Europe’s Bold Foray into Next-Gen Therapies

The European continent is emerging as a formidable force in the global cell and gene therapy landscape through first-ever product approvals and expansion of manufacturing facilities. The growing demand for advanced therapies that beat the untreatable conditions, strong regulatory frameworks, skilled professionals, and government backing are positioning Europe at the forefront in cell and gene therapies.

Is VYJUVEK, Europe’s First Topical Gene Therapy, a Beacon of Hope for Rare Skin Disease Sufferers?

April 28, 2025, Krystal Biotech, an emerging biotech company, achieves European approval for VYJUVEK (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB), an ultra rare skin disease. This milestone is a major step forward in the topical gene therapy era in Europe. This approval is bringing new hope to dystrophic epidermolysis bullosa patients.

Will Krystal Biotech acquire a major share in topical gene therapy market in Europe?

DEB is an ultra-rare genetic disease that impacts the expression of messenger gene, which is important in collagen production. Due to this genetic mutation, the upper and mucosal skin becomes highly sensitive, and gentle friction leads to chronic wounds and scarring. According to Orphanet, the prevalence of DEB is between 1 in 120,0000-350,000 holding an immense market opportunity.

Cristina Has, M.D. professor and dermatologist in Germany at the University of Freiburg, stated thatVyjuvek has a bright future, as it addresses the very first stage in the mechanism of DEB.

What was the Efficacy Rate of VYJUVEK During Clinical Evaluation?

VYJUVEK is a genetically modified herpes-simplex virus type 1 (HSV-1) vector-based gene therapy that delivers human collagen VII alpha 1. It is manufactured to address gene mutation through the introduction of HSV-1 viral vector carrying functional copies of COL7A1, to provide wound recovery. This suspension is applied to the DEB wounds once a week. This gene therapy induces fibroblasts to produce functional COL7, which reconstructs skin. 

VYJUVEK has demonstrated robust clinical results in DEB:

• 71% of the wounds treated completely healed by 3 months
• Wounds treated for 6 months were healed by 67%
• However, placebo-treated wounds healed by only 20%–22%

Chairman & CEO of Krystal Biotech, Krish S. Krishnan, stated that “the very first approval of VYJUVEK as a topical gene therapy is a new beginning to improve treatment of genetic diseases, and it is an important milestone for rare disease patients affected by DEB."

As Europe embraces novel therapies like VYJUVEK through progressive regulatory pathways, the need for robust, scalable manufacturing infrastructure becomes critical need. Thus, growing emphasis on accessibility and commercialization of cell and gene treatments is now being matched with strategic industrial investments. One such example is Ferring Pharmaceuticals’ recent expansion of its manufacturing facility in Finland.

Ferring’s Finland Facility Will Power Global Supply Chain for Gene Therapy?

In October 2024, Ferring Pharmaceutical, a global biopharmaceutical company, announced the opening of a global manufacturing facility for Adstiladrin, a non-replicating gene therapy, in Finland. This is also a significant milestone in Europe’s emergence as a competitive region in biologics manufacturing. This manufacturing hub is spread across 50,000 square feet, and it is designed for the production of Adstiladrin only.

Adstiladrin is currently the only FDA-approved intravesical, non-replicating gene therapy for bladder cancer. The global burden of bladder cancer, combined with Adstiladrin’s pioneering position, presents a significant market opportunity, one that Ferring is strategically addressing through this facility and regional supply chain optimization.

The facility expansion closely followed the U.S. commercial launch of Adstiladrin in January 2024. With the therapy gaining traction across the U.S., the need for a robust and geographically diversified manufacturing base became evident. According to Bipin Dalmia, Global Head of Uro-Oncology at Ferring, the Finnish plant is integral to ensuring “stable and sustainable global supply” in response to growing demand.

Moreover, Armin Metzger Executive Vice President & CTO said, “Such growth and continue investment and expansion of our gene therapy manufacturing facility, highlights the Ferring’s commitment to provide advanced treatments for patients with bladder cancer.”

Why Finland?

Finland is known for its streamlined regulated framework and skilled workforce, which makes it an emerging hotspot in the Nordic region. This newly opened site is equipped with fully integrated modern technologies, cutting-edge manufacturing suites that help in the development and commercialization of vector-based advanced therapies in large facilities. As per Armin Metzger, Executive VP and CTO at Ferring, the facility reinforces the company’s long-term commitment to offering advanced therapies for complex diseases such as bladder cancer, which remains the 9th most common cancer worldwide. According to the American Cancer Society, 84,870 new cases of bladder cancer are expected in 2025, underlining the urgency and commercial opportunity for such therapies.

Industry and Economic Implications:

• Europe a strategic hub for CGT innovation backed by regulatory frameworks: VYJUVEK has also received an orphan medicine designation as it improves the health of patients suffering from rare diseases. This therapy will also be launched in Liechtenstein, Norway, and Iceland.
• Strengthened Biotech Manufacturing Sovereignty: Ferring’s facility in Finland supports the localization of advanced manufacturing, helping reduce reliance on non-European supply chains.
• Economic and Employment Growth: The establishment of advanced therapy manufacturing sites fuels regional economic development, creates high-value jobs, and stimulates biotech cluster formation in areas like Finland and the broader Nordic region.