A Breakthrough Year for Skin Care: Gene Therapy, New Treatments, and Precision Medicine

The year 2025 marked a revolutionary turning point in dermatology, with the FDA approving groundbreaking treatments including the first-ever gene therapy for a rare skin disorder and researchers discovering new biomarkers that could transform how we treat chronic itching conditions. These advances span from cutting-edge genetic therapies to precision medicine approaches that target the root causes of skin diseases.

What Makes the New Gene Therapy So Revolutionary?

In April 2025, the FDA approved Zevaskyn, the first autologous cell-based gene therapy for treating recessive dystrophic epidermolysis bullosa (RDEB), a devastating inherited disorder that causes severe skin fragility and chronic wounds. This one-time treatment harvests a patient's own skin cells, genetically modifies them to produce the missing protein, and cultures them into sheets that are surgically applied to wound sites.

The results from the phase 3 VIITAL study were remarkable: 81% of wounds treated with Zevaskyn showed 50% or more healing at six months, compared to just 16% of wounds receiving standard care. "The FDA approval of Zevaskyn marks a monumental step forward for individuals living with RDEB and their families, offering a much-needed, long-lasting treatment option for this devastating condition," said Dr. Anna L. Bruckner, co-director of the Epidermolysis Bullosa Clinic at Children's Hospital of Colorado.

How Are New Targeted Treatments Changing Skin Care?

Beyond gene therapy, 2025 brought two other significant FDA approvals that offer patients more precise treatment options. Anzupgo became the first therapeutic specifically indicated for moderate-to-severe chronic hand eczema in adults, affecting nearly one in 10 U.S. adults. This topical Janus kinase (JAK) inhibitor works by blocking inflammatory pathways and offers a steroid-free alternative for patients who haven't responded to traditional treatments.

The approval was based on two phase 3 trials where roughly one-fourth of patients treated with Anzupgo achieved clear or almost clear skin, compared to only 8% of those receiving placebo treatment. Additionally, Rhapsido, an oral Bruton's tyrosine kinase (BTK) inhibitor, was approved for chronic spontaneous urticaria, providing patients with an oral alternative to injectable therapies.

What Breakthrough Discovery Could Help Millions with Chronic Itch?

University of Maryland researchers made a groundbreaking discovery that could revolutionize treatment for millions suffering from chronic pruritus of unknown origin (CPUO) - a condition causing severe itching with no identifiable cause. The study found that patients with this condition have significantly lower levels of specific amino acids in their blood plasma compared to healthy individuals.

The research identified deficits in nine amino acids that correlate with itch severity, including important building blocks for neurotransmitters that play a role in the body's itch response. These findings provide the first concrete biomarkers for a condition that has long puzzled doctors and left patients without targeted treatments.

• Tryptophan and Glycine: Key amino acids found depleted in CPUO patients that serve as building blocks for neurotransmitters involved in itch response
• Immune System Metabolites: Several other metabolites involved in immune system regulation were found at lower than normal levels
• Correlation with Severity: The lower the amino acid levels, the more severe the itching symptoms experienced by patients

"Our study found a distinct deficit in certain metabolite biomarkers, including several important amino acids and other metabolites involved in immune system regulation in patients with CPUO compared to a healthy control group," said Dr. Shawn Kwatra, the Joseph W. Burnett Endowed Professor and Chair of Dermatology at the University of Maryland School of Medicine.

The discovery builds on previous animal studies showing that boosting neurotransmitters like serotonin with medications such as antidepressants reduced itch symptoms in mice. This research opens the door for developing targeted therapies that could address the underlying biochemical imbalances causing chronic itch rather than just managing symptoms.

These 2025 advances represent a shift toward precision medicine in dermatology, where treatments are tailored to the specific genetic, molecular, or biochemical factors driving each patient's condition. From gene therapies that correct fundamental genetic defects to biomarker discoveries that reveal the hidden causes of mysterious symptoms, the field is moving beyond one-size-fits-all approaches to truly personalized skin care.