A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

RDEB is a congenital and progressive orphan skin disease caused by the deficiency of the
protein type VII collagen (COL7). The objective of this study is evaluate the safety FCX-007
intradermal injections in RDEB subjects. Additionally, the trial will evaluate type VII
collagen expression, the presence of anchoring fibrils resulting from FCX-007, as well
evidence of wound healing.

Six adult subjects are expected to be treated with FCX-007 in the Phase I portion of the
trial and six subjects age 7 or older in the Phase II portion of the trial. All subjects will
receive FCX-007 to one or more paired target RDEB wounds. Proof of mechanism will be
monitored through digital photography of target wounds and assays conducted on biopsies taken
from target wounds.

Inclusion Criteria:

1. Age

1. Phase I (subjects 1 through 6): Eighteen (18) years or older.

2. Phase II (subjects 7 through 12): Seven (7) years or older.

2. Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)

3. NC1/NC2 Status (to be tested for if unknown)

4. Subjects, who are, in the opinion of the Investigator, able to understand the study,
co-operate with the study procedures and are willing to return to the clinic for all
the required follow-up visits

Exclusion Criteria:

1. Medical instability limiting ability to travel to the investigative center.

2. Active infection with HIV, hepatitis B or hepatitis C

3. A positive study specific immunofluorescence result

4. Evidence of systemic infection

5. Current evidence of metastatic squamous cell carcinoma at the site to be injected

6. Known allergy to any of the constituents of the product

7. Active drug or alcohol addiction

8. Hypersensitivity to type of anesthesia chosen

9. Receipt of a chemical or biological study product for the specific treatment of RDEB
in the past six months

10. Women who are pregnant or breast-feeding

11. Abnormal clinically significant laboratory result

12. Clinically significant abnormalities on NCI toxicity scale