A phase 2 clinical trial of type VII collagen gene therapy for recessive dystrophic epidermolysis bullosa (Marinkovich 3)Completed
|Prof Peter Marinkovich
|Stanford University, Stanford, USA
|Start date / Duration
|01. Mar 2017 / 41 months
|Funder(s) / Co-Funder(s)
|DEBRA Austria, MSAP/EBEP Recommended
|Skin cancer & fibrosis, Molecular therapy
Short lay summary
The project aimed to develop an improved stem cell therapy for RDEB. Biopsies were to be used to study the stem cells before transplantation to patients and the behaviour and survival of the stem cells in the grafts. It was recommended for funding by MSAP through CliniCall 2015. The 3-year project had an original duration of 2017 to 2021, but a report received in early 2020 indicated that minimal progress had been made on one objective (developing improved methods for assessing patient suitability for transplantation). There was ambiguity on another objective. Ultimately, the project had to be terminated early due to contractual disagreements.
One positive outcome is some work the group has completed on Objective 1A, which relates to identifying patients for whom stem cell transplantation would be appropriate.
For the reasons explained above, Stanford's project contract was terminated on 2 August 2020.