This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15 years post gene therapy treatment. No investigational drug product will be administered during this study.
Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.
Rocket Pharmaceuticals (RCKT) is the sponsoring company for this trial. BiotechSign currently grades this company F (28/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.