The NIHR/Wellcome Trust Imperial Clinical Research Facility (CRF) last week welcomed the enrolment of the first patient in a phase 1/2 trial which is studying a new approach to the therapy of haemophilia A using gene therapy.
The investigational gene therapy, called BMN270, is designed to restore coagulation factor VIII plasma concentrations in people with haemophilia A, who are either missing or have a defective version of this protein that is essential for blood clotting.
This study will be evaluating the safety and efficacy of BMN270 gene therapy in up to 12 patients with severe haemophilia A, with patients receiving the therapy and then being monitored for a further five years.
The therapy has been developed by biotechnology company BioMarin, whose representatives were at the CRF to observe the first patient being enrolled.
The study is also supported by the NHS Imperial College Healthcare Trust’s pharmacy team, who provide oversight and preparation of the infusions.
The gene therapy programme for haemophilia A was originally licensed from University College London and St. Jude Children’s Research Hospital in February 2013 and has since been developed at BioMarin’s facilities.
NIHR Clinical Research Facility enrol first patient into ground breaking trial