Allogeneic ABCB5-positive Limbal Stem Cells for Treatment of LSCD

This is an interventional, open-label, phase I/IIa clinical trial to investigate the efficacy
and safety of up to four doses of the IMP topically administered on the target eye of
patients with LSCD. Patients will be treated in up to four ascending dose groups.

The allogeneic investigational product LSC2 contains ABCB5-positive limbal stem cells (from
corneal rims of cadaveric donors, expanded ex vivo, isolated and stored in a donor cell
bank).

The IMP will be applied on the target eye. Prior to application, the conjunctival pannus will
be removed under general anesthesia.

Patients will be followed up for efficacy for 1 year. Efficacy of the IMP will be monitored
by assessing neovascularization and epithelial defects.

To assess long-term safety of LSC2 one follow-up visit at Month 24 post IMP application is
included.

Inclusion Criteria:

1. Male or female patients aged 18 to 85 years

2. Patients with bilateral or unilateral LSCD e.g. due to thermal and/or chemical burn or
equivalent disease (injury that caused LSCD at least 6 months prior to inclusion)

3. Neovascularization: Vessel penetration of at least 2 quadrants, with central cornea
involved

4. Patients understand 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 blood pregnancy test at Visit 1

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

Exclusion Criteria:

1. Compromised eyelid mobility and/or symblepharon; patient can be re-screened after
appropriate treatment

2. Presence of eyelid malposition; patient can be re-screened after appropriate treatment

3. Active local ocular or systemic infection and/or inflammation. Patient can be
re-screened after infection and/or inflammation is resolved.

4. Tumor diseases or history of tumor disease

5. Active ocular neoplastic disease (exclusion will be based on investigator's
assessment)

6. Corneal erosion or ulcer is bigger than 4 mm2; corresponding to less than 95% of
continuous corneal epithelium. Patient can be re-screened after erosion or ulcer is
resolved (≤ 4 mm2).

7. Positive for human immunodeficiency virus (HIV) 1 and/or 2 (diagnosed by serologic
testing)

8. Any known allergies to components of the IMP or per protocol pre-planned concomitant
medications

9. Contraindications to the surgical procedure (e.g. removing of the conjunctival pannus)

10. Clinically significant or unstable concurrent disease or other clinical
contraindications to stem cell transplantation

11. Further clinical contraindications to IMP application (exclusion will be based upon
investigator's judgment)

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

13. Previous participation in this clinical trial (except screening failure due to
inclusion criterion 2 and/or exclusion criterion 1 and/or 2 and/or 3 and/or 6)

14. Known abuse of alcohol, drugs, or medicinal products

15. Patients unwilling or unable to comply with the requirements of the protocol

16. Lactating women

17. Evidence of any other medical conditions (such as psychiatric illness, 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

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