Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing Stem Cells Genetically Modified for Epidermis Restoration in Patients With Junctional Epidermolysis Bullosa

Inclusion Criteria:

1. Signed and dated informed consent prior to any study-related procedures.

2. Male and female patients between 6 months and 65 years old;

3. Diagnosis of generalized intermediate LAMB3-dependent JEB, confirmed by NGS orSanger sequencing and/or immunofluorescence;

4. Detectable residual expression of laminin 332 (and its beta-3 chain) byimmunofluorescence and/or Western Blot analysis;

5. Presence of blisters and/or ≥ 6 cm2 erosions (persistent or recurrent for more than 3 months); the area of the erosion can be considered as the sum of smaller areas inthe same body part (i.e. leg, thigh, arm, ...)

6. A cooperative attitude to follow the study procedures (caregivers in case ofchildren);

7. Patients' compliance with the study schedule and procedures, including completeimmobilization of the transplanted areas for at least two weeks and hospitalizationup to 1 month after transplantation.

Exclusion Criteria:

1. Known or suspected intolerance to anaesthesia;

2. Bad general condition (ECOG index >1);

3. Presence of any skin cancers in the area(s) qualified for treatment;

4. Clinical and/or laboratory signs of acute systemic infections at the time ofscreening. Patient can be re-screened after appropriate treatment;

5. Female subjects: Pregnant (as evident by a positive urine hCG or serum-hCG test) orlactating women and all women physiologically capable of becoming pregnant (i.e.women of childbearing potential [WOCBP]) UNLESS they are willing to use highlyeffective birth control methods;

6. Allergy, sensitivity or intolerance to study medication excipients or other materialrequired by study protocol (as per Investigator's brochure)

7. Contraindications to the local or systemic antibiotics and/or corticosteroidsforeseen by the protocol;

8. Contraindications to undergo extensive surgical procedures;

9. Presence of i) systemic diseases, ii) clinically significant or unstable concurrentdisease, iii) other concomitant medical conditions, iv) other clinicalcontraindications to stem cell transplantation, which based on Investigator'sjudgment, in consultation with the Sponsor Medical Expert may affect theparticipation in the study or the grafting procedure;

10. Patients (or parents in case of paediatric subject) unlikely to comply with thestudy protocol or unable to understand the nature and scope of the study or thepossible benefits or unwanted effects of the study procedures and treatments.

11. Previous treatments or clinical trials envisaging the use of cells (including bonemarrow transplantation, BMT) and/or both in vivo or ex vivo gene therapy products.