Gene Therapy's New Frontier: ZEVASKYN Expansion Redefines RDEB Care

CLEVELAND, OH – December 11, 2025 – In a significant move that bridges the gap between breakthrough science and patient access, Abeona Therapeutics has activated The University of Texas Medical Branch (UTMB) as a new Qualified Treatment Center (QTC) for its novel gene therapy, ZEVASKYN. This expansion into Texas is more than a logistical update; it represents a critical step in the complex, high-stakes rollout of one of the first disease-modifying treatments for recessive dystrophic epidermolysis bullosa (RDEB), a devastating rare genetic disorder.

For patients living with RDEB, often called 'butterfly children' due to their extremely fragile skin, life is a constant cycle of painful blistering, chronic wounds, and relentless daily care. The announcement offers a tangible beacon of hope, expanding the reach of a therapy designed not just to manage symptoms, but to correct the underlying genetic defect at the wound site. As these advanced therapies move from the lab to the clinic, Abeona's strategy highlights the intricate dance of innovation, market strategy, and risk management required to commercialize medicine's next frontier.

A New Paradigm for a Devastating Disease

Recessive dystrophic epidermolysis bullosa is caused by a defect in both copies of the COL7A1 gene, rendering the body unable to produce functional type VII collagen. This protein is the essential 'glue' that anchors the layers of the skin together. Without it, the slightest friction or trauma can cause the skin to separate, leading to severe wounds that often fail to heal, leading to infection, debilitating pain, and an increased risk of aggressive skin cancers. The lifetime cost of care for a patient with RDEB can soar into the millions, a figure that only hints at the immense physical and emotional toll on patients and their families.

ZEVASKYN (prademagene zamikeracel) represents a fundamental shift in treating this condition. It is an autologous, gene-modified cellular sheet therapy. The process is a marvel of personalized medicine: physicians take a small biopsy of a patient's own skin cells, which are then sent to Abeona's manufacturing facility in Cleveland. There, the cells are genetically modified ex vivo using a retroviral vector to carry a functional copy of the COL7A1 gene. These corrected cells are then cultured and grown into sheets, which are surgically applied to the patient's most severe, chronic wounds in a single procedure.

The results from clinical trials are compelling. The pivotal Phase 3 VIITAL™ study showed that 81% of ZEVASKYN-treated wounds achieved at least 50% healing at six months, compared to just 16% of control wounds. Critically, the therapy also demonstrated a significant reduction in pain, a primary driver of poor quality of life in RDEB. As Dr. Michael G. Wilkerson, Chair of the Department of Dermatology at UTMB, noted, “We are proud to be a designated treatment center for ZEVASKYN and to be at the forefront of delivering this novel, FDA-approved gene therapy that offers new hope for healing debilitating wounds associated with this disease.”

The Strategic Importance of a Qualified Network

The activation of UTMB, home to the first department of dermatology in Texas, is a calculated move in Abeona’s commercial strategy. Gene therapies like ZEVASKYN are not off-the-shelf products. Their administration requires a highly specialized infrastructure, multidisciplinary medical teams, and rigorous logistical coordination. The Qualified Treatment Center model is Abeona’s answer to this complexity, ensuring that the therapy is delivered safely and effectively by institutions with proven expertise in complex skin diseases and wound care.

“Activation of UTMB as a QTC significantly expands patient access to ZEVASKYN across Texas and the Gulf Coast region,” said Dr. Madhav Vasanthavada, Abeona's Chief Commercial Officer. The selection of UTMB, with its renowned wound care clinic and deep experience in dermatologic surgery, creates a vital hub for patients in a vast geographical area who previously faced significant travel burdens for specialized care.

This strategic expansion also places ZEVASKYN within a dynamic and competitive market. Just last year, Krystal Biotech's Vyjuvek, a topical gene therapy gel for DEB, gained FDA approval. While both therapies target the same underlying genetic defect, their delivery and application differ significantly. Vyjuvek is applied weekly by a healthcare professional, making it suitable for managing various wounds over time. In contrast, ZEVASKYN’s one-time surgical application is positioned as a durable solution for large, intractable wounds. Analysts suggest the two therapies may ultimately prove to be complementary rather than purely competitive, offering clinicians a multi-faceted toolkit to manage this complex disease.

Navigating the Financial and Safety Frontiers

With innovation comes a staggering price tag. ZEVASKYN's list price is reported to be approximately $3.1 million per treatment, placing it among the most expensive therapies in the world. This cost presents a formidable barrier to access and a significant challenge for Abeona's commercialization efforts. To address this, the company is pioneering outcomes-based agreements with payers and has announced a warranty program that would reimburse a portion of the cost if the treated wound requires additional therapy within three years. This strategy aims to shift the financial risk, aligning the cost of the therapy with its long-term value and clinical success.

Navigating insurance coverage is paramount. With an estimated 60% of RDEB patients covered by commercial plans and 30% by Medicaid, Abeona is actively engaging payers. Early signs, such as Aetna’s policy deeming the therapy medically necessary for qualifying patients, are promising. For patients and families, programs like Abeona Assist® are indispensable, providing personalized support to navigate insurance benefits, financial assistance options, and the complex travel and logistical arrangements required for treatment.

Beyond the financial considerations lies the critical issue of long-term safety. Because ZEVASKYN works by integrating a new gene into a patient's cells, the FDA has noted a potential theoretical risk of cancer development and requires lifelong monitoring for patients. This is a common and crucial consideration for integrating gene therapies. To its credit, Abeona has been transparent about this risk, and data from clinical trials with a median follow-up of over three years have not shown any cases of squamous cell carcinoma—the most common cancer in RDEB patients—in treated wounds. The company is establishing a long-term observational study to continue monitoring patient outcomes, a necessary step in building confidence and a comprehensive understanding of the therapy’s lifelong impact.

As ZEVASKYN becomes available to more patients through centers like UTMB, it marks not only a new era for the RDEB community but also a case study in the evolution of healthcare. The successful deployment of such a complex, high-value therapy depends on a sophisticated ecosystem of specialized medical centers, innovative payment models, and robust long-term safety monitoring, creating a strategic blueprint for the future of personalized medicine.