🦋 Gene Therapy for Epidermolysis Bullosa: Advances in Pediatric Treatment and Accessibility for Adults
Published 28 October 2026
Last update 03 November 2026
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25–31 October is International Epidermolysis Bullosa Awareness Week. In this rare inherited disease, the skin and mucous membranes are damaged even by minor mechanical impact. Patients require daily care, special dressings, and assistance from specialists of various profiles.
The "Butterfly Children" foundation supports about 2,600 patients with epidermolysis bullosa and ichthyosis in Russia and the CIS countries. There are 15 regional centres for genodermatoses in Russia where medical and psychosocial assistance can be obtained. "Butterfly Children" Foundation, foundation information on the ASI website.
What Has Changed in Treatment
An important achievement has been the emergence of gene therapy for dystrophic epidermolysis bullosa.
The "Circle of Good" foundation procures beremagene geperpavec (Vyjuvek). In 2025, 69 children and adolescents from 31 regions of Russia began receiving treatment; in 2026, 12 more patients joined the programme. Foundation information on the ASI website.
The product is applied to wounds as a gel. It delivers functional copies of the COL7A1 gene into cells, ensuring the production of type VII collagen, which is necessary for binding the layers of the skin. This promotes healing of the treated wounds. The therapy involves repeated application, and the need for comprehensive care remains. In the US, Vyjuvek is approved for the treatment of wounds in children and adults with dystrophic epidermolysis bullosa associated with COL7A1 mutations. FDA prescribing information.
In April 2025, the FDA approved another product — Zevaskyn, intended for children and adults with the recessive dystrophic form of the disease. For its manufacture, the patient's own skin cells are genetically modified by introducing functional copies of COL7A1. Cell sheets are then grown and grafted onto the wounds. FDA materials.
Therapy for Other Manifestations of the Disease
The investigational product KB803 is being studied in the Phase III IOLITE trial for the treatment and prevention of corneal erosions in dystrophic epidermolysis bullosa. According to the developer, key results are expected in Q4 2026. This area is still at the clinical trial stage. Krystal Biotech announcement.
Access to Treatment After Adulthood
The Russian provision programme through "Circle of Good" is focused on children, with support for beneficiaries continuing until they reach 19 years of age. Therefore, determining the further source of funding for treatment in advance is a necessary condition for its continuity. The "Butterfly Children" foundation notes that provision for adult patients remains one of the most acute problems. Foundation information on the ASI website.
Meanwhile, according to data presented by Alexander Materikin in October 2026, adults account for 41% of the more than one thousand known patients with epidermolysis bullosa in Russia. MIR24 report.
The issue of financing therapy for adult patients with rare diseases remains unresolved, especially for those not covered by the "14 VZN" programme. At the same time, the high cost and other characteristics of high-tech products (including gene therapy) cast doubt on the possibility of funding them even for the nosologies included in this programme.
According to data for Q3 2026, 49 gene therapy products have been approved worldwide, including for the treatment of adults. ASGCT and Citeline report.
Expanding treatment options requires the development of new sources and mechanisms of financing for adult patients. Also, reaching adulthood should not become a reason for interrupting treatment already started. Achievements in providing therapy to children lose their meaning if, upon transition to adulthood, treatment becomes inaccessible.
The "Butterfly Children" foundation supports about 2,600 patients with epidermolysis bullosa and ichthyosis in Russia and the CIS countries. There are 15 regional centres for genodermatoses in Russia where medical and psychosocial assistance can be obtained. "Butterfly Children" Foundation, foundation information on the ASI website.
What Has Changed in TreatmentAn important achievement has been the emergence of gene therapy for dystrophic epidermolysis bullosa.
The "Circle of Good" foundation procures beremagene geperpavec (Vyjuvek). In 2025, 69 children and adolescents from 31 regions of Russia began receiving treatment; in 2026, 12 more patients joined the programme. Foundation information on the ASI website.
The product is applied to wounds as a gel. It delivers functional copies of the COL7A1 gene into cells, ensuring the production of type VII collagen, which is necessary for binding the layers of the skin. This promotes healing of the treated wounds. The therapy involves repeated application, and the need for comprehensive care remains. In the US, Vyjuvek is approved for the treatment of wounds in children and adults with dystrophic epidermolysis bullosa associated with COL7A1 mutations. FDA prescribing information.
In April 2025, the FDA approved another product — Zevaskyn, intended for children and adults with the recessive dystrophic form of the disease. For its manufacture, the patient's own skin cells are genetically modified by introducing functional copies of COL7A1. Cell sheets are then grown and grafted onto the wounds. FDA materials.
Therapy for Other Manifestations of the DiseaseThe investigational product KB803 is being studied in the Phase III IOLITE trial for the treatment and prevention of corneal erosions in dystrophic epidermolysis bullosa. According to the developer, key results are expected in Q4 2026. This area is still at the clinical trial stage. Krystal Biotech announcement.
Access to Treatment After AdulthoodThe Russian provision programme through "Circle of Good" is focused on children, with support for beneficiaries continuing until they reach 19 years of age. Therefore, determining the further source of funding for treatment in advance is a necessary condition for its continuity. The "Butterfly Children" foundation notes that provision for adult patients remains one of the most acute problems. Foundation information on the ASI website.
Meanwhile, according to data presented by Alexander Materikin in October 2026, adults account for 41% of the more than one thousand known patients with epidermolysis bullosa in Russia. MIR24 report.
The issue of financing therapy for adult patients with rare diseases remains unresolved, especially for those not covered by the "14 VZN" programme. At the same time, the high cost and other characteristics of high-tech products (including gene therapy) cast doubt on the possibility of funding them even for the nosologies included in this programme.
According to data for Q3 2026, 49 gene therapy products have been approved worldwide, including for the treatment of adults. ASGCT and Citeline report.
Expanding treatment options requires the development of new sources and mechanisms of financing for adult patients. Also, reaching adulthood should not become a reason for interrupting treatment already started. Achievements in providing therapy to children lose their meaning if, upon transition to adulthood, treatment becomes inaccessible.
