UCL Innovation and Enterprise


UCL Business licenses gene therapy programme for haemophilia to BioMarin Pharmaceuticals

22 February 2013

UCLB today announced that it has licensed a Factor VIII gene therapy program for haemophilia A to BioMarin Pharmaceuticals Inc (BMRN) using the research from Professor Amit Nathwani and his team at UCL and  St. Jude Children’s Research Hospital.

UCL Business

“Gene therapy is emerging as a powerful and viable way to treat genetic disorders and is complementary to our current suite of commercial products and research programs,” said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. “Haemophilia is an attractive target for gene therapy as factor levels in the blood serve as good biomarkers, relatively low factor levels are required for a clinically important benefit in severe patients and the current standard of care of intravenous infusions three times a week is quite onerous.

“We remain committed to maintaining a rich pipeline with the goal of filing an IND every twelve to eighteen months.”

Mr. Cengiz Tarhan, Managing Director of UCLB said, “This is an excellent partnership for UCLB, which combines the world class translational research strenghts of Professor Nathwani and his team with the significant development and commercialisation capabilities of BioMarin to progress  this ground breaking therapy for haemophilia A”.

Professor Stephen Caddick, Vice-Provost (Enterprise) at UCL added, “UCL and BioMarin each bring distinct strengths to the partnership.

“UCL is a world leader in the biomedical sciences, with an unremitting commitment to outstanding research and translation into healthcare benefits for patients.

“We welcome this partnership which will continue to build on the excellence of our research to fully explore the potential of gene therapy as a life-saving treatment for people with haemophilia.”