Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

This is an interventional, single arm, non-randomized, open label, phase I/IIa clinical trial
to investigate the efficacy and safety of the IMP allo-APZ2-EB in patients with RDEB.

Patients will undergo treatment with the IMP (three repeated intravenous applications) and
will be followed up for efficacy for 12 weeks. To assess long-term safety of allo-APZ2-EB one
follow-up visit at Month 12 and one follow-up visit at Month 24 post IMP applications is
included.

Determination of the EB linked symptoms and quality of life will be assessed by using the
EBDASI score, the iscorEB, the change in pain and itch perception, and patient's quality of
life in EB. The wound healing process will be documented by photography.

Inclusion Criteria:

1. Male or female patients aged between ≥12 months and ≤55 years;

Staggered design for patient enrollment:

1. at least 3 adult patients (safety assessment 2 weeks after last treatment of third
patient),

2. at least 3 patients ≥12 to <18 years (safety assessment 2 weeks after first treatment
of third patient),

3. at least 3 patients ≥5 to <12 years (safety assessment 2 weeks after first treatment
of third patient), and

4. at least 3 patients ≥12 months to <5 years;

2. Diagnosed with RDEB (combined diagnosis by genotype assessment [mutation analysis] and
correlating phenotype assessment [wound assessment]);

3. Patient is eligible to participate in this clinical trial based on general health
condition at the investigator's discretion;

4. Patient/legal representative understands the nature of the procedure and are providing
written informed consent prior to any clinical trial procedure;

5. Women of childbearing potential must have a negative urine pregnancy test at Visit 1;

6. Women of childbearing potential and their partner must be willing to use highly
effective contraceptive methods during the course of the clinical trial.

Exclusion Criteria:

1. Tumor diseases or history of tumor disease;

2. Known positive result for human immunodeficiency virus 1 and/or 2;

3. Any known allergies to components of the IMP;

4. Evidence of any other medical conditions (such as psychiatric illness or active
infection) based on physical examination, or laboratory findings that may interfere
with the planned treatment, affect the patient's compliance, or place the patient at
high risk of complications related to the treatment; at investigators discretion;

5. History of prior thrombosis or patients at risk for thrombosis;

6. Clinically significant or unstable concurrent disease or other clinical
contraindications (based upon investigator's judgment);

7. Patient/legal representative anticipated to be unwilling or unable to comply with the
requirements of the protocol;

8. Pregnant or lactating women;

9. Current or previous (within 30 days of enrollment) treatment with another IMP, or
participation and/or under follow-up in another clinical trial;

10. Previous participation in this clinical trial (except for screening failures due to an
exclusion criterion);

11. Known abuse of alcohol, drugs, or medicinal products;

12. Employees of the sponsor, or employees or relatives of the investigator.