A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Inclusion Criteria:

   1. Be at least 16 years of age.

   2. Has signed the current approved informed consent form.

   3. Has a diagnosis of RDEB based on genetic analysis and consistent with a recessive
   inheritance pattern.

   4. Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.

   5. Has at least 1 unhealed wound 10-200 cm2 for at least 6 weeks at the Screening Visit.

   6. Agrees to use contraception as follows:

      - For women of childbearing potential (WOCBP) agrees to use highly effective
      contraceptive (including abstinence) methods from Screening, through the study,
      and for at least 10 weeks after the last dose of study drug. Non-childbearing
      potential is defined as a female who meets either of the following criteria: age
      ≥50 years and no menses for at least 1 year or documented hysterectomy, bilateral
      tubal ligation, or bilateral oophorectomy (see Section 7.4.1.2 for details on the
      definition of non-childbearing potential).

      - For males, agrees to use a condom with any WOCBP sexual partner from Day 1 of
      study treatment, through the study, and at least 10 weeks after the last dose of
      study drug.

   7. Be willing and able to comply with this protocol.

Exclusion Criteria:

   1. Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.

   2. Is pregnant or nursing.

   3. Has received in the last six months any investigational gene therapy product or in the
   last three months any non-gene therapy investigational products.

   4. Is anticipated to receive new regimens of antibiotics or other anti-infectives during
   the trial.

   5. Has any other medical or personal condition that, in the opinion of the Investigator,
   may potentially compromise the safety or compliance of the patient, or may preclude
   the patient's successful completion of the clinical study.