UCL Innovation and Enterprise


Gene Therapy at UCL: a powerful new way to treat haemophilia A

26 February 2015

An urgent medical challenge


Haemophilia A is a life-threatening genetic condition that affects the blood’s ability to clot and causes increased or spontaneous bleeding. Mostly affecting men, it occurs in about 1 of every 6,000 male births in the UK and crosses all racial and ethic groups, with approximately one boy in every 5,000 born with it worldwide.

Haemophiliacs lack the blood-clotting protein Factor VIII, which is vital for blood coagulation. There is currently no cure for the condition, and treatment is demanding both in terms of time and cost. This means that its availability is limited to a subset of patients in the most economically advanced countries.

New frontiers

A long-awaited breakthrough occurred in February 2013, when UCL Business (UCLB) signed a licensing agreement with US biotechnology firm Biomarin to develop a new Factor VIII gene therapy programme. The agreement came after a 2011 trial in which the Royal Free London NHS Foundation Trust reported that six people with haemophilia B saw a reduction in symptoms after gene therapy on liver cells. This breakthrough might pave the way for potentially curative life-saving treatment to become widely available.

Gene therapy goes global

Gene therapy inserts therapeutic DNA into a patient’s cells, usually using DNA that encodes a functional gene to replace a mutated one. It’s a relatively new way of treating diseases, functioning as a direct treatment to the root cause. The Royal Free trial was the first ever to report positive results using it. The agreement with BioMarin, as Professor Amit Nathwani of the UCL Cancer Institute and Director of the Katherine Dormandy Haemophilia Centre and Thrombosis Unit at the Royal Free London NHS, says: “brings us an ability to scale up everything we’re doing so that a larger number of patients can benefit from the advances we’ve made in the laboratory.”

The right conditions for treatment

The deal was brokered by UCLB, who supported it by taking Professor Nathwani’s team through negotiations and contracts, helping them rapidly find an agreed basis for continued collaboration.

Cengiz Tarhan, Managing Director of UCL Business says, “This is an excellent partnership for UCL Business, which combines the world-class translational research strengths of Professor Nathwani and his team with the significant development and commercialization capabilities of BioMarin to progress this groundbreaking therapy for haemophilia A.” This achievement was recognised in May 2014 when Professor Amit Nathwani won the UCL Business Award of the Year at the UCL Awards for Enterprise 2014.