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Aim Epidermolysis bullosa (EB) is a rare, inherited skin disorder that causes extreme fragility, leading to blistering and tearing from minimal friction1. There are limited treatment options, particularly for paediatric patients, who experience significant pain, have an increased infection risk, and impaired quality of life. 1 This initiative aimed to introduce and evaluate Beremagene Geperpavec (BVEC) therapy, the UK’s first unlicensed topical gene therapy for EB, in a two-year-old child.2 BVEC therapy is applied in a hospital setting to open wounds weekly until they are closed. The objective was to assess its feasibility, safety, and early clinical impact, paving the way for wider adoption in paediatric EB care.Method BVEC therapy was initiated within the organisation following a multidisciplinary approach involving paediatric consultant dermatologists, dermatology registrars, clinical nurse specialists, wider nursing team as well as specialist pharmacists.Key steps included Regulatory and Ethical Approval:Multidisciplinary team (MDT) discussions to ensure adherence to national guidance provided by the specialist pharmacy services working group for advanced therapeutic medicinal products were utilised to support implementation of this unlicensed gene therapy.3 Internal approval was sought from the Drug and Therapeutics Committee, Genetically Modified Organism Safety Committee (GMOSC) and ethics committee. Subsequently, patient consent was obtained prior to initiating BVEC therapy.Preparation and Administration:Safe handling, storage, reconstitution, receipt of medication and administration requirements were discussed with the wider MDT and GMOSC prior to implementing BVEC in a paediatric setting; this resulted in the development of a standard operating procedure.Clinical Monitoring and Outcome Assessment:Clinical pictures of wounds were undertaken on a weekly basis, to evaluate wound healing, signs of infection, reduction in blisters as well as overall patient tolerability. All pictures were dated to ascertain duration of wound closure.Family and Carer Education:Parents and caregivers were supported throughout BVEC initiation and involved in MDT conversations to ensure safe and effective application. Furthermore, play specialists were utilised during BVEC treatment to improve patient experience.Results BVEC therapy has been successfully adopted in a paediatric tertiary centre which has demonstrated:Improved duration of wound closure and enhanced skin integrity with no reported adverse effectsReduction in infection frequency, mitigating the need for continuous systemic antibiotics.BVEC has proven feasible for an individual patient; however, implementation on a larger scale will require more strategic planning to ensure efficiency and sustainability.Conclusion The successful introduction of BVEC therapy in this paediatric patient since November 2024, marks a significant advancement in EB treatment. As the first UK centre to implement this novel therapy, our experience provides a framework for wider adoption in both paediatric and adult settings. This case highlights the critical role of specialist pharmacists to support safe administration of gene therapies to patients whilst ensuring all teams comply to local governance processes to mitigate adverse outcomes to patients and operators. Further long-term studies are needed to assess sustained efficacy and broader application of BVEC. Overall, continued research and multi-disciplinary collaboration is paramount to improve outcomes for children with EB.References DEBRA the butterfly skin charity. Available online from: Lifestage-graphic-2000px.jpg (2000×436) Date Accessed [22-06-2025].Vyjuvek. Beremagene Geperpavec-svdt. Available online from: VYJUVEK® (beremagene geperpavec-svdt) DEB Treatment Date Accessed [22-06-2025].Specialist Pharmacy Services. Pan UK Pharmacy Working Group for ATMPs Gene Therapy Medicinal Products Governance and Preparation Requirements. Available online from: PAN-UK-PWG-for-ATMPs-Gene-Therapy-Guidance-V3.pdf Date Accessed [22-06-2025].