EB), a group of rare, inherited disorders characterized by mechanical fragility of the skin and mucosal tissues, resulting in blistering, erosions, and impaired wound healing following minimal trauma. In addition to underlying genetic defects affecting dermal–epidermal adhesion, chronic inflammation plays a central role in EB disease progression and contributes significantly to clinical severity.

allo-APZ2-OTS consists of a homogeneous population of allogeneic ABCB5+ mesenchymal stromal cells and is administered intravenously, designed to address both local and systemic inflammatory processes associated with EB.

Lead indication at a glance

The Disease Opportunity

EB is more than a skin disease

Epidermolysis bullosa is a group of rare, debilitating multisystem disorders characterized by chronic wounds, impaired healing and systemic inflammatory complications.

CHRONIC & RECURRENT WOUNDS Persistent wounds, pain, scarring and impaired healing.

SYSTEMIC DISEASE BURDEN Severe EB can affect multiple organ systems and is associated with substantial morbidity and mortality.

HIGH UNMET NEED Current treatment approaches predominantly address individual wounds, highlighting the need for a systemic therapeutic approach.

CHRONIC & RECURRENT WOUNDS

Persistent wounds, pain, scarring and impaired healing.

SYSTEMIC DISEASE BURDEN

Severe EB can affect multiple organ systems and is associated with substantial morbidity and mortality.

HIGH UNMET NEED

Current treatment approaches predominantly address individual wounds, highlighting the need for a systemic therapeutic approach.

CHRONIC & RECURRENT WOUNDS

Persistent wounds, pain, scarring and impaired healing.

SYSTEMIC DISEASE BURDEN

Severe EB can affect multiple organ systems and is associated with substantial morbidity and mortality.

HIGH UNMET NEED

Current treatment approaches predominantly address individual wounds, highlighting the need for a systemic therapeutic approach.

Our Approach

Targeting inflammation systemically

ABCB5+ MSCs are intended to restore immune homeostasis and support wound healing through secretion of IL-1RA. This differentiated approach has been shown to:

• Reduce chronic inflammation
• Modulate the wound microenvironment
• Support tissue repair and wound closure

PHASE III Clinical Program

A global Phase III program in EB

A global, randomized, double-blind, placebo-controlled study including recessive dystrophic EB and junctional EB in pediatric and adult patients.

• 85 RDEB and 10 JEB patients enrolled across 17 trial sites in 12 countries spanning Europe, North America, Latin America and Asia
• 880 investigational product administrations completed
• More than 1,100 patient visits conducted, including ongoing safety follow-up visits

• Global, multicenter study
• Randomized and placebo-controlled
• Includes patients with RDEB and JEB
• Pediatric and adult patients
• Phase III study completed

• Total of 211 doses
• No treatment-related SAEs
• Includes RDEB and JEB
• Safety data collected beyond the 6-month double-blinded period (ongoing) continues showing a favorable safety profile

From Development to Comercial Readiness

Built beyond clinical development

RHEACELL has built an integrated manufacturing platform designed to support the development and potential commercialization of ABCB5+ cell therapies.

Intellectual Property

A strong intellectual property position

RHEACELL has established a broad intellectual property position around its proprietary ABCB5+ cell platform and therapeutic applications.

Upcoming Milestones

Advancing toward the next stage

Building on the successful competition of our Phase III program, we are advancing allo-APZ2-OTS toward regulatory submission and beyond.

Leadership

Leadership for the next stage of RHEACELL

Dr. Christoph Ganss

Chief Executive Officer

Founder / Vision / Corporate Strategy

Dr. Jasmina Esterlechner

Chief Operating Officer

Operations / GMP Manufacturing / Regulatory & Inspection Readiness

Dr. Cristina Daniele

Director Clinical Development

Late-stage Clinical Development / Phase III Program

Dr. Nabil Uddin

Chief Business Officer

US Strategy / Business & Investor Perspective

Meet the team behind RHEACELL

Our broader team brings together the scientific, clinical, regulatory, manufacturing and business expertise needed to advance our programs through late-stage development and beyond.

Investor Contact / Footer

Investor Relations

For investor and corporate inquiries, please contact:

Kin Ying Chan

Director Business Development

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