STUDY DESIGN: This is a phase 1/2A, non randomized, multi-center, ascending dose, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.~Eligible subjects will undergo a one-month observation period to confirm that the targeted wound is chronic (only single wounds with evidence of less than 20% closure over that period will be eligible for treatment). Once this has been established, up to 6 administrations of BM-MSC EVs will occur, at each to be given over a period of no more than 3 months. A maximum of 50 cm2 in total wound surface will be treated, and each administration will occur 14 days (+/- 7 days) but no less than 7 days apart. If the wound closes prior to 6 administrations, no additional doses will be given. Wound closure will be determined by complete re-epithelialization that is not subject to re-injury during dressing changes or as a result of normal daily activities (e.g. wearing clothing, eating, sleeping). After the 6 doses of BM-MSC EVs are given, the wound will be followed monthly for a period of 4 months to the termination of the study at 8 months or, in the event the wound closes before receiving all 6 doses, for 4 month after the wound closes. The ARANZ SilhouetteStarTM will be used to measure the target lesion at all visits.~STUDY OBJECTIVES:~Primary Objective: The primary objective is to determine the safety of applying single administrations of 2 ascending dose levels of EVs derived from allogeneic MSCs to DEB wounds between 10 and 50 cm2 that have persisted for more than one month~Secondary Objectives: The secondary objectives are to determine the safety of applying multiple administrations of 2 ascending dose levels of EVs derived from allogeneic MSCs to DEB wounds between 10 and 50 cm2 that have persisted for more than one month and to determine if there is clinical benefit (>50% closure) of applying BM-MSC EVs to DEB wounds~PLANNED SAMPLE SIZE: 10 subjects will be treated on the protocol with AGLE-102.
INVESTIGATIONAL PRODUCT: AGLE-102 is an allogeneic derived extracellular vesicle (EV) product derived from normal donor mesenchymal stem cells (MSCs).~INDICATION AND RATIONALE: The aim of the study is to assess the safety and efficacy of AGLE-102 in the treatment of lesions in subjects with Epidermolysis Bullosa (EB).~STUDY DESIGN: This is a phase 1/2A, non randomized, multi-center, ascending dose, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.