By Raffaele Pereno
STOCKHOLM, Sweden – Aegle Therapeutics, a clinical-stage biopharmaceutical company, is pioneering the use of extracellular vesicle (EV) therapies derived from mesenchymal stem cells (MSCs) to treat rare and severe immuno-dermatological diseases. During Biotech Showcase 2025, Aegle presented its lead product, AGLE-102™, as a first-in-class therapy for Dystrophic Epidermolysis Bullosa (DEB), a devastating genetic disorder characterized by fragile skin prone to blistering.
Shelley Hartman, CEO of Aegle Therapeutics, told CheckOrphan, “We are thrilled to announce the launch of our Phase 1/2a clinical trial evaluating AGLE-102 in recessive dystrophic epidermolysis bullosa (RDEB), the most severe form of the disease. This represents a critical step in our commitment to developing transformative therapies for rare diseases with high unmet needs”.
What Makes AGLE-102™ Unique?
Unlike traditional stem cell therapies, AGLE-102™ harnesses extracellular vesicles (EVs)—natural nanoparticles secreted by MSCs that carry active biomolecules such as proteins and nucleic acids. These EVs function as the mechanism of action behind MSC therapies but offer distinct advantages:
- Non-Cellular Approach – EVs do not proliferate or migrate, eliminating safety concerns associated with live cell therapies.
- Targeted Mechanism of Action – EVs deliver bioactive molecules directly to diseased cells, inducing regenerative healing.
- Safe and Scalable – EVs have low immunogenicity, ensuring compatibility across patient populations and enabling large-scale production.
- Resilient in Harsh Environments – Unlike live cells, EVs survive in harsh wound and gastrointestinal (GI) environments, making them ideal for topical and systemic administration.
Competitive Advantage Over VYJUVEK™
Currently, people with RDEB can be treated with the gene therapy VYJUVEK™, which requires viral vectors for gene delivery. These vectors pose potential safety concerns such as insertional mutagenesis and immune responses. In contrast, AGLE-102™ is a cell-free therapy that does not rely on viral vectors, eliminating these risks while maintaining strong regenerative potential.
Additionally, VYJUVEK™ is currently priced at approximately $1.3 million for the first year of treatment and $631,000 for each subsequent year, with limitations on the skin surface area that can be treated weekly.
AGLE-102™ is expected to be competitively priced, with a strong safety profile that may support broader treatment coverage over larger body areas. Aegle’s scalable EV manufacturing process further enhances accessibility by avoiding the complex and costly production requirements associated with gene therapies.
The Science Behind AGLE-102™
EVs play a crucial role in cell-to-cell communication, transferring proteins, mRNA, and microRNA to correct dysfunctional cellular pathways. AGLE-102™ delivers key biomolecules specific to DEB, including COL7A1 mRNA, which stimulates fibroblasts to produce collagen VII—a protein essential for strengthening the skin’s structure.
“By delivering COL7A1 and other regenerative molecules, AGLE-102 offers a multifaceted approach—not only addressing the genetic deficiency of collagen VII but also modulating inflammation and immune response,” explained Dr. Evangelos Badiavas, Co-Founder and Chief Scientific Officer of Aegle Therapeutics.
Clinical Trial Progress & Promising Early Results
The Phase 1/2a trial, titled: “A Safety Study of the Administration of Mesenchymal Stem Cell Extracellular Vesicles in the Treatment of Dystrophic Epidermolysis Bullosa Wounds,” is a prospective, non-randomized, multi-center study evaluating AGLE-102 in chronic DEB wounds. Early data from the first enrolled patient demonstrated remarkable wound closure, with 100% healing after just three doses. The treatment showed no adverse events, underscoring its excellent safety profile.
Regulatory Pathway and Market Availability
Aegle Therapeutics has received Fast Track and Rare Pediatric Disease Designation from the FDA, positioning AGLE-102™ for accelerated clinical development. The company plans to meet with regulatory authorities in the first half of 2026 following the completion of its ongoing Phase 1/2a study to align pivotal trial design.
If timelines remain on track, Aegle aims to submit a Biologics License Application (BLA) by 2029, bringing AGLE-102™ to market for patients in need.
Beyond DEB: A Platform Therapy for Other Indications
Aegle is also investigating AGLE-102™ in other high-impact dermatological and inflammatory diseases, including other epidermolysis bullosa subtypes due to the current success with RDEB severe burns are also of interest, because previous studies showed accelerated healing and reduced inflammation after a single dose.
Another disease of interest is graft-versus-host disease (GvHD). Aegle is exploring EVs’ potential in immunomodulation, and they have seen promising results in a bone marrow transplants murine model.
Aegle’s Vision & Next Steps
With the ability to modulate immune responses, reduce inflammation, and promote regenerative healing, AGLE-102™ has the potential to address a wide range of conditions beyond dermatology, including ophthalmic and orthopedic applications.
“Aegle is dedicated to advancing first-in-class regenerative therapies that provide meaningful and lasting benefits to patients with severe dermatological diseases,” Hartman told CheckOrphan. “We look forward to partnering with investors and biotech leaders to bring AGLE-102 to market.”
For more information, visit: www.aegletherapeutics.com
Contact
info@aegletherapeutics.com