UCLB licences Gene Therapy Program for Haemophilia A to BioMarin
Published on 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
“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 strengths 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.”
About Haemophilia A
The current market for haemophilia A products is about $6.0 billion worldwide. There are approximately
90,000 patients in territories where BioMarin has commercial operations and an annual incidence of
about 400 new patients in the U.S. The standard of care for the 60 percent of haemophilia A patients who
are severe is a prophylactic regimen of IV infusions three times per week. Even with the likely prospect of less frequently dosed products coming to the market, feedback from thought leaders indicates that
significant unmet need will remain as factor replacement therapy will inevitably leave patients vulnerable to bleeding events. Many patients on factor replacement therapy still have bleeding events and experience debilitating damage to joints as a result of chronically low factor levels.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include BMN-110 (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit www.BMRN.com.
UCLB is a leading technology transfer company that supports and commercialises research and innovations arising from UCL, one of the UK’s top research-led universities.
UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from the laboratory to market.
UCLB supports UCL’s Grand Challenges of increasing UCL’s positive impact on and contribution to Global Health, Sustainable Cities, Intercultural Interaction and Human Wellbeing.
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